AskBio
AcquiredDurham, US · 510 employees on LinkedIn · 6 known investors
AskBio is a gene therapy company developing adeno-associated virus (AAV)-based genetic medicines for rare and other diseases, with clinical programs such as AB-1009 for late-onset Pompe disease. It operates as a wholly owned, independent subsidiary of Bayer AG.
Also known as AskBio Inc. · Asklepios BioPharmaceutical, Inc.
Investors · 6
Also in the syndicate · 3
Company profile
researched Aug 2026Asklepios BioPharmaceutical, Inc. (AskBio) is a clinical-stage gene therapy company built around adeno-associated virus (AAV) technology. Its integrated platform includes the Pro10 proprietary cell line manufacturing process, an extensive library of chimeric AAV capsids, synthetic promoters and DNA, self-complementary AAV, and delivery methods intended to lower the cost and increase the supply of gene therapy production. The company reports over 350,000 square feet of laboratory, research and in-house AAV manufacturing space and a portfolio of more than 800 patents and patent applications covering gene therapy development, clinical processes and manufacturing.
The clinical pipeline spans central nervous system, neuromuscular, metabolic and cardiovascular disorders, with lead research programs historically focused on Pompe disease, Parkinson's disease and congestive heart failure. Out-licensed clinical candidates cover hemophilia (via Chatham Therapeutics, acquired by Takeda) and Duchenne muscular dystrophy (via Bamboo Therapeutics, acquired by Pfizer). AskBio's technology and AAV therapeutic assets have been acquired or licensed by Bayer, Pfizer, Takeda and Novartis. Alongside its own pipeline, the company operates a contract development and manufacturing organization (CDMO) based on the Pro10 cell line.
AskBio is headquartered in Research Triangle Park, North Carolina, and has operated as a wholly owned, independently operated subsidiary of Bayer AG since late 2020, forming a cornerstone of Bayer's Cell & Gene Therapy platform.
Founding story
Founded in 2001 by R. Jude Samulski, PhD, the first scientist to clone AAV; Xiao Xiao, PhD, who first developed a miniaturized dystrophin gene enabling gene therapy for Duchenne muscular dystrophy; and Sheila Mikhail, JD, MBA, a life sciences executive who later served as CEO.
Business model
AskBio develops proprietary AAV gene therapy candidates while also licensing or out-licensing technology and therapeutic assets to larger pharmaceutical companies and operating a revenue-generating contract development and manufacturing organization built on its Pro10 cell line process.
Revenue sources described in the sources include contract development and manufacturing services for third parties and licensing/out-licensing of AAV technology and therapeutic assets to pharmaceutical partners such as Pfizer, Takeda and Novartis.
Traction
The company has advanced multiple clinical-stage programs, including dosing of the first participant in PROGRESS-GT LOPD, a Phase 1/Phase 2 trial of AB-1009 for late-onset Pompe disease. It presented six abstracts at the European Society of Gene and Cell Therapy 32nd Annual Meeting in 2025 and reports approximately 527 employees as of July 2026 with estimated annual revenue of $100M-$250M per a third-party business data provider.
Latest developments
AskBio dosed the first participant in the PROGRESS-GT LOPD Phase 1/Phase 2 trial of AB-1009 for late-onset Pompe disease. It entered a strategic partnership with China-based Belief BioMed in September 2024 to advance new gene therapies, announced plans in April 2024 to roughly double its Triangle-area footprint with space in Morrisville, North Carolina, and presented six abstracts at the European Society of Gene and Cell Therapy 32nd Annual Meeting in October 2025.
▸Full profile — market position, technology, go-to-market, geography, history
Market position
AskBio positions itself as an early innovator and established platform company in AAV gene therapy, noting that two of the first FDA-approved gene therapies are based on the research of its founders and its technology, and that its assets have been acquired or licensed by Bayer, Pfizer, Takeda and Novartis. Comparable companies cited include Rocket Pharmaceuticals, Orchard Therapeutics, bluebird bio, Adverum Biotechnologies, GenSight Biologics and Dyno Therapeutics.
Differentiators cited include the Pro10 cell line manufacturing process, a large chimeric capsid and synthetic promoter library, self-complementary AAV, an intellectual property estate of more than 800 patents and applications, scaled in-house manufacturing across more than 350,000 square feet, and founder-level scientific provenance in AAV cloning.
Technology
The platform is built on AAV vectors and includes the Pro10 high-yield cell line manufacturing process, an expansive chimeric capsid library, synthetic promoters and DNA, and self-complementary AAV. The company reports hundreds of proprietary third-generation AAV capsids and promoters designed for improved efficacy, immune response profile, and tissue and organ specificity, plus new delivery methods aimed at accelerating and lowering the cost of therapeutic production.
Go-to-market
Clinical development of internal candidates is combined with research collaborations, licensing and out-licensing of assets to large pharmaceutical companies, partnerships such as the collaboration with China-based Belief BioMed, and CDMO services for external customers.
Patients with rare and genetic disorders across central nervous system, neuromuscular, metabolic and cardiovascular indications, together with pharmaceutical and biotechnology partners that license AskBio technology or use its contract manufacturing capabilities.
Geography
US headquarters at 20 T.W. Alexander Drive, Durham/Research Triangle Park, North Carolina, with additional US research and development sites in Philadelphia, Pennsylvania and Upper Arlington, Ohio. European locations include Edinburgh, Scotland (Roslin Innovation Centre); Heidelberg, Germany; Paris, France; and a gene therapy manufacturing facility in San Sebastián, Spain. A business data provider lists employees across North America, Europe and Asia.
History
The scientific origins trace to 1984, when a co-founder asked whether a virus could be used to target genetic disease; the company's scientific co-founder was the first to demonstrate that AAV could be cloned for therapeutic purposes. AskBio was founded in 2001 by Jude Samulski, Xiao Xiao and Sheila Mikhail. Over time it acquired and sold gene therapy companies, out-licensing hemophilia (Chatham Therapeutics, later acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics, later acquired by Pfizer) programs. In April 2019 it raised $235 million from TPG Capital, Vida Ventures, founders and board members, and in November 2019 received Southeast BIO's "Best Venture Funding" award for that round. Bayer AG agreed to acquire the company in October 2020, and AskBio became a wholly owned, independently operated Bayer subsidiary in late 2020.
Compiled by commissioned research from 8 cited public sources — announcements, filings, and press listed under research sources below.
Key figures
latest reportedCompany-reported or press-reported figures, each dated to when it was claimed — not independently audited.
Timeline · 6
launches, deals, and filingsAskBio dosed the first participant in PROGRESS-GT LOPD, a Phase 1/Phase 2 clinical trial evaluating the investigational AAV gene therapy AB-1009 in late-onset Pompe disease.
AskBio announced six presentations at the European Society of Gene and Cell Therapy 32nd Annual Meeting.
AskBio entered a strategic partnership with China-based Belief BioMed to advance the development of new gene therapies.
AskBio announced plans to roughly double its local footprint by acquiring space on Airport Road in Morrisville, North Carolina.
Bayer announced an agreement to acquire Asklepios BioPharmaceutical, adding an AAV gene therapy platform, IP portfolio and CDMO to its Cell & Gene Therapy unit. Terms were USD 2 billion upfront and up to USD 2 billion in success-based milestones, with roughly 75% of contingent payments expected within five years. AskBio was to continue operating autonomously on an arm's-length basis; closing was expected in Q4 2020. Existing minority shareholders TPG Capital and Vida Ventures supported the transaction.
$2B source ↗
Southeast BIO gave AskBio its "Best Venture Funding" award at the SEBIO Investor & Partnering Forum in Miami, recognizing the company's $235 million round earlier in 2019.
Dated company events from announcements, filings, and press; legal rows summarize public dockets and regulator releases.
▸Research sources · 8
primary sources listed
- AskBioaskbio.com · web
8 public sources were cited for this profile; the first-party ones are listed here.
Frequently asked questions
- What does AskBio do?
- AskBio is a Bayer-owned AAV gene therapy company developing treatments for neuromuscular, CNS, cardiac and metabolic diseases.
- Who are AskBio's investors?
- AskBio's investors include TPG, Vida Ventures, Columbus Venture Partners.
- Where is AskBio headquartered?
- AskBio is headquartered in Durham, US.