Asklepios BioPharmaceutical

What Asklepios BioPharmaceutical does

Asklepios BioPharmaceutical, Inc. (AskBio) is a clinical-stage biotechnology company developing adeno-associated virus (AAV) gene therapies for genetic disorders, founded in 2001 by R. Jude Samulski and Sheila Mikhail on the basis of Samulski's pioneering AAV research dating to 1984. Headquartered in Research Triangle Park, North Carolina, the company operates across five countries with over 900 employees and runs an integrated AAV technology platform composed of proprietary chimeric capsids, Synpromics synthetic promoters designed via the PromPT bioinformatics engine, the Pro10 high-yield HEK293 suspension cell line, and neDNA enzymatic DNA technology (licensed from Touchlight). Clinical and commercial GMP AAV manufacturing is delivered through its Spanish subsidiary Viralgen, and enzymatic DNA raw material through TAAV Biomanufacturing Solutions, giving AskBio control of the full AAV production chain from capsid design through commercial supply.

AskBio's clinical pipeline includes AB-1002 (Phase 2 in non-ischemic congestive heart failure, enrollment completed April 2026 across 12 countries), AB-1003 (Phase 1/2 in limb-girdle muscular dystrophy 2I/R9), AB-1005/AAV2-GDNF (Phase 2 in Parkinson's disease and Phase 1 in MSA-Parkinsonian type), and AB-1009 (Phase 1/2 in late-onset Pompe disease, first patient dosed May 2026). Each lead asset carries multiple regulatory designations including FDA Fast Track, and in some cases RMAT, Orphan Drug, Rare Pediatric Disease, MHRA Innovation Passport, and Japan Pioneering Regenerative Medical Product status. The company monetizes today through CDMO fees (Viralgen), technology licensing royalties (Pfizer, Bayer, Takeda, Novartis), and sponsored research collaborations (ReCode, Editas, SQZ, Belief BioMed, Selecta); commercial product sales are pre-revenue.

Since October 2020, AskBio has been a wholly owned subsidiary of Bayer AG, acquired for approximately $2 billion, and operates on an arm's-length basis as the cornerstone of Bayer's Cell & Gene Therapy Platform. Bayer board representation, ongoing capital support from the parent, and a recent leadership refresh (CEO Gustavo Pesquin in March 2023, CSO Mansuo Shannon in 2024) position the company to advance late-stage clinical readouts, including initial GenePHIT CHF results expected in H1 2027.

Name

Asklepios BioPharmaceutical

Legal name

Asklepios BioPharmaceutical, Inc.

Website

https://askbio.com

Company type

Private

Founded year

2001

Operating status

Operating

Headcount range

501–1,000 employees

Short description

Asklepios BioPharmaceutical (AskBio) is a clinical-stage biotechnology company developing AAV gene therapies for cardiovascular, neuromuscular, metabolic, and CNS disorders, operating an integrated platform of proprietary capsids, synthetic promoters, high-yield cell lines, and enzymatic DNA manufacturing across five countries as a wholly owned Bayer AG subsidiary.

Ownership category

akta.pro rank

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

AAV Vector Gene Therapy Developers (HLAAACAA)

akta.pro secondary industries

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), Viral Vector Manufacturing Services (AAV, lentiviral, adenoviral) (HLAAAKAD)

Keywords

• AAV gene therapy
• Biopharmaceutical therapeutics
• CDMO manufacturing services
• Genetic disorder treatments
• Cell and gene therapy

Headquarters

HQ city

Chapel Hill

HQ country

United States

HQ region

North America

Markets served

Revenue model

1. Future one-time gene therapy product sales: Investigational AAV gene therapies (AB-1002 for CHF, AB-1005 for Parkinson's/MSA, AB-1003 for LGMD2I/R9, AB-1009 for LOPD) are pre-revenue in clinical development; intended commercial model is one-time administration per patient at multi-million-dollar price points typical of AAV gene therapeutics.
2. Technology licensing royalties: AAV technology and therapeutic assets licensed to leading pharmaceutical companies including Bayer, Pfizer, Takeda and Novartis; Pfizer's DMD gene therapy program is based on AskBio's technology and clinical materials. Generates licensing/royalty revenue.
3. CDMO / AAV contract manufacturing services (Viralgen): Independently operated Viralgen subsidiary offers cGMP AAV manufacturing services to third parties using Pro10™ cell line; TAAV Biomanufacturing Solutions produces and supplies neDNA™ raw materials. Operates as a focused CDMO organization.
4. Strategic research collaborations and sponsored research: Research collaboration and licensing agreements with universities and biopharma partners (UNC Angelman Syndrome, ReCode single vector gene editing, Editas Medicine in vivo genome editing, SQZ immune tolerization, Belief BioMed, CYTOO, Selecta ImmTOR) generate collaboration funding and milestones.

Pricing tiers

Go-to-market motion1 record

Core offering

Asklepios BioPharmaceutical develops investigational adeno-associated virus (AAV) gene therapies for serious genetic disorders across cardiovascular, neuromuscular, metabolic, and central nervous system indications. Its integrated AAV platform combines proprietary chimeric capsids, Synpromics synthetic promoters, the Pro10™ high-yield suspension cell line, and neDNA™ enzymatic DNA manufacturing, supported by GMP manufacturing through its Viralgen CDMO subsidiary and TAAV Biomanufacturing Solutions.

Product overview

AskBio offers a single integrated AAV gene therapy platform-plus-pipeline architecture anchored by the AskBio AAV Technology Platform, which combines three core components — chimeric capsids, Synpromics synthetic promoters (powered by the PromPT™ bioinformatics engine), and the Pro10™ high-yield suspension cell-line manufacturing system — together with neDNA™ enzymatic DNA technology and GMP manufacturing via the Viralgen CDMO subsidiary. The TAAV Biomanufacturing Solutions subsidiary supplies the proprietary neDNA™ starting material. Built on this platform, AskBio runs a clinical pipeline of investigational AAV gene therapies including AB-1002 (congestive heart failure, Phase 2), AB-1003 (limb-girdle muscular dystrophy 2I/R9, Phase 1/2), AB-1005/AAV2-GDNF (Parkinson's disease Phase 2 and MSA-P Phase 1), and AB-1009 (late-onset Pompe disease, Phase 1/2). Discovery and translational work is delivered through the AskPath™ research collaboration program, while the AskFirst™ program serves as the patient advocacy and education layer connecting patients, advocacy groups, and clinical trial participants to the pipeline.

Differentiator

Problem solved

Functional benefit

Brands

• Pro10™: High-yield HEK293-based suspension cell line technology for scalable AAV manufacturing, offering 2x-10x higher yields than most other transfection methods. Used in Viralgen manufacturing facilities and licensed by leading global biopharma companies.
• neDNA™
• AskFirst™
• Aava™
• PromPT™
• AskPath™
• Viralgen

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

AI capability1 record

Feature7 records

Recent moves6 records

Expansion highlights6 records

Emerging players

• Passage Bio: Clinical-stage AAV gene therapy company focused on CNS rare diseases, with programs in GM1 gangliosidosis, frontotemporal dementia, and Krabbe disease. Partial overlap with AskBio on CNS AAV delivery and neurodegenerative disease pipeline.
• Taysha Gene Therapies: Clinical-stage AAV gene therapy company focused on CNS and rare disease indications with a pipeline across Rett syndrome, GAN, and other monogenic disorders. Comparable platform-and-pipeline structure and CNS AAV delivery approach.

Direct peers

• Regenxbio: Clinical-stage gene therapy company developing AAV-based therapeutics using a proprietary NAV capsid platform. Direct competitor to AskBio in AAV technology licensing and pipeline development, including CNS, retinal, and metabolic disease programs.
• Sarepta Therapeutics: Commercial-stage gene therapy company with multiple approved AAV-based products (Elevidys for DMD, others) and a deep neuromuscular disease pipeline. Directly comparable to AskBio on AAV technology and overlapping neuromuscular indications.
• Krystal Biotech: Commercial-stage gene therapy company with an FDA-approved topical AAV-based therapy (Vyjuvek) and pipeline across respiratory, ophthalmology, and dermatology. Comparable in AAV platform-plus-pipeline model and use of proprietary manufacturing.
• uniQure: Gene therapy company developing AAV-based therapies for CNS, liver-directed, and cardiovascular indications. Operates an AAV manufacturing platform and has commercial AAV product (Hemgenix via CSL partnership), making it a direct competitor in AAV technology and pipeline.
• Solid Biosciences: Clinical-stage gene therapy company developing AAV-based treatments for Duchenne muscular dystrophy and other neuromuscular disorders. Direct competitor in AAV gene therapy for muscle disorders, overlapping with AskBio's LGMD program.
• Voyager Therapeutics: Clinical-stage gene therapy company developing AAV-based therapies for CNS diseases including Parkinson's and Huntington's, with a proprietary capsid engineering platform. Direct overlap with AskBio on Parkinson's program (AB-1005) and capsid technology.

Broad incumbents

• BioMarin Pharmaceutical: Large rare-disease biopharma with an established AAV gene therapy franchise (Roctavian for hemophilia A). Comparable on commercial-stage gene therapy commercialization, regulatory navigation, and rare disease focus.
• Catalent (Paragon Gene Therapy): Major CDMO with dedicated AAV gene therapy manufacturing capabilities through Paragon Gene Therapy. Directly comparable to AskBio's Viralgen subsidiary as a third-party AAV contract manufacturer and platform supplier.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat6 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/ask_bio
• YouTubeyoutube.com/channel/UC-NjG9pxoDcb1NlAr-Oj6vw
• Facebookfacebook.com/askbio
• LinkedInlinkedin.com/company/askbio

Compliance13 records

Revenue estimate

Valuation estimate

Number of profiles

Subsidiaries8 records

Funding overview

Funding rounds4 records

Investors4 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A3 records

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Asklepios BioPharmaceutical