NASDAQ: BNTC

Our proprietary technology platform is designated as DNA-directed RNA interference, or “ddRNAi”, and “silence and replace.” ddRNAi is designed to produce permanent silencing of disease-causing genes, by combining RNA interference, or RNAi, with viral delivery agents typically associated with the field of gene therapy (i.e., viral vectors). Modified AAV vectors are employed to deliver genetic constructs which encode short hairpin RNAs that are, then, serially expressed and processed to produce siRNA molecules within the transduced cell for the duration of the life of the target cell. These newly introduced siRNA molecules drive permanent silencing of the expression of the mutant gene. The silence and replace approach further bolsters the biological benefits of permanent silencing of disease-causing genes by incorporating multifunctional genetic constructs within the modified AAV vectors to create an AAV-based gene therapy agent that is designed to silence the expression of disease-causing genes (to slow, or halt, the underlying mechanism of disease progression) and to simultaneously replace the mutant genes with normal, functional genes.

Overview of RNAi and the siRNA Approach

RNA interference ("RNAi") is a biological mechanism that employs small nucleic acid molecules, called small interfering RNAs ("siRNAs"), to temporarily silence the expression of disease-causing genes. While several companies have demonstrated clinical efficacy using siRNA-based approaches, these approaches have a number of key limitations, including the need for repeat dosing to maintain efficacy, unstable therapeutic concentrations over time, long-term patient compliance challenges, potential adverse immune responses, and the inability to simultaneously replace defective genes with normally functioning genes. Our proprietary silence and replace technology is designed to overcome these limitations by combining permanent gene silencing with functional gene replacement following a single administration.

Our Approach to the Treatment of Genetic Diseases—ddRNAi and Silence and Replace

Our proprietary silence and replace approach to the treatment of genetic diseases combines RNAi with functional gene replacement to permanently silence the mutant genes and replace with functional genes potentially providing a permanent solution with a single administration of the therapeutic agent. Benitec employs ddRNAi in combination with classical gene therapy (i.e., transgene delivery via viral vectors) to overcome several of the fundamental limitations of RNAi.

The silence and replace approach to the treatment of genetic disorders employs adeno-associated viral vectors (“AAVs”) to deliver genetic constructs which may, after a single administration to the target tissues:

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Chronically express RNAi molecules inside of the target, diseased, cells (to serially silence the intracellular production of mutant, disease-causing, protein and the functional protein of interest);

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Simultaneously drive the expression of a functional variant of the protein of interest (to restore native intracellular biological processes); and

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AAV vectors can accommodate the multi-functional DNA expression cassettes containing the engineered functional transgenes and the novel genes encoding short hairpinRNA/microRNA molecules (shRNA/miRNA) that are required to support the development of therapeutic agents capable of the achievement of the goals of the silence and replace approach to therapy.

Our silence and replace technology utilizes proprietary DNA expression cassettes to foster continuous production of gene silencing shRNAs and wild type proteins (via expression of the wild type transgene). A range of viral and non-viral gene therapy vectors can be used to deliver the DNA construct into the nucleus of the target cell and, upon delivery, shRNA molecules are expressed and subsequently processed by intracellular enzymes into siRNA molecules that silence the expression of the mutant, disease-causing protein.

In the silence and replace approach:

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A DNA construct is delivered to the nucleus of the target cell by a gene therapy vector such as an AAV vector;

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Once inside of the nucleus, the DNA construct drives the continuous production of shRNA molecules which are processed by an enzyme called Dicer into siRNAs;

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The processed siRNA is incorporated into RISC and silences the target gene; and

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When the DNA expression cassette is additionally comprised of a wild type transgene, upon entry of the DNA construct into the nucleus of the target cell via the use of the AAV vector, the DNA construct also drives the continuous production of wild type protein (to restore native intracellular biological processes).

Our strategy is to discover, develop and commercialize treatments that leverage the capabilities of ddRNAi and the silence and replace approach to disease management.

For selected product candidates, at the appropriate stage, we may collaborate with large biopharmaceutical companies to further co-develop and, if approved, commercialize our ddRNAi-based and silence and replace-based products to achieve broad clinical and commercial distribution. For specific clinical indications that we deem to be outside of our immediate areas of focus, we will continue to out-license, where appropriate, applications of our ddRNAi and silence and replace technology to facilitate the development of differentiated therapeutics, which could provide further validation of our proprietary technology and approach to disease management.

Our cash and cash equivalents will be deployed for the advancement of our product candidate BB-301 for the treatment of OPMD-derived dysphagia, including the natural history lead-in study and the Phase 1b/2a BB-301 treatment study, for the continued advancement of development activities for other existing and new product candidates, for general corporate purposes and for strategic growth opportunities.

Oculopharyngeal Muscular Dystrophy—OPMD

OPMD is an insidious, autosomal-dominant, late-onset degenerative muscle disorder that typically presents in patients at 40-to-50 years of age. The disease is characterized by progressive swallowing difficulties (dysphagia), eyelid drooping (ptosis) and limb weakness. OPMD is caused by a specific mutation in the poly(A)-binding protein nuclear 1, or PABPN1, gene. OPMD is a rare disease; however, patients have been diagnosed with OPMD in approximately 35 countries. Patient populations suffering from OPMD are well-identified, and significant geographical clustering has been noted for patients with this disorder, which could simplify clinical development and global commercialization efforts.

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Our Pipeline

The following table sets forth our current product candidate and the development status:

Table 1. Pipeline: Oculopharyngeal Muscular Dystrophy

The Investigational New Drug (IND) application for BB-301 was approved to proceed by the U.S. Food and Drug Administration in June 2023. The first study subject was safely treated in the BB-301 Phase 1b/2a clinical trial (NCT06185673) in November 2023. All Cohort 1 subjects have safely completed the 12-month post-BB-301-treatment follow-up period. The Company has currently treated three subjects in Cohort 2. BB-301 is the lead investigational gene therapy agent under development by Benitec, and the key attributes of BB-301 are outlined in Figure 1.

Figure 1

No therapeutic agents are approved for the treatment of OPMD. Additionally, there are no surgical interventions available to OPMD patients that modify the natural history of the disease, which is principally comprised of chronic deterioration of the swallowing function. BB-301 has received Orphan Drug Designation and Fast Track Designation in the United States and Orphan Drug Designation in the European Union and, upon achievement of regulatory approval for BB-301 in these respective jurisdictions, the

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Orphan Drug Designations would provide commercial exclusivity independent of intellectual property protection. While OPMD is a rare medical disorder, we believe the commercial opportunity for a safe and efficacious therapeutic agent in this clinical indication exceeds $1 billion over the course of the commercial life of the product.

BB-301 is our Lead, Silence and Replace-Based, OPMD Therapeutic Agent

BB-301 is composed of a modified AAV serotype 9 (AAV9) capsid that expresses a bifunctional construct under the control of a single muscle specific Spc5-12 promoter to achieve co-expression of both the codon-optimized PABPN1 mRNA and two shmiR molecules directed against wild type and mutant PABPN1. BB-301 is designed to correct the genetic defect underlying OPMD following a single localized administration.

BB-301—Design and Mechanism of Action

BB-301 is designed to target two distinct regions of the PABPN1 mRNA to accomplish gene silencing via the concomitant expression of two distinct shmiRs from a single DNA construct (Figure 2). BB-301 is also engineered to drive the simultaneous expression of a codon-optimized, siRNA-resistant, version of the wild type PABPN1 gene (Figure 2).

Figure 2

Summary of the Key Regulatory Interactions:

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In June 2023 the U.S. Food and Drug Administration ("FDA") cleared the Investigational New Drug (IND) application for BB-301 which allowed dosing of BB-301 to begin for OPMD subjects that are eligible for enrollment into the Phase 1b/2a treatment study (NCT06185673) described below.

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A Type C meeting was held with the FDA in the third quarter of 2026 (calendar year) to discuss the BB-301 pivotal study design.

Intellectual Property

Benitec seeks to actively procure rights to and protect the intellectual property and proprietary technology that it believes is important to its business. Such intellectual property rights include, but are not limited to, patents claiming our proprietary ddRNAi and silence and replace technologies, and specific product candidates employing those technologies, as well as know-how and trade secrets related to our product candidates and proprietary technology.

ddRNAi-based treatment for OPMD

Benitec’s patent portfolio for OPMD includes four active patent families relating to shRNA and shmiRs targeting PABPN1 (the causative gene for OPMD), "silence and replace" therapeutics and treatment strategies for OPMD, as well as interoperative delivery methods and delivery devices for use in such treatment strategies. These four families cover: (i) the individual shmiRs comprised within the OPMD therapeutic candidate, BB-301, under development at Benitec, (ii) the "silence and replace" construct within

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BB-301, (iii) treatment strategies for OPMD that silence PABPN1 which is causative for OPMD and replace with functional PABPN1, (iv) Benitec’s proprietary AAV vector for delivery of BB-301, (v) Benitec’s proprietary injection needle for delivery of BB-301 to the pharyngeal muscle of OPMD patients, (vi) pre-filled multi-injection devices used for delivery of BB-301, and (vii) an interoperative method to enable delivery of BB-301 to the pharyngeal muscle of OPMD patients. In this regard, BB-301 is a "silence and replace" construct encoding two shmiRs targeting the endogenous PABPN1 (including variants causative of OPMD) internally designated shmiR-13 and shmiR-17, as well as a codon-optimized PABPN1 replacement construct, the transcript of which is not targeted by shmiR-13 and shmiR-17. Both shmiRs and the codon-optimized PABPN1 replacement construct are under the control of a muscle-specific promoter and packaged within an AAV9 vector with a modified capsid protein. BB-301 is administered to the pharyngeal muscle via an interoperative method using a proprietary injection needle design for optimized delivery. Multi-injection delivery devices fitted with the proprietary injection needle and pre-filled with appropriate dosage volumes of BB-301 have been developed for delivery of BB-301 to the pharyngeal muscle of affected subjects via the intraoperative method.

The first patent family, entitled “Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof (OPMD family #1)”, arose out of a collaboration with Royal Holloway University of London (RHUL) and relates to three shRNA target regions within PABPN1. RHUL assigned its ownership interests in this patent family to Benitec, and the PCT application and the related U.S. priority document were filed solely in the name of Benitec. This patent family is directed to RNAi agents targeting specific regions within mutant PABPN1 variants causative of OPMD, as well as use of those RNAi agents in combination with PABPN1 replacement constructs to treat OPMD. More specifically, this family includes claims covering shmiR17 of BB-301. This patent family entered the national/regional phase in October/November 2018.

The second patent family, entitled “Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof (OPMD family #2)” relates to a second set of target regions within PABPN1, as well as the "silence and replace" construct BB-301 under development at Benitec. The PCT application and the related U.S. priority document were filed solely in the name of Benitec, and this family entered the national/regional phase in June/July 2019. This patent family is directed to RNAi agents targeting specific regions within mutant PABPN1 variants causative of OPMD, as well as "silence and replace" constructs and use of the same for treatment of OPMD. More specifically, this family includes claims covering shmiR13 and shmiR17 of BB-301 separately and in combination, as well as the full BB-301 "silence and replacement" construct. In certain jurisdictions, claims also cover shmiR16.

A third patent family, entitled “Methods for Treating Oculopharyngeal Muscular Dystrophy (OPMD) (OPMD family #3)” was filed by Benitec’s former licensee, Axovant Therapeutics, on Benitec’s behalf to pursue claims which are broadly directed to the ‘silence and replace’ treatment concept for OPMD, relying on RNAi agents to knockdown PABPN1 and replacement with functional PABPN1 which is not targeted by the RNAi agents. The claims in this application are not limited to the regions targeted by BB-301. The PCT application and the related U.S. priority document were filed solely in the name of Benitec, and this family entered the national/regional phase in April/May 2021. While a number of patent applications remain pending, OPMD family #3 has been passively abandoned in line with Benitec’s evolving IP strategy for the OPMD program. As such, OPMD family #3 is no longer considered an active patent family for the OPMD program.

A fourth patent family, entitled “Methods for Treating Oculopharyngeal Muscular Dystrophy (OPMD) (OPMD family #4)” has been filed to specifically claim the OPMD therapeutic candidate developed by Benitec, BB-301, encompassing the "silence and replace" construct (described herein) packaged with Benitec’s proprietary AAV9 vector having a modified phospholipase (PLA2) domain within its capsid (See “AAV with modified phospholiphase domain” below). his PCT application and the related U.S. priority document were filed solely in the name of Benitec, and this family entered the national/regional phase in August/September 2022.

A fifth patent family, entitled “Device and methods for administering a therapeutic composition to the pharyngeal muscle (OPMD family #5)” has been filed to capture (i) the interoperative delivery method developed by Benitec for administration of BB-301 to the pharyngeal muscle, (ii) the proprietary needle developed by Benitec for use in the interoperative delivery method, (iii) multi-injection devices fitted with Benitec’s proprietary needle and pre-filled with BB-301, and (iv) sets of the multi-injection devices suitable for treatment of a single patient. This patent family includes supporting preliminary clinical data generated for the first two patients participating in the Phase 2a clinical trial (the results of which are described herein). The PCT application, and both the PCT application and the related U.S. priority document were filed solely in the name of Benitec, and the family entered the national/regional phase in December 2025/January 2026.

AAV with modified phospholipase domain

The Benitec patent portfolio also includes a single patent family, entitled “Adeno-associated virus (AAV) with modified phospholipase domain,” which relates to an AAV having a modified phospholipase (PLA2) domain in the capsid. The modified AAV will be used as the delivery system for the OPMD therapeutic, BB-301. The PCT application and the related U.S. priority document were filed solely in the name of Benitec, and this family entered the national/regional phase in February/March 2021.

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Know-How

In addition to patent protection of ddRNAi and other technology and our product candidates, we also rely on proprietary know-how that is not patentable or that we elect not to patent, as valuable intellectual property for our business. This know-how is related to the areas of, among others, identifying nucleic acid targets for ddRNAi technology and designing ddRNAi constructs for targeting preferred genes. We have implemented a number of security measures designed to safeguard our know-how including limiting access to our research facilities, databases and networks. We also seek to protect our know-how by way of confidentiality agreements when engaging with external providers for progressing our pipeline of therapeutic candidates.

Laws and Regulations Regarding Patent Terms

The term of individual patents depends upon the legal terms of the patents in the countries in which they are obtained. In most countries in which we file, the patent term is 20 years from the earliest date of filing a non-provisional (or "complete") patent application. In the United States, a patent term may be shortened if a patent is terminally disclaimed over another patent owned by the same assignee. A patent’s term may also be lengthened by a patent term adjustment (PTA), which compensates a patentee for administrative delays by the USPTO in granting a patent. However, calculation of PTA also takes into account delays by the patentee during patent prosecution, which may partially or completely offset any additional term accorded to account for delays by the USPTO. The patent term of a European patent is 20 years from its complete filing date, which, unlike in the United States, is not subject to patent term adjustments due to delays by the European Patent Office (EPO) or patentee during prosecution.

The term of a patent that covers an FDA-approved drug substance may also be eligible for patent term extension (PTE) as compensation for the portion of the patent term that the patentee is able to commercially exploit the patent due to the lengthy FDA regulatory review process which is required for marketing of the drug substance. The Drug Price Competition and Patent Term Restoration Act of 1984, or the Hatch-Waxman Act, permits a PTE of up to five years beyond the expiration of the patent. The length of the PTE issued is related to the length of time the drug substance is under clinical testing and regulatory review during the term of the patent. However, PTE cannot extend the term of a patent beyond a total of 14 years from the date of marketing approval for the drug substance and only one patent applicable to an approved drug substance may be extended under PTE. Similar provisions are available in Europe and other jurisdictions to extend the term of a patent that covers an approved drug substance, although the eligibility requirements and criteria for calculating the duration of such extensions, vary. In the future, if and when our products receive FDA approval, and/or approval from an equivalent regulatory body in another jurisdiction in which patent protection is sought or obtained, we expect to apply for patent term extensions on patents covering those products.

Trademarks

Our trademarks include registrations for company branding and product names for our pipeline in development. The trademarks that we use in connection with our business include the following:

Country or Territory

Trademark (program)

Application or

Registration

number

Status

USA

BENITEC BIOPHARMA

4636053

Registered

USA

SILENCING GENES FOR LIFE

50015912

Pending

Australia

SILENCING GENES FOR LIFE BENITEC

1448041

Registered

Australia

BIOPHARMA

1448046

Registered

Australia

BENITEC-logo

1448052

Registered

International Bureau (WIPO) - designating EU; UK and US

GIVING DISEASE THE SILENT TREATMENT

1389399

Registered

USA

BENITEC

50016174

Pending

European Union

BENITEC

19399575

Accepted

Australia

BENITEC

1728797

Registered

Australia

BENITEC

1103049

Registered

Australia

GIVING DISEASE THE SILENT TREATMENT

1851660

Registered

United Kingdom

BENITEC

3238275

Registered

Manufacturing

The manufacture of the biological products required for gene therapy is complex and difficult. We do not currently own or operate manufacturing facilities for the production of preclinical, clinical or commercial quantities of any of our product candidates. We are exploring long-term manufacturing alliances with a number of potential partners to investigate manufacturing processes in order to

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produce materials at reasonable scale and cost of goods to support future commercialization efforts. We do not have a long-term agreement with any third-party manufacturer, but we plan to establish such a relationship with an appropriate manufacturer to serve our long-term needs.

Manufacturing is subject to extensive regulations that impose various procedural and documentation requirements, which govern record keeping, manufacturing processes and controls, personnel, quality control and quality assurance, among others. Our contract manufacturing organizations manufacture our product candidates under cGMP conditions. cGMP is a regulatory standard for the production of pharmaceuticals that will be used in humans.

Sales and Marketing

We have not yet established sales, marketing or product distribution operations because our product candidates are in preclinical or clinical development. If we receive marketing and commercialization approval for any of our product candidates, we intend to market the product through strategic alliances and distribution agreements with third parties. In certain cases, we may market an approved product directly worldwide or in selected geographical segments. The ultimate implementation of our strategy for realizing the financial value of our product candidates is dependent on the results of clinical trials for our product candidates, the availability of funds and the ability to negotiate acceptable commercial terms with third parties.

Competition

The biopharmaceutical industry is characterized by intense and dynamic competition to develop new technologies and proprietary therapies.

Any product candidates that we successfully develop and commercialize will have to compete with existing therapies and new therapies that may become available in the future. While we believe that our proprietary technology and scientific expertise in gene silencing using ddRNAi provide us with competitive advantages, we face potential competition from many different sources, including larger and better-funded pharmaceutical, specialty pharmaceutical and biotechnology companies, as well as from academic institutions and governmental agencies and public and private research institutions that may develop potentially competitive products or technologies. We are aware of several companies focused on developing gene therapy or gene silencing product candidates.

We are not aware of any companies developing a gene therapy or gene silencing approach for OPMD. Our product candidates, if approved, would also compete with treatments that have already been approved and accepted by the medical community, patients and third-party payers.

Many of our competitors and potential competitors, alone or with their strategic partners, have substantially greater financial, technical and human resources than we do and significantly greater experience in the discovery and development of product candidates, obtaining FDA and other regulatory approvals of treatments and the commercialization of those treatments. Mergers and acquisitions in the biotechnology and pharmaceutical industries may result in even more resources being concentrated among a smaller number of our competitors. These competitors also compete with us in recruiting and retaining qualified scientific and management personnel and establishing clinical study sites and subject registration for clinical studies, as well as in acquiring technologies complementary to, or necessary for, our programs. Smaller or early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and established companies.

We anticipate that we will face intense and increasing competition as new products enter the market and advanced technologies become available. We expect any treatments that we develop and commercialize to compete on the basis of, among other things, efficacy, safety, convenience of administration and delivery, price, the level of competition and the availability of reimbursement from government and other third party-payers.

Our commercial opportunity could be reduced or eliminated if our competitors develop and commercialize products that are safer, more effective, have fewer or less severe side effects, are more convenient or are less expensive than any products that we may develop. Our competitors also may obtain FDA or other regulatory approval for their products more rapidly than we may obtain approval for ours, which could result in our competitors establishing a strong market position before we are able to enter the market. In addition, we expect that our therapeutic products, if approved, will be priced at a significant premium over competitive products and our ability to compete may be affected in many cases by insurers or other third-party payers seeking to encourage the use of competitive products including biosimilar or generic products.

This increasingly competitive landscape may compromise the development of our product candidates.

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Government Regulation

As a clinical-based biotechnology company developing biological products, we and our product candidates are subject to extensive regulation by the U.S. Food and Drug Administration, or the FDA, and other federal, state, and local regulatory agencies. The Federal Food, Drug, and Cosmetic Act, or the FDC Act, the Public Health Service Act, or PHS Act, and their implementing regulations set forth, among other things, requirements for the research, testing, development, manufacture, quality control, safety, effectiveness, approval, labeling, storage, record keeping, reporting, distribution, import, export, advertising and promotion of biological products. A failure to comply with any applicable requirements during the product development, approval, or post-approval periods, may lead to administrative or judicial sanctions. These sanctions could include the imposition by the FDA or an IRB, of a suspension on clinical trials, refusal to approve pending marketing applications or supplements, withdrawal of approval, warning letters, product recalls, product seizures, total or partial suspension of production or distribution, injunctions, fines, civil penalties or criminal prosecution.

Although the discussion below focuses on regulation in the United States, we anticipate seeking approval for the testing and marketing of our products in other countries. Generally, our activities in other countries will be subject to regulations that are similar in nature and scope as those imposed in the United States, although there can be important differences. In the European Union, gene therapy products are classified as advanced therapy medicinal products, or ATMPs, and marketing authorization is available only through the centralized procedure administered by the European Medicines Agency, or EMA, under Regulation (EC) No 1394/2007 and Regulation (EC) No 726/2004.

Government regulation may delay or prevent testing or marketing of our product and candidates and impose costly procedures upon our activities.

Government Regulation in the United States

The FDA has provided guidance for the development of gene therapy products. Review of gene therapy products is conducted by the Office of Therapeutic Products, or OTP, within the Center for Biologics Evaluation and Research (CBER). OTP was established in 2023 as a "super office" succeeding the former Office of Tissues and Advanced Therapies. The FDA may also convene an advisory committee to obtain outside expert advice on applications for novel products, including gene therapy products. In addition, the FDA has issued a growing body of clinical guidelines, chemical, manufacturing and control, or CMC, guidelines, regenerative medicine guidelines and other guidelines, all of which are intended to facilitate industry’s development of gene therapy products.

Marketing Approval

In the United States, for premarket approval purposes, the FDA regulates gene therapy products as biologics under the FDC Act, the PHS Act and related regulations.

The steps required before a new biologic may be marketed in the United States generally include:

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nonclinical pharmacology and toxicology laboratory and animal tests according to good laboratory practices, or GLPs, and applicable requirements for the humane use of laboratory animals or other applicable regulations;

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submission of an investigational new drug, or IND, application which must become effective before human clinical trials may begin;

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adequate and well-controlled human clinical trials according to good clinical practices, or GCPs, and any additional requirements for the protection of human research subjects and their health information to establish the safety and efficacy of the investigational product for each targeted indication;

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submission of a biologics license application, or BLA, to the FDA;

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FDA’s pre-approval inspection of manufacturing facilities to assess compliance with current good manufacturing practices, or cGMPs, and, if applicable, the FDA’s good tissue practices, or GTPs, for the use of human cellular and tissue products to prevent the introduction, transmission, or spread of communicable diseases;

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FDA’s audit of clinical trial sites that generated data in support of the BLA; and

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FDA approval of a BLA, which must occur before a product can be marketed or sold.

Product Development Process

Before testing any biologic in humans, the product candidate enters the nonclinical, or preclinical, testing stage. Nonclinical tests include laboratory evaluations of product chemistry, toxicity, and formulation, as well as animal studies to assess the potential safety and activity of the product candidate. The conduct of nonclinical tests intended to support an IND must comply with GLPs.

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Research involving recombinant or synthetic nucleic acid molecules that is conducted at or sponsored by an institution receiving National Institutes of Health, or NIH, funding for such research is subject to the NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules, which require review and approval by the institution's institutional biosafety committee, or IBC. Effective April 2019, the NIH Guidelines were amended to eliminate the requirement that human gene transfer protocols be registered with, and reviewed by, the NIH Office of Science Policy, or OSP, and the former Recombinant DNA Advisory Committee. Institutional IBC review obligations continue to apply.

The Company submits the results of the nonclinical testing, together with manufacturing information, analytical data, any available clinical data or literature, and a proposed clinical protocol, to the FDA in an IND, which is a request for authorization from the FDA to administer an investigational product to humans. The IND automatically becomes effective 30 days after receipt by the FDA unless the FDA, within the 30-day time period, raises concerns or questions about the conduct of the clinical trial and places the clinical trial on a clinical hold. In such case, the IND sponsor must resolve any outstanding concerns with the FDA before the clinical trial may begin. An IRB for each site proposing to conduct the clinical trial must review and approve the plan for any clinical trial before it commences at that site, and the informed written consent of each participating subject is required in a form and content approved by the IRB. If the site has an IBC, it may also have to review and approve the proposed clinical trial. Each protocol involving testing on U.S. patients and subsequent protocol amendments must be submitted to the FDA as part of the IND.

The clinical investigation of an investigational product is generally divided into three phases, which may overlap or be combined:

Phase 1 trials are the initial introduction of the investigational product into humans and are principally directed to safety, tolerability and pharmacologic activity; Phase 2 trials evaluate effectiveness in patients with the disease under study and refine dosing; and Phase 3 trials are conducted in an expanded patient population to further evaluate effectiveness and safety and to provide an adequate basis for approval. To obtain a BLA approval, a sponsor must demonstrate that the product is safe, pure and potent, which includes a showing of substantial evidence of effectiveness. The FDA has stated that substantial evidence may be established by one adequate and well-controlled clinical investigation plus confirmatory evidence, and that flexibility in the quantity and type of evidence may be appropriate for products intended to treat rare diseases and for gene therapy products, for which large or replicate trials may not be feasible.

Annual progress reports detailing the results of the clinical trials must be submitted to the FDA. Written IND safety reports must be promptly submitted to the FDA and the investigators for serious and unexpected adverse events; any findings from other studies, tests in laboratory animals or in vitro testing that suggest a significant risk for human subjects; or any clinically important increase in the rate of a serious suspected adverse reaction over that listed in the protocol or investigator brochure. The sponsor must submit an IND safety report within 15 calendar days after the sponsor determines that the information qualifies for reporting. The sponsor also must notify the FDA of any unexpected fatal or life-threatening suspected adverse reaction within seven calendar days after the sponsor’s initial receipt of the information. The FDA recommends that sponsors of gene therapy products conduct long-term follow-up observation of trial subjects for delayed adverse events, with the recommended duration depending on the vector system and the persistence of the genetic material. The FDA has recommended long-term follow-up of up to 15 years for products with integrating vectors or genome-editing components, and a shorter observation period, generally up to five years, for products using vectors such as adeno-associated virus vectors that are not expected to integrate into the host genome.

The FDA may place a clinical hold and order the temporary, or permanent, discontinuation of a clinical trial at any time, or impose other sanctions, if it believes that the clinical trial either is not being conducted in accordance with FDA requirements or presents an unacceptable risk to the clinical trial patients. In some cases, clinical trials are overseen by an independent group of qualified experts organized by the trial sponsor, referred to as a data safety monitoring board, or DSMB, which reviews interim data and advises whether a trial may proceed. These decisions are based on the limited access to data from the ongoing trial. The suspension or termination of a clinical trial can occur during any phase of clinical trials if it is determined that the participants or patients are being exposed to an unacceptable health risk. In addition, there are requirements for the registration of ongoing clinical trials of drugs and biologics on public registries and the disclosure of certain information pertaining to the trials as well as clinical trial results after completion.

Assuming successful completion of all required testing in accordance with all applicable regulatory requirements, detailed investigational product information is submitted to the FDA in the form of a BLA for a biologic to request marketing approval for the product in specified indications.

Biologics License Application Approval Process

In order to obtain approval to market a biologic in the United States, a BLA must be submitted to the FDA that provides data from nonclinical studies and clinical trials and manufacturing information establishing to the FDA’s satisfaction the safety, purity, and potency or efficacy of the investigational product for the proposed indication. The BLA must be accompanied by a substantial user fee payment unless a waiver or exemption applies. The FDA has 60 days from receipt of a BLA to determine whether the application is sufficiently complete to be accepted for filing, and thereafter conducts a substantive review that includes an assessment of the manufacturing facilities for cGMP compliance.

The FDA may require a risk evaluation and mitigation strategy, or REMS, as a condition of approval if it determines that a REMS is necessary to ensure that the benefits of the product outweigh its risks.

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After the FDA completes its initial review of a BLA, it will either license the product or issue a complete response letter describing the deficiencies that must be addressed before the application can be approved.

Orphan Drug Designation

Under the Orphan Drug Act, the FDA may grant orphan designation to a drug or biological product candidate intended to treat a rare disease or condition, which is generally a disease or condition that affects fewer than 200,000 individuals in the United States, or more than 200,000 individuals in the United States and for which there is no reasonable expectation that the cost of developing and making a drug or biological product available in the United States for this type of disease or condition will be recovered from sales of the product candidate. Orphan product designation must be requested before submitting a BLA. After the FDA grants orphan product designation, the identity of the therapeutic agent and its potential orphan use are disclosed publicly by the FDA. Orphan product designation does not convey any advantage in or shorten the duration of the regulatory review and approval process. We have received orphan drug designation for BB-301 in the United States and the European Union.

If a product candidate that has orphan designation subsequently receives the first FDA approval for the disease or condition for which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any other applications to market the same drug or biological product for the same disease or condition for seven years, except in limited circumstances, such as a showing of clinical superiority to the product with orphan exclusivity. Competitors, however, may receive approval of different products for the indication for which the orphan product has exclusivity or obtain approval for the same product but for a different indication than the one for which the orphan product has exclusivity. Orphan drug exclusivity held by a third party could also block approval of one of our product candidates. Orphan drug status in the European Union has similar, but not identical, benefits.

Expedited Development and Review Programs

The FDA administers several programs intended to expedite the development or review of products that treat serious or life-threatening conditions and address unmet medical needs, including fast track designation, breakthrough therapy designation, priority review and accelerated approval. Fast track, breakthrough therapy and priority review may shorten development or review timelines but do not change the standards for approval; accelerated approval permits approval based on an effect on a surrogate or intermediate clinical endpoint reasonably likely to predict clinical benefit, subject to required confirmatory trials and expedited withdrawal procedures.

Section 3033 of the 21st Century Cures Act, enacted in December 2016, created the regenerative medicine advanced therapy, or RMAT, designation, codified at Section 506(g) of the FDC Act. A product is eligible for RMAT designation if it is a regenerative medicine therapy, including a gene therapy intended to durably modify the expression of a gene, that is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the product has the potential to address unmet medical needs for that condition. RMAT designation provides the sponsor with increased interaction with the FDA, including early interactions to discuss potential surrogate or intermediate endpoints, comparable to the interactions available for breakthrough therapy designated products, and makes the product eligible for priority review and accelerated approval if the applicable criteria are met.

We have received fast track designation for BB-301, but we have not obtained RMAT or breakthrough therapy designation for BB-301. We have not sought a rare pediatric disease priority review voucher for BB-301.

Expanded Access and Right to Try

Under Section 561 of the FDC Act and 21 C.F.R. Part 312, Subpart I, individual patients, intermediate-size patient populations and larger populations may seek access to investigational products outside of clinical trials. Section 3032 of the 21st Century Cures Act requires sponsors of investigational products for serious diseases to make publicly available their policy for evaluating and responding to expanded access requests. Separately, the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017 provides an alternative pathway under which eligible patients may obtain access to certain investigational products that have completed a Phase 1 trial, without FDA authorization or IRB review.

User Fee Programs

Under the Prescription Drug User Fee Act, as most recently reauthorized by the FDA User Fee Reauthorization Act of 2022, the FDA assesses annual program fees for approved products and application fees for BLAs, and establishes performance goals for the review of applications. Under the FDA’s current performance goals, the agency seeks to review and act on standard original BLAs within ten months, and priority original BLAs within six months, in each case from the 60-day filing date. The FDC Act provides for

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waivers of the application fee in specified circumstances, including for the first human drug application submitted by a qualifying small business that has no approved product.

FDA Additional Requirements and Post-Approval Requirements

The FDA may require, or companies may pursue, additional clinical trials after a product is approved. These so-called Phase 4 clinical trials may be made a condition to be satisfied for continuing drug and biologic approval.

Even if a product candidate receives regulatory approval, the approval may be limited to specific indications, patient populations and dosages, or might contain significant limitations on use in the form of warnings, precautions or contraindications, or a REMS, restrictions on distribution, or post-marketing study requirements.

Any products manufactured or distributed by us or on our behalf pursuant to FDA approvals would be subject to continuing regulation by the FDA, including requirements for record-keeping, reporting of adverse experiences with the biologic or drug, and submitting biological product deviation reports to notify the FDA of unanticipated changes in distributed products. Manufacturers are required to register their facilities with the FDA and certain state agencies and are subject to periodic announced or unannounced inspections by the FDA and certain state agencies for compliance with cGMP requirements. We rely, and expect to continue to rely, entirely on third parties for the manufacture of BB-301, and those third parties are subject to these requirements. The Drug Supply Chain Security Act, or DSCSA, enacted in November 2013 as Title II of the Drug Quality and Security Act, or DQSA, establishes requirements for tracing prescription drug products, including certain biological products, through the pharmaceutical distribution supply chain. The DSCSA’s enhanced electronic, interoperable product tracing requirements took effect for supply chain trading partners following a phased implementation period. These requirements would apply to us only if and when we have an approved product that is distributed in the United States. Noncompliance with cGMP or other requirements can result in issuance of warning or untitled letters, civil and criminal penalties, seizures, and injunctive action.

The FDA and other federal and state agencies closely regulate the labeling, marketing and promotion of drugs and biologics. The Department of Justice, the Office of Inspector General of the Department of Health and Human Services and state authorities enforce restrictions on the promotion of drugs and biologics, including through the federal civil False Claims Act and the federal Anti-Kickback Statute. While doctors are free to prescribe any product approved by the FDA for any use, a company can only make claims relating to safety and efficacy of a product that are consistent with FDA approval, and the company is allowed to market a product only for the particular use and treatment approved by the FDA. Failure to comply with these requirements can result in adverse publicity, warning or untitled letters, corrective advertising, injunctions, potential civil and criminal penalties, criminal prosecution, and agreements with governmental agencies that materially restrict the manner in which a company promotes or distributes products.

Manufacturing of AAV-Based Gene Therapy Products

Gene therapy products are subject to product-specific chemistry, manufacturing and controls requirements in addition to general cGMP requirements. The FDA’s January 2020 guidance “Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications” addresses vector characterization, lot release testing, and control of viral vector manufacturing processes, and 21 C.F.R. Part 610 requires that licensed biological products meet standards for potency, purity, identity and safety. The FDA has issued guidance addressing potency assurance for cellular and gene therapy products and manufacturing changes and comparability for human cellular and gene therapy products, which address the analytical and, where necessary, clinical evidence used to demonstrate that product manufactured after a process or site change is comparable to product used in earlier clinical studies. We depend entirely on third parties for the manufacture of BB-301, and those third parties are responsible for compliance with these requirements at the facilities they operate.

Patent Term Restoration and Marketing Exclusivity

Depending on the timing, duration and specifics of FDA marketing approval of our product candidates, some of our U.S. patents may be eligible for limited patent term extension under the Hatch-Waxman Amendments. The Hatch-Waxman Amendments permit a patent restoration term of up to five years as compensation for patent term lost during product development and the FDA regulatory review process. However, patent term restoration cannot extend the remaining term of a patent beyond a total of 14 years from the product’s FDA marketing approval date. Only one patent applicable to an approved biological is eligible for the extension and the application for the extension must be submitted prior to the expiration of the patent and within sixty days of the FDA issuing an approval of the biological product. The USPTO, in consultation with the FDA, reviews and approves the application for any patent term extension or restoration. A number of our patents issued in countries outside the U.S. (including but not limited to, the European Union, United Kingdom, Japan, Australia, Canada, China, South Korea, Israel and Singapore) may also be eligible for limited patent term extension under analogous provisions, although the availability, duration and scope of any such extension will depend on the applicable laws and regulatory approval pathway in the relevant jurisdiction.

The Biologics Price Competition and Innovation Act of 2009, which was included within the Patient Protection and Affordable Care Act, created an abbreviated approval pathway for biological products shown to be similar to, or interchangeable with, an

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FDA-licensed reference biological product, and grants a reference biologic twelve years of exclusivity from the time of first licensure, during the first four years of which an application under the abbreviated pathway may not be submitted.

Pediatric exclusivity provides an additional six months of exclusivity attached to any existing marketing exclusivity or listed patent protection, granted upon completion of a pediatric study in accordance with an FDA-issued written request. Requirements to conduct pediatric assessments under the Pediatric Research Equity Act do not apply to applications for products with orphan drug designation for the designated indication.

Government Regulation Outside the United States

In addition to regulations in the United States, we expect to be subject to a variety of regulations in other jurisdictions governing, among other things, clinical trials and any commercial sales and distribution of our products.

In the European Union, clinical trials are governed by Regulation (EU) No 536/2014, the Clinical Trials Regulation, or CTR, which became applicable on January 31, 2022 and replaced Directive 2001/20/EC. Under the CTR, a sponsor submits a single clinical trial application, or CTA, through the Clinical Trials Information System, or CTIS, identifying the member states in which the trial is to be conducted; the member states conduct a coordinated assessment of the scientific and general aspects of the application, while each member state separately assesses national and ethical aspects, including ethics committee review. Trials authorized under the prior Directive were required to transition to the CTR by January 31, 2025. Clinical trials in all cases are conducted in accordance with good clinical practices and the ethical principles that have their origin in the Declaration of Helsinki.

Because AAV-based gene therapy products contain or consist of genetically modified organisms, clinical trials with such products in the European Union are also subject to national implementations of the European Union’s deliberate release and contained use legislation, Directive 2001/18/EC and Directive 2009/41/EC, which impose environmental risk assessment and authorization requirements that are applied differently across member states.

Following the withdrawal of the United Kingdom from the European Union, the CTR and CTIS do not apply in Great Britain. Clinical trials in the United Kingdom require authorization from the Medicines and Healthcare products Regulatory Agency, or MHRA, and a favorable opinion from a research ethics committee, and marketing authorizations for Great Britain are granted by the MHRA rather than the European Commission. Separate arrangements apply in Northern Ireland under the Windsor Framework.

To obtain regulatory approval of a biological product under European Union regulatory systems, we must submit a marketing authorization application to the EMA under the centralized procedure, which is mandatory for ATMPs. Upon receiving marketing authorization, a new medicinal product generally receives eight years of data exclusivity and an additional two years of market exclusivity, extendable by one additional year if the marketing authorization holder obtains an authorization during the initial eight-year period for one or more new indications demonstrating significant clinical benefit over existing therapies.

Orphan drugs in the European Union are eligible for 10-year market exclusivity, which may be reduced to six years if at the end of the fifth year the product no longer meets the criteria for orphan designation. Market exclusivity does not prevent authorization of a similar product in limited circumstances specified in Regulation (EC) No 141/2000. We have received orphan designation for BB-301 in the European Union. In April 2023 the European Commission proposed a comprehensive revision of the European Union's general pharmaceutical legislation that would, among other things, modify the duration and structure of regulatory data protection, market protection and orphan market exclusivity. That proposal has not been adopted in final form, and the periods described above remain those provided under the currently applicable legislation.

Other Healthcare Laws

Our business activities, including our arrangements with investigators, clinical trial sites, consultants and third-party manufacturers, are subject to healthcare fraud and abuse laws. These include the federal Anti-Kickback Statute, 42 U.S.C. Section 1320a-7b(b), which prohibits knowingly and willfully soliciting, offering, receiving or providing remuneration to induce or reward referrals of, or the purchase, order or recommendation of, items or services reimbursable under a federal healthcare program; the federal civil False Claims Act, 31 U.S.C. Sections 3729-3733, which prohibits knowingly presenting or causing to be presented false or fraudulent claims for payment and permits qui tam actions by private persons; the federal civil monetary penalties laws; the federal criminal healthcare fraud statutes enacted as part of the Health Insurance Portability and Accountability Act of 1996, or HIPAA; and the federal Physician Payments Sunshine Act, 42 U.S.C. Section 1320a-7h, which requires applicable manufacturers of covered drugs, devices, biologicals and medical supplies to report annually payments and other transfers of value to covered recipients. Analogous state laws, including state anti-kickback, false claims, marketing disclosure and transparency laws, may also apply, and certain of these state laws apply regardless of payor. Because we have no approved products and no commercial operations, many of these requirements would apply to us principally upon and following any marketing approval and commercialization, although the Anti-Kickback Statute and False Claims Act may apply to research and pre-commercial arrangements.

Data Privacy and Security

We are subject to laws governing the privacy and security of personal information, including health information collected in clinical trials. In the United States, HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act, imposes

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privacy, security and breach notification obligations on covered entities and their business associates, and the Federal Policy for the Protection of Human Subjects and FDA informed consent regulations govern the use of subject information in research. Numerous states have enacted comprehensive consumer privacy laws, including the California Consumer Privacy Act as amended by the California Privacy Rights Act, and certain states have enacted laws directed specifically at consumer health data. In the European Union, the General Data Protection Regulation, Regulation (EU) 2016/679, and in the United Kingdom, the UK General Data Protection Regulation and the Data Protection Act 2018, impose requirements on the processing of personal data, including health data, and restrict transfers of personal data to countries outside those jurisdictions absent an adequacy decision or another lawful transfer mechanism such as standard contractual clauses. Failure to comply with these requirements can result in significant administrative fines and other enforcement action.

Pharmaceutical Coverage, Pricing and Reimbursement

Sales of our products, when and if approved for marketing, will depend, in part, on the extent to which our products will be covered by third-party payers, such as federal, state, and foreign government healthcare programs, commercial insurance and managed healthcare organizations. The U.S. government, state legislatures and foreign governments have continued implementing cost containment programs, including price controls, restrictions on coverage and reimbursement and requirements for substitution of interchangeable products. The Inflation Reduction Act of 2022 made significant changes to the Medicare program, including establishing a drug price negotiation program under which the Centers for Medicare & Medicaid Services negotiates maximum fair prices for selected high-expenditure drugs and biologicals, imposing rebates on manufacturers for price increases exceeding inflation, and redesigning the Medicare Part D benefit. The statute provides an exclusion from selection for negotiation for certain drugs designated as orphan drugs, subject to statutory conditions. Because we have no approved or marketed products, these provisions do not currently apply to us.

Import, Export and Biosafety Requirements

The import and export of biological materials and investigational products are subject to requirements under the FDC Act, including Sections 801 and 802, and to the requirements of the U.S. Customs and Border Protection. Importation of infectious biological agents and materials may require a permit issued by the Centers for Disease Control and Prevention under 42 C.F.R. Section 71.54, and the U.S. Department of Agriculture administers permitting requirements for certain biological materials. The transport of biological materials is subject to hazardous materials requirements administered by the U.S. Department of Transportation and to international dangerous goods requirements. Work with recombinant or synthetic nucleic acid molecules and viral vectors is subject to biosafety requirements, including institutional biosafety committee oversight under the NIH Guidelines at institutions subject to those Guidelines, and, in the European Union, to the genetically modified organism requirements described above.

Employees

As of June 30, 2026, we had 24 full-time employees, one of whom has an MD, PhD, eight have a PhD, four have a Master’s Degree, two have a biotechnology certificate, and one has an MBA, for a total of 13 with post-graduate degrees. Of our full-time employees, 17 are engaged in research and development activities and seven are engaged in finance, legal, human resources, facilities and general management. None of our employees are represented by any labor union. All employees are in the United States.

Corporate Information

We were incorporated as a Delaware corporation on November 22, 2019, and completed our re-domiciliation from Australia to the United States of America on April 15, 2020. Our predecessor, Benitec Limited, was incorporated under the laws of Australia in 1995. Our principal executive offices are located at 3940 Trust Way, Hayward, California 94545.

Our common stock began trading on The Nasdaq Capital Market, or Nasdaq, at the start of trading on April 15, 2020 under the symbol “BNTC.”

Available Information

Our telephone number is (510) 780-0819, and our Internet website is www.benitec.com. The information on, or that can be accessed through, our website is not part of this Annual Report on Form 10-K and is not incorporated by reference herein.

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