Capsida Los Angeles
Capsida Biotherapeutics is a clinical-stage gene therapy company engineering AAV capsids for targeted delivery to the central nervous system and eye. It develops treatments for neurological and ocular diseases via internal programs and strategic partnerships with major pharmaceutical companies.
- Company typePrivate
- Founded2019
- HeadquartersNewbury Park, United States
- Headcount101–250
- GTM typeB2B
- OfferingHardware or Manufacturing
What Capsida Los Angeles does
Capsida Biotherapeutics, Inc. is a clinical-stage, private biotechnology company developing next-generation adeno-associated virus (AAV) gene therapies for rare and common neurological and ocular diseases. Founded in 2019 in Thousand Oaks, California and based on capsid engineering technology originating from Professor Viviana Gradinaru's laboratory at Caltech, the company operates a high-throughput AAV engineering platform that screens large engineered capsid libraries in non-human primates and human cell lines to identify capsids optimized for central nervous system (CNS) and ocular delivery. Preclinical data show >100-fold improvement over AAV9 in CNS expression, >70% neuronal transduction brain-wide, and identification of novel human blood-brain-barrier receptors (ADAM15, RECK) that are conserved between primates and humans, enabling non-invasive intravenous dosing for CNS indications. The pipeline comprises CAP-001 (MPS II/Hunter Syndrome), CAP-002 (STXBP1-DEE), CAP-003 (PD-GBA), CAP-004 (Friedreich's ataxia), and a set of ophthalmology programs using intravitreal and suprachoroidal delivery. The CAP-002 SYNRGY trial was paused and subsequently closed after a patient death in September 2025, and the CAP-003 PD-GBA trial was closed in 2026; the company is repositioning around its ocular franchise and next-generation capsids.
Capsida is vertically integrated, running all process development, analytical activities, and cGMP manufacturing in a 15,000 sq ft Thousand Oaks facility using an HEK293 two-plasmid platform scalable from 2L to 200L, eliminating dependence on contract development and manufacturing organizations. The business model is a hybrid of strategic pharmaceutical partnerships and contract manufacturing: the company generates revenue from upfront payments, equity investments, milestone payments, and royalties under collaborations with AbbVie (CNS and ophthalmology, originally $80M upfront + $10M equity + $530M/program milestones + high single-digit royalties), CRISPR Therapeutics (ALS and Friedreich's ataxia), and Eli Lilly's Prevail division ($55M upfront + up to $685M in milestones + tiered royalties), supplemented by contract manufacturing revenue from Kate Therapeutics. The platform can deliver gene replacement, gene editing, gene silencing, and vectorized antibody cargos.
The company is financed by Versant Ventures and Westlake Village BioPartners, both of which co-led the April 2021 $50M Series A alongside a $90M AbbVie strategic collaboration, and has an issued U.S. patent (11,149,256) covering brain-wide transducing capsids with reduced liver targeting plus 30-40+ pending patent applications. Leadership includes Interim CEO Mina Kim (former CEO and CLO of Acelyrin), CSO Romuald Corbau (ex-CSO of Adverum Biotechnologies, acquired by Eli Lilly), CFO Amar Duvvur (former SVP Finance at Acelyrin, VP at AbbVie), co-founder Nicholas Flytzanis (Chief Research and Innovation Officer), and Chief Manufacturing and Quality Officer Rob Murphy, with board representation from Versant (Clare Ozawa), Westlake (Beth Seidenberg), and independent directors Rita Balice-Gordon and Frank Verwiel. Headcount is reported at 50-100 employees.
Capsida Los Angeles firmographics
Firmographics- Name
- Capsida Los Angeles
- Legal name
- Capsida Biotherapeutics, Inc.
- Website
- https://capsida.com
- Company type
- Private
- Founded year
- 2019
- Operating status
- Operating
- Headcount range
- 101–250 employees
- Short description
- Capsida Biotherapeutics is a clinical-stage gene therapy company engineering AAV capsids for targeted delivery to the central nervous system and eye. It develops treatments for neurological and ocular diseases via internal programs and strategic partnerships with major pharmaceutical companies.
- Ownership category
- akta.pro rank
Capsida Los Angeles industry classification
Industry- Product category
- Biotechnology - Gene Therapy
- NAICS
- Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in Biotechnology (except Nanobiotechnology) (541714)
- SIC
- Biological Products, (No Disgnostic Substances) (2836)
- akta.pro primary industry
- Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream) (HLAAACAN)
- akta.pro secondary industries
- AAV Vector Gene Therapy Developers (HLAAACAA), Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Gene Therapy Delivery Devices & Administration Systems (catheters, intrathecal, ocular delivery) (HLAAACAL)
Keywords
Where Capsida Los Angeles is headquartered
LocationHeadquarters
- HQ city
- Newbury Park
- HQ country
- United States
- HQ region
- North America
Offices2 records
Markets served
Capsida Los Angeles business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Infrastructure, Supply Chain, Operations
Revenue model
- Strategic Partnerships and Licensing: Capsida receives upfront payments, equity investments, and milestone payments from pharmaceutical partners (AbbVie, CRISPR Therapeutics, Lilly/Prevail) for developing gene therapies using its AAV platform
- Collaboration Revenue: Revenue from strategic collaborations including option fees, development milestones, and commercial royalties. AbbVie collaboration includes $530M per program in option and development milestones plus high single-digit royalties. Prevail collaboration includes up to $685M in R&D and commercial milestones plus tiered royalties
- Internal Pipeline Development: Development of proprietary gene therapy programs (CAP-002, CAP-003, CAP-004) for rare and common neurological diseases targeting future commercial revenue
- Contract Manufacturing: Manufacturing services for external partners including Kate Therapeutics for next-generation gene therapies
Go-to-market motion2 records
Distribution channels4 records
Marketing channels6 records
Capsida Los Angeles product offering
Product offeringCore offering
Capsida Biotherapeutics is a fully integrated genetic medicine company developing engineered adeno-associated virus (AAV) capsids and gene therapies targeting rare and common neurological and ocular diseases. Its core offering is a high-throughput capsid engineering platform that screens billions of engineered capsid variants in non-human primates to identify capsids capable of crossing the blood-brain barrier with intravenous delivery, combined with an internal pipeline of gene therapy programs (CAP-001, CAP-002, CAP-003, CAP-004) and proprietary cGMP manufacturing capabilities.
Product overview
Capsida Biotherapeutics is a fully integrated next-generation genetic medicine company developing a pipeline of gene therapies for rare and common neurologic and ocular diseases. The company's core offering consists of its proprietary AAV Capsid Engineering Platform, which enables the development of engineered capsids for targeted gene delivery. The integrated pipeline includes: CAP-001 (MPS II), CAP-002 (STXBP1-DEE), CAP-003 (PD-GBA), and CAP-004 (Friedreich's ataxia), alongside ophthalmology programs utilizing IVT and SCS delivery routes. The company operates state-of-the-art cGMP manufacturing facilities for end-to-end production control.
Differentiator
Problem solved
Functional benefit
Products and services
- CAP-001 Engineered AAV gene therapy for MPS II (Hunter Syndrome), a lysosomal storage disorder, delivering IDS transgene using novel capsid technology.
- CAP-002 IV-delivered AAV gene therapy for STXBP1 Developmental and Epileptic Encephalopathy (STXBP1-DEE), designed to stably replace STXBP1 protein throughout the brain after single intravenous infusion.
- CAP-003 IV-delivered AAV gene therapy for Parkinson's disease associated with GBA mutations (PD-GBA), designed to increase GCase activity in the brain.
- CAP-004 Gene therapy for Friedreich's ataxia targeting CNS and cardiac symptoms, delivered as single intravenous infusion addressing underlying frataxin deficiency.
- Capsid Engineering Platform High-throughput AAV engineering platform using automated screening of large diverse engineered capsid libraries in non-human primates to identify optimal capsids for CNS and ocular targets.
- cGMP Manufacturing Services State-of-the-art cGMP manufacturing operations using HEK293 platform with two-plasmid transfection system, scalable from 2L to 200L, supporting IND packages and contract manufacturing for external partners.
Quantifiable outcome
- >100-fold improvement over AAV9 in CNS expression in NHPs
- +3 more outcomes
Companies that use Capsida Los Angeles
Customer profileNamed customers4 records
Segments6 records
Ideal customer profiles2 records
Capsida Los Angeles technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature8 records
Capsida Los Angeles partnerships and signals
Strategic signalPartnerships
Four partnerships are on record, tiered minor and core.
- Kate TherapeuticsminorStrategic collaboration for Capsida to manufacture KateTx's next-generation gene therapies, leveraging state-of-the-art cGMP manufacturing capabilities
- Prevail Therapeutics (Eli Lilly)coreMulti-year collaboration to develop IV-administered gene therapies for CNS diseases using Capsida's AAV platform. Capsida receives $55M upfront plus potential up to $685M in milestones and royalties. Capsida leads capsid discovery; Prevail handles preclinical and IND-enabling studies.
- CRISPR TherapeuticscoreStrategic partnership to research, develop, manufacture and commercialize in vivo gene editing therapies for familial ALS and Friedreich's ataxia. CRISPR leads Friedreich's ataxia program and gene-editing; Capsida leads ALS program and capsid engineering. Each company has option to co-develop and co-commercialize the program the other leads.
- AbbViecoreMulti-year strategic collaboration to develop best-in-class, targeted gene therapies for three CNS disease targets. Capsida receives $80M upfront + $10M equity, eligible for $530M per program in milestones plus high single-digit royalties. AbbVie leads late-stage development and commercialization. Collaboration expanded in 2023 to include three ophthalmology programs.
Scale indicators10 records
Recent moves7 records
Expansion highlights6 records
Capsida Los Angeles competitors and assessment
Company assessmentBroad incumbents
- BioMarin Pharmaceutical: BioMarin is a commercial-stage rare disease biotech with the first FDA-approved AAV gene therapy (ROCTAVIAN for hemophilia A). It is a broad incumbent in gene therapy commercialization with CNS and rare disease pipeline overlap with Capsida.
- Spark Therapeutics (Roche): Spark, a Roche subsidiary, commercialized LUXTURNA (first FDA-approved AAV gene therapy) and develops gene therapies for CNS, ophthalmic, and hematologic indications. It is a broad incumbent in the AAV gene therapy space overlapping Capsida's ocular and rare disease focus.
- Sarepta Therapeutics: Sarepta is the leading commercial gene therapy company with multiple approved AAV micro-dystrophin products for Duchenne muscular dystrophy. It represents the established commercial benchmark and potential acquirer/partner for capsid-engineered CNS assets.
- Pfizer (Gene Therapy Unit): Pfizer is a broad biopharmaceutical incumbent with AAV gene therapy assets (e.g., beqvez for hemophilia B) and Capsida's new CSO Romuald Corbau initiated Pfizer's first gene therapy program. Functions as both a potential partner/acquirer and a competitor in CNS AAV delivery.
Direct peers
- Taysha Gene Therapies: Taysha develops AAV gene therapies for CNS diseases including Rett syndrome and GAN, directly overlapping with Capsida's CNS pipeline. Similar clinical-stage profile and reliance on partnership economics to advance pipeline.
- Adverum Biotechnologies: Adverum (acquired by Eli Lilly in 2024) developed intravitreal AAV gene therapies for ocular diseases, directly competing with Capsida's ocular capsid programs. Capsida's new CSO Romuald Corbau was previously CSO at Adverum, underscoring the strategic linkage.
- uniQure: uniQure is an AAV-based gene therapy company with commercial product HEMGENIX (hemophilia B) and pipeline assets in Huntington's disease (similar CNS focus to Capsida) and other indications. Direct comparable on modality and CNS target space.
- Voyager Therapeutics: Voyager is a leading AAV capsid engineering company with a TRACER-derived capsid platform for CNS and other indications. It is the most direct competitor to Capsida's engineered-AAV-for-CNS thesis and has similar partnership economics with Pfizer, Novartis, and others.
- REGENXBIO: REGENXBIO develops AAV gene therapies using its NAV Technology Platform across rare and chronic diseases including CNS, retinal, and metabolic indications. It is a direct peer on the AAV platform and delivery side, with commercial-stage product ABECMA-related royalty stream and clinical pipeline overlap with Capsida.
Emerging players
- Krystal Biotech: Krystal Biotech is a commercial-stage gene therapy company (VYJUVEK for DEB) with a delivery platform applicable to rare diseases. Smaller and more focused than Capsida, it provides a comp on rare-disease gene therapy commercial economics.
Market position
Strengths4 records
Weaknesses4 records
Competitive moat6 records
Key risks5 records
Key highlights6 records
Customer concentration
Capsida Los Angeles social profiles
Digital presenceCapsida Los Angeles compliance and trust
Trust signalCompliance4 records
Capsida Los Angeles financial estimates
Financial estimateRevenue estimate
Valuation estimate
Capsida Los Angeles leadership team
Management profileNumber of profiles
Profiles9 records
Capsida Los Angeles funding detail
Funding detailFunding overview
Funding rounds3 records
Investors3 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Capsida Los Angeles M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Capsida Los Angeles
What does Capsida Los Angeles do?
Capsida Biotherapeutics is a fully integrated genetic medicine company developing engineered adeno-associated virus (AAV) capsids and gene therapies targeting rare and common neurological and ocular diseases. Its core offering is a high-throughput capsid engineering platform that screens billions of engineered capsid variants in non-human primates to identify capsids capable of crossing the blood-brain barrier with intravenous delivery, combined with an internal pipeline of gene therapy programs (CAP-001, CAP-002, CAP-003, CAP-004) and proprietary cGMP manufacturing capabilities.
Is Capsida Los Angeles a public or private company?
Capsida Los Angeles is a private company. It is classified as venture growth investor backed and is currently operating.
When was Capsida Los Angeles founded?
Capsida Los Angeles was founded in 2019. It employs 101 to 250 people.
Where is Capsida Los Angeles based?
Capsida Los Angeles is headquartered in Newbury Park, United States, in the North America region.
How does Capsida Los Angeles make money?
Four revenue lines are on record. Strategic Partnerships and Licensing is the primary driver. The others are collaboration Revenue, internal Pipeline Development and contract Manufacturing.
Who are Capsida Los Angeles's main competitors?
Broad incumbents on record are BioMarin Pharmaceutical, Spark Therapeutics (Roche), Sarepta Therapeutics and Pfizer (Gene Therapy Unit). Direct peers are Taysha Gene Therapies, Adverum Biotechnologies, uniQure, Voyager Therapeutics and REGENXBIO. Krystal Biotech is listed as an emerging player.
Does Capsida Los Angeles have an API?
No public API is recorded for Capsida Los Angeles.
What industry is Capsida Los Angeles in?
Capsida Los Angeles's product category is Biotechnology - Gene Therapy. Its primary akta.pro industry code is HLAAACAN, Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream), with a secondary code of HLAAACAA, AAV Vector Gene Therapy Developers. Its NAICS code is 325414 and its SIC code is 2836.