Company intelligence / Profile preview
AskBio
Research Triangle Park, North Carolina, USAaskbio.com ↗
- Ownership
- Private
- Employees
- ~900
- Development stage
- Other
01
Overview
AskBio has a rapidly expanding pipeline of investigational AAV-based gene therapies targeting rare genetic diseases. Several proprietary vectors have entered clinical testing. The company leverages its platform to develop treatments aimed at erasing genetic diseases with ongoing clinical programs in neuromuscular and CNS indications. It is advancing multiple candidates through early to mid-stage clinical development[1][2][3][4][5][6].
- Therapeutic areas
- PulmonologyRare DiseasesNeurologyCardiovascular
- Modalities
- AAV (Adeno-associated virus) gene therapy vectorsProprietary cell line manufacturing process (Pro10™)Synthetic promoters and DNA technologyExtensive capsid library for gene delivery
- Industry
- Biotech
02
Drug pipeline
16 assets15 more assets in the full pipeline.
03
Partnerships
Collaborations/licensing agreements with Pfizer (Duchenne muscular dystrophy)Takeda (Hemophilia via Chatham Therapeutics)Novartis/AveXis among others who license or use their technologies[3][4].
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