Benitec Biopharma
Benitec Biopharma is a clinical-stage biotechnology company developing BB-301, an AAV-based gene therapy using its proprietary ddRNAi 'Silence and Replace' platform to treat Oculopharyngeal Muscular Dystrophy (OPMD), a rare genetic muscle disorder with no approved therapies.
- Company typePublic
- Founded1995
- HeadquartersHayward, United States
- Headcount11–50
- GTM typeB2B
- OfferingHardware or Manufacturing
What Benitec Biopharma does
Benitec Biopharma Inc. is a clinical-stage biotechnology company developing genetic medicines based on its proprietary DNA-directed RNA interference (ddRNAi) platform. The 'Silence and Replace' approach combines RNA interference-mediated gene silencing with AAV-based gene therapy to simultaneously silence mutated disease-causing genes and replace them with wild-type genes following a single administration. The company's lead product candidate, BB-301, is an AAV-based gene therapy in Phase 1b/2a clinical development for Oculopharyngeal Muscular Dystrophy (OPMD), a rare genetic muscle disorder affecting approximately 15,000 patients across North America, Europe, and Israel that causes progressive dysphagia and muscle weakness with no currently approved therapies.
BB-301 has received FDA Orphan Drug Designation, FDA Fast Track Designation (November 2025), and EMA Orphan Drug Designation, and has demonstrated 100% response rate in Cohort 1 of its Phase 1b/2a trial with durable responses extending to 24 months and statistically significant improvements across multiple dysphagia measures. The company is headquartered in Hayward, California (re-domiciled from Australia to Delaware in April 2020), is listed on Nasdaq under ticker BNTC, and has 11-50 employees.
The company is pre-revenue with no approved products; historical revenue consisted solely of minimal licensing royalties (approximately $55-56K in FY2021 periods). Revenue model is anticipated to shift to product sales upon FDA approval of BB-301. The company has been funded through equity financings totaling approximately $169.4M since 2020, most recently a $100M public offering in November 2025, and holds $184.8M in cash as of March 2026. The go-to-market motion is enterprise/field-sales oriented for clinical-stage engagement with FDA, clinical trial sites, and academic conferences (MDA, ASGCT), with planned commercial distribution to be established upon regulatory approval.
Benitec Biopharma firmographics
Firmographics- Name
- Benitec Biopharma
- Legal name
- Benitec Biopharma Inc.
- Website
- https://benitec.com
- Company type
- Public
- Founded year
- 1995
- Operating status
- Operating
- Headcount range
- 11–50 employees
- Short description
- Benitec Biopharma is a clinical-stage biotechnology company developing BB-301, an AAV-based gene therapy using its proprietary ddRNAi 'Silence and Replace' platform to treat Oculopharyngeal Muscular Dystrophy (OPMD), a rare genetic muscle disorder with no approved therapies.
- Ownership category
- akta.pro rank
Benitec Biopharma industry classification
Industry- Product category
- Gene Therapy Biopharmaceuticals
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)
- SIC
- Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834)
- akta.pro primary industry
- Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)
- akta.pro secondary industries
- Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA), Neurology/CNS Gene Therapies (HLAAACAH)
Keywords
Where Benitec Biopharma is headquartered
LocationHeadquarters
- HQ city
- Hayward
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
Benitec Biopharma business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations, Marketing or Sales
Revenue model
- Gene Therapy Development: Pre-revenue clinical-stage biotechnology company. No products approved for commercial sale. Revenue historically from licensing fees and royalties from intellectual property licensing arrangements.
- Future Product Revenue: Anticipated revenue from commercialization of BB-301 upon FDA approval for OPMD treatment. Company maintains robust cash position ($184.8M as of March 2026) to fund development through pivotal study completion.
Go-to-market motion1 record
Distribution channels1 record
Marketing channels4 records
Benitec Biopharma product offering
Product offeringCore offering
Benitec Biopharma is a clinical-stage biotechnology company developing BB-301, an AAV-based gene therapy candidate for Oculopharyngeal Muscular Dystrophy (OPMD) using its proprietary DNA-directed RNA interference (ddRNAi) 'Silence and Replace' platform. The therapy combines gene silencing with wild-type gene replacement to address chronic and life-threatening genetic diseases with no approved treatments. The company has no products approved for commercial sale and currently derives revenue from licensing its intellectual property.
Product overview
Benitec Biopharma is a clinical-stage biotechnology company developing genetic medicines using its proprietary DNA-directed RNA interference (ddRNAi) 'Silence and Replace' platform. The company's lead product candidate is BB-301, an AAV-based gene therapy for Oculopharyngeal Muscular Dystrophy (OPMD) that combines gene silencing with wild-type gene replacement. BB-301 is currently in Phase 1b/2a clinical trials and has received Orphan Drug Designation from both FDA and EMA, plus FDA Fast Track Designation. The company operates as a single-segment biopharmaceutical development organization with no external API or integration offerings.
Differentiator
Problem solved
Functional benefit
Products and services
- BB-301 BB-301 is an AAV-based gene therapy candidate for Oculopharyngeal Muscular Dystrophy (OPMD) using a proprietary 'Silence and Replace' DNA-directed RNA interference (ddRNAi) platform. It silences mutated disease-causing genes and replaces them with wild-type genes to restore function in diseased cells. Currently in Phase 1b/2a clinical trials.
- ddRNAi Platform DNA-directed RNA interference (ddRNAi) technology platform combining RNA interference with gene therapy to create medicines enabling sustained silencing of disease-causing genes following a single administration. The platform supports the company's 'Silence and Replace' therapeutic approach.
Quantifiable outcome
- 100% response rate in Cohort 1 (6 patients) with statistically significant responses across multiple dysphagia measures
- +4 more outcomes
Companies that use Benitec Biopharma
Customer profileSegments1 record
Ideal customer profiles2 records
Benitec Biopharma technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
Benitec Biopharma partnerships and signals
Strategic signalScale indicators9 records
Recent moves7 records
Expansion highlights4 records
Benitec Biopharma competitors and assessment
Company assessmentBroad incumbents
- Regenxbio: Regenxbio is an established AAV gene therapy platform company whose NAV Technology underpins multiple approved and clinical-stage rare-disease gene therapies. Comparable as an AAV platform incumbent in the same modality space, though it operates at greater scale and across many more indications than Benitec.
- Alnylam Pharmaceuticals: Alnylam is the established commercial-stage leader in RNA interference (RNAi) therapeutics, with multiple approved siRNA-based drugs for rare genetic diseases. Highly relevant as the dominant incumbent in the underlying RNAi technology that Benitec's ddRNAi platform builds upon, although Alnylam uses siRNA rather than AAV delivery.
- Pfizer: Pfizer is a large pharmaceutical incumbent that markets Beqvez, an FDA-approved AAV gene therapy for adult hemophilia B, and has historical programs in DMD gene therapy. Comparable as a broad incumbent in the AAV gene therapy space targeting rare monogenic diseases, though operating at vastly greater scale than Benitec.
Direct peers
- Sarepta Therapeutics: Sarepta is the leading commercial-stage gene therapy company for Duchenne muscular dystrophy (DMD), with Elevidys approved for DMD and a deep pipeline of AAV-based treatments. It is the closest direct peer to Benitec given shared modality (AAV gene therapy), overlapping disease space (rare inherited muscular dystrophies), and shared regulatory pathway (Orphan Drug, accelerated approval).
- uniQure: uniQure is a clinical-to-commercial gene therapy company developing AAV-based therapies for rare diseases including hemophilia B and Huntington's disease. Comparable as a small-cap pure-play AAV gene therapy developer targeting rare monogenic diseases via a single-administration modality.
- Capricor Therapeutics: Capricor Therapeutics is a clinical-stage biotechnology company developing cell and gene therapy candidates for Duchenne muscular dystrophy. Directly comparable to Benitec as a small-cap rare-disease developer targeting muscular dystrophy with an advanced modality and similar regulatory positioning.
- Abeona Therapeutics: Abeona Therapeutics is a clinical-stage gene therapy company developing AAV-based treatments for rare diseases including recessive dystrophic epidermolysis bullosa. Directly comparable as a small-cap clinical-stage AAV gene therapy developer targeting a single rare monogenic disease with FDA Orphan Drug Designation.
- Solid Biosciences: Solid Biosciences is a clinical-stage gene therapy company developing AAV-based treatments for Duchenne muscular dystrophy and other rare neuromuscular diseases. Directly comparable to Benitec in target indication family (rare muscular dystrophy), modality (AAV gene therapy), and clinical-stage development profile.
- Krystal Biotech: Krystal Biotech is a commercial-stage gene therapy company with Vyjuvek approved for the rare skin disease dystrophic epidermolysis bullosa. Directly comparable to Benitec as a small-cap AAV-delivered gene therapy developer focused on a single rare monogenic disease, with a similar regulatory and commercial pathway.
Emerging players
- Voyager Therapeutics: Voyager Therapeutics is a clinical-stage gene therapy company developing AAV-based treatments for CNS and rare neurological diseases using a proprietary capsid platform. Comparable as an emerging AAV gene therapy developer focused on rare monogenic indications, though its CNS focus differs from Benitec's muscular indication.
Market position
Strengths4 records
Weaknesses5 records
Competitive moat3 records
Key risks6 records
Key highlights6 records
Customer concentration
Benitec Biopharma social profiles
Digital presenceBenitec Biopharma compliance and trust
Trust signalCompliance3 records
Benitec Biopharma financial estimates
Financial estimateRevenue estimate
Valuation estimate
Benitec Biopharma leadership team
Management profileNumber of profiles
Profiles7 records
Benitec Biopharma subsidiaries and ownership
Company hierarchySubsidiaries7 records
Benitec Biopharma funding detail
Funding detailFunding overview
Funding rounds9 records
Investors5 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Benitec Biopharma M&A and investment
M&A and investmentM&A1 record
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Benitec Biopharma
What does Benitec Biopharma do?
Benitec Biopharma is a clinical-stage biotechnology company developing BB-301, an AAV-based gene therapy candidate for Oculopharyngeal Muscular Dystrophy (OPMD) using its proprietary DNA-directed RNA interference (ddRNAi) 'Silence and Replace' platform. The therapy combines gene silencing with wild-type gene replacement to address chronic and life-threatening genetic diseases with no approved treatments. The company has no products approved for commercial sale and currently derives revenue from licensing its intellectual property.
Is Benitec Biopharma a public or private company?
Benitec Biopharma is a public company. It is classified as public and is currently operating.
When was Benitec Biopharma founded?
Benitec Biopharma was founded in 1995. It employs 11 to 50 people.
Where is Benitec Biopharma based?
Benitec Biopharma is headquartered in Hayward, United States, in the North America region.
How does Benitec Biopharma make money?
Two revenue lines are on record. Gene Therapy Development is the primary driver. The others are future Product Revenue.
Who are Benitec Biopharma's main competitors?
Broad incumbents on record are Regenxbio, Alnylam Pharmaceuticals and Pfizer. Direct peers are Sarepta Therapeutics, uniQure, Capricor Therapeutics, Abeona Therapeutics, Solid Biosciences and Krystal Biotech. Voyager Therapeutics is listed as an emerging player.
Does Benitec Biopharma have an API?
No public API is recorded for Benitec Biopharma.
What industry is Benitec Biopharma in?
Benitec Biopharma's product category is Gene Therapy Biopharmaceuticals. Its primary akta.pro industry code is HLAAACAE, Gene Therapy for Rare/Monogenic Diseases, with a secondary code of HLAIAIAA, Genetic & Genomic Rare Disease Therapeutics. Its NAICS code is 541714 and its SIC code is 2836.