Jaguar Gene Therapy
Jaguar Gene Therapy is a clinical-stage, pre-revenue biotechnology company developing AAV9-based gene therapies — lead candidate JAG201 for SHANK3 haploinsufficiency/Phelan-McDermid syndrome (in first-in-human dosing), plus JAG101 (galactosemia) and JAG301 (Type 1 diabetes) — for patients with severe genetic diseases lacking approved treatments.
- Company typePrivate
- Founded2020
- HeadquartersLake Forest, United States
- Headcount11–50
- GTM typeB2B
- OfferingHardware or Manufacturing
What Jaguar Gene Therapy does
Jaguar Gene Therapy is a clinical-stage biotechnology company developing AAV9-based gene therapies for severe genetic diseases with significant unmet medical need. Founded in 2020 by former AveXis leadership and backed by Deerfield Management, the company is headquartered in Lake Forest, Illinois, with process sciences and manufacturing operations in Cary and Durham, North Carolina. Its core product candidates are JAG201, an AAV9 vector delivering a functional SHANK3 minigene via intracerebroventricular (ICV) injection for SHANK3 haploinsufficiency / Phelan-McDermid syndrome (lead clinical-stage program, currently in first-in-human dosing); JAG101, delivering the GALT gene for Type 1 galactosemia (preclinical); and JAG301, using PAX4-mediated transdifferentiation to convert alpha cells into beta cells for Type 1 diabetes (preclinical). A fourth program, AXV101 for BBS1-related Bardet-Biedl syndrome, is being developed by majority-owned subsidiary Axovia Therapeutics.
The company leverages a proven AAV9 delivery platform, exclusive licenses to foundational IP from the Broad Institute (JAG201) and Tulane University (JAG301), and vertically integrated CMC and manufacturing capabilities through a $125 million, 174,000-square-foot commercial facility in Durham, NC, owned by Alexandria Real Estate. JAG201 has received Rare Pediatric Disease, Fast Track, and Orphan Drug designations from the FDA, and Cohort 1 dosing was completed in February 2026 with no treatment-related serious adverse events reported.
Jaguar is pre-revenue and monetizes through eventual commercialization of one-time gene therapy treatments, supplemented by patient identification partnerships (e.g., CureSHANK, Phelan-McDermid Syndrome Foundation, Galactosemia Foundation, NORD) and a diagnostic collaboration with GeneDx. Its go-to-market is anchored in deep engagement with rare-disease patient communities and KOLs at leading academic centers such as Mount Sinai, Rush University, and Boston Children's Hospital, while investor syndicate includes Eli Lilly, Deerfield Management, ARCH Venture Partners, Goldman Sachs, Nolan Capital, Samsung Ventures, and the JDRF T1D Fund.
Jaguar Gene Therapy firmographics
Firmographics- Name
- Jaguar Gene Therapy
- Legal name
- Jaguar Gene Therapy, LLC
- Website
- https://jaguargenetherapy.com
- Company type
- Private
- Founded year
- 2020
- Operating status
- Operating
- Headcount range
- 11–50 employees
- Short description
- Jaguar Gene Therapy is a clinical-stage, pre-revenue biotechnology company developing AAV9-based gene therapies — lead candidate JAG201 for SHANK3 haploinsufficiency/Phelan-McDermid syndrome (in first-in-human dosing), plus JAG101 (galactosemia) and JAG301 (Type 1 diabetes) — for patients with severe genetic diseases lacking approved treatments.
- Ownership category
- akta.pro rank
Jaguar Gene Therapy industry classification
Industry- Product category
- Gene Therapy / Rare Disease Biologics
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714)
- SIC
- Biological Products, (No Disgnostic Substances) (2836)
- akta.pro primary industry
- AAV Vector Gene Therapy Developers (HLAAACAA)
- akta.pro secondary industries
- Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Neurology/CNS Gene Therapies (HLAAACAH), Cardiometabolic & Other Systemic Gene Therapies (HLAAACAI), Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream) (HLAAACAN)
Keywords
Where Jaguar Gene Therapy is headquartered
LocationHeadquarters
- HQ city
- Lake Forest
- HQ country
- United States
- HQ region
- North America
Offices3 records
Markets served
Jaguar Gene Therapy business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Infrastructure, Supply Chain, Marketing or Sales, Operations
Revenue model
- Gene Therapy Development and Commercialization: As a clinical-stage biotechnology company, Jaguar Gene Therapy is pre-revenue. The company is developing investigational gene therapies (JAG101, JAG201, JAG301) targeting severe genetic diseases. Revenue will be generated through commercialization of approved gene therapy products once clinical trials are completed and regulatory approvals are obtained.
Go-to-market motion2 records
Distribution channels1 record
Marketing channels5 records
Jaguar Gene Therapy product offering
Product offeringCore offering
Jaguar Gene Therapy develops investigational AAV9-based gene therapies targeting the root causes of severe genetic diseases with no FDA-approved treatments. Its lead candidate JAG201 delivers a functional SHANK3 gene to treat Phelan-McDermid syndrome/genetic autism, while preclinical programs JAG101 (GALT for Type 1 galactosemia) and JAG301 (PAX4 for Type 1 diabetes) extend the pipeline. All programs leverage the company's internal CMC capabilities and commercial-scale GMP manufacturing facility in Durham, NC.
Product overview
Jaguar Gene Therapy is advancing a pipeline of AAV9-based gene therapies targeting severe genetic diseases with significant unmet medical need. The core portfolio consists of three investigational programs: JAG201 (lead clinical-stage candidate for SHANK3 haploinsufficiency/Phelan-McDermid syndrome/genetic autism), JAG101 (preclinical for Type 1 galactosemia delivering functional GALT gene), and JAG301 (preclinical for Type 1 diabetes using PAX4-mediated transdifferentiation). Additionally, AXV101 for Bardet-Biedl syndrome is being developed by majority-owned subsidiary Axovia Therapeutics. The company leverages an AAV gene therapy platform with internal CMC capabilities and a 174,000 sq ft commercial manufacturing facility in Durham, NC.
Differentiator
Problem solved
Functional benefit
Products and services
- JAG201 Investigational AAV9-based gene therapy delivering a functional SHANK3 minigene via intracerebroventricular (ICV) injection to treat SHANK3 haploinsufficiency, a monogenic cause of autism spectrum disorder clinically diagnosed as Phelan-McDermid syndrome. Designed to restore synaptic function required for learning and memory. Currently in Phase I clinical trials for patients with genetic ASD and Phelan-McDermid syndrome.
- JAG101 Investigational AAV9-based gene therapy delivering a functional GALT gene to address the root cause of Type 1 galactosemia by reducing toxic metabolites (galactose, Gal-1P, galactitol). Currently in preclinical development with encouraging preclinical data showing increased GALT enzyme expression approaching or surpassing wild-type levels in liver and skeletal muscle.
- JAG301 Investigational AAV-based gene therapy using the PAX4 gene to convert alpha cells to beta cells (transdifferentiation) for the treatment of Type 1 diabetes. Currently in preclinical development.
- AXV101 Investigational gene therapy treatment for BBS1, a subset of Bardet-Biedl syndrome (BBS). Developed by Axovia Therapeutics, a majority-owned subsidiary of Jaguar Gene Therapy focused on ciliopathies.
Quantifiable outcome
- JAG201 first-in-human trial: Cohort 1 dosing complete with first 3 patients receiving starting dose via ICV injection; first 2 patients in Cohort 2 already dosed. No treatment-related serious adverse events (SAEs) or dose-limiting toxicities (DLTs) reported to date. Early indications of clinical benefit observed across neurodevelopmental domains including communicative, motor, cognitive and social.
- +2 more outcomes
Companies that use Jaguar Gene Therapy
Customer profileNamed customers4 records
Segments1 record
Ideal customer profiles3 records
Jaguar Gene Therapy technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature4 records
Jaguar Gene Therapy partnerships and signals
Strategic signalPartnerships
Eight partnerships are on record, tiered core and flagship.
- Andelyn BiosciencescorePartnership for late-stage Process Performance Qualification (PPQ) manufacturing of JAG201 gene therapy. Andelyn provides AAV vector manufacturing and GMP production expertise to support JAG201's clinical advancement.
- GeneDxcoreLaunched Autism Partnership Program in New York collaborating with Jaguar to offer free exome and genome testing to eligible individuals. Program aims to reduce barriers to genetic testing and facilitate earlier diagnosis and targeted treatment development.
- Alexandria Real Estate Equities, Inc.coreNation's leading provider of life science real estate. Owns and operates the 174,000-square-foot commercial manufacturing facility in Durham, NC for Jaguar.
- Broad Institute of MIT and HarvardflagshipExclusive license for JAG201 program targeting SHANK3 haploinsufficiency. Program based on groundbreaking work of Guoping Feng, Ph.D. from Broad Institute. Preclinical proof-of-concept data generated at Broad.
- Tulane UniversityflagshipExclusive license for JAG301 program for Type 1 diabetes using PAX4 gene for transdifferentiation. Research agreement in place. Encouraging preclinical proof-of-concept data produced at Tulane.
- Emory UniversitycoreResearch agreement in place for JAG101 Type 1 galactosemia program. Encouraging preclinical proof-of-concept data produced in partnership with Emory researchers.
- University of UtahcoreResearch agreement in place for JAG101 Type 1 galactosemia program. Encouraging preclinical proof-of-concept data produced in partnership with University of Utah researchers.
- Advanced Medicine PartnerscoreManufacturing partner for Jaguar's therapeutics. Described as 'soon to be spun out from Jaguar.'
Scale indicators4 records
Recent moves9 records
Expansion highlights6 records
Jaguar Gene Therapy competitors and assessment
Company assessmentDirect peers
- Regenxbio: Clinical-stage AAV gene therapy platform company developing AAV-based treatments for rare diseases including CNS indications. Direct peer in AAV delivery technology, manufacturing infrastructure, and rare disease strategy.
- Taysha Gene Therapies: Clinical-stage gene therapy company developing AAV-based treatments for rare CNS diseases. Closely comparable to Jaguar in stage, platform (AAV9), therapeutic area (CNS/monogenic), and pipeline depth.
- Passage Bio: Clinical-stage AAV-delivered gene therapy company focused on rare monogenic CNS diseases, including programs using AAV1 and AAVrh10. Directly comparable target patient population, delivery modality, and pipeline stage to Jaguar's JAG201.
- Solid Biosciences: Clinical-stage gene therapy company developing AAV-based treatments for rare genetic diseases, including CNS and muscular indications. Comparable in modality (AAV), target patient population (rare/monogenic), and development stage.
- Voyager Therapeutics: Clinical-stage gene therapy company developing AAV-based treatments primarily for CNS diseases including Parkinson's and Huntington's. Comparable in CNS-targeted AAV platform, pipeline stage, and rare disease focus.
Broad incumbents
- Novartis (AveXis): Owner of Zolgensma — the first FDA-approved AAV9 gene therapy developed by the same leadership team now at Jaguar. Functions as both a direct platform/technology peer and the source of Jaguar's key personnel and IP heritage.
- Sarepta Therapeutics: Established commercial-stage genetic medicines company with multiple approved AAV and gene editing therapies for rare neuromuscular diseases. Comparable in gene therapy modality and rare disease focus, but at significantly larger scale and commercialization stage.
- BioMarin Pharmaceutical: Commercial-stage rare disease company with approved AAV gene therapy (Roctavian for hemophilia A) and a broader rare disease pipeline. Comparable in gene therapy modality and rare disease commercial strategy, but at substantially larger scale.
- Ultragenyx Pharmaceutical: Commercial-stage rare disease company developing multiple modalities including gene therapy for serious genetic conditions. Comparable in focus on severe genetic/rare diseases, but with a much larger approved-product portfolio.
- Pfizer (Gene Therapy Unit): Large pharmaceutical company with an active gene therapy franchise (e.g., beqvez for hemophilia B) and ongoing AAV pipeline. Comparable as a competitor for AAV clinical talent, manufacturing capacity, and rare disease partnerships.
Market position
Strengths4 records
Weaknesses4 records
Competitive moat5 records
Key risks5 records
Key highlights6 records
Customer concentration
Jaguar Gene Therapy social profiles
Digital presenceJaguar Gene Therapy financial estimates
Financial estimateRevenue estimate
Valuation estimate
Jaguar Gene Therapy leadership team
Management profileNumber of profiles
Profiles13 records
Jaguar Gene Therapy subsidiaries and ownership
Company hierarchySubsidiaries1 record
Jaguar Gene Therapy funding detail
Funding detailFunding overview
Funding rounds4 records
Investors7 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Jaguar Gene Therapy 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 Jaguar Gene Therapy
What does Jaguar Gene Therapy do?
Jaguar Gene Therapy develops investigational AAV9-based gene therapies targeting the root causes of severe genetic diseases with no FDA-approved treatments. Its lead candidate JAG201 delivers a functional SHANK3 gene to treat Phelan-McDermid syndrome/genetic autism, while preclinical programs JAG101 (GALT for Type 1 galactosemia) and JAG301 (PAX4 for Type 1 diabetes) extend the pipeline. All programs leverage the company's internal CMC capabilities and commercial-scale GMP manufacturing facility in Durham, NC.
Is Jaguar Gene Therapy a public or private company?
Jaguar Gene Therapy is a private company. It is classified as venture growth investor backed and is currently operating.
When was Jaguar Gene Therapy founded?
Jaguar Gene Therapy was founded in 2020. It employs 11 to 50 people.
Where is Jaguar Gene Therapy based?
Jaguar Gene Therapy is headquartered in Lake Forest, United States, in the North America region.
How does Jaguar Gene Therapy make money?
One revenue line is on record: gene Therapy Development and Commercialization.
Who are Jaguar Gene Therapy's main competitors?
Direct peers on record are Regenxbio, Taysha Gene Therapies, Passage Bio, Solid Biosciences and Voyager Therapeutics. Broad incumbents are Novartis (AveXis), Sarepta Therapeutics, BioMarin Pharmaceutical, Ultragenyx Pharmaceutical and Pfizer (Gene Therapy Unit).
Does Jaguar Gene Therapy have an API?
No public API is recorded for Jaguar Gene Therapy.
What industry is Jaguar Gene Therapy in?
Jaguar Gene Therapy's product category is Gene Therapy / Rare Disease Biologics. Its primary akta.pro industry code is HLAAACAA, AAV Vector Gene Therapy Developers, with a secondary code of HLAAACAE, Gene Therapy for Rare/Monogenic Diseases. Its NAICS code is 541714 and its SIC code is 2836.