Date: 2017-12-14
Type of information: Establishment of a new subsidiary in the US
Compound:
Company: Asklepios BioPharmaceutical (USA - NC) Actus Therapeutics (USA - NC)
Therapeutic area: Rare diseases - Genetic diseases - Metabolic diseases
Type agreement: establishment of a new subsidiary in the US
Action mechanism: gene therapy
Disease: Pompe disease
Details:
- • On December 14, 2017, Asklepios BioPharmaceutical (AskBio) announced the formation of Actus Therapeutics, a new portfolio company based on AskBio’s industry-leading gene therapy platform. Actus will benefit from AskBio’s ability to generate organ and tissue-specific single-stranded and self-complementary genome containing adeno-associated virus vectors (AAVs), the company’s manufacturing platform and managements’ experience progressing AAV gene therapy programs into the clinic. Actus will pursue therapies for multiple rare genetic diseases, and will initially focus on developing a gene therapy for Pompe Disease based on the research of Dwight Koeberl, M.D., Ph.D., professor of pediatrics and a medical genetics specialist at Duke University.
- Actus will create targeted low-dose gene therapies for orphan diseases using AskBio’s AAV platform technologies and manufacturing process. The company is currently recruiting 20 adult patients with late-onset Pompe Disease for the clinical trial (NCT03533673). The trial is using AAV2/8-LSPhGAA (Adeno-associated virus serotype 8 carrying the human acid-alpha-glucosidase (GAA) gene under the control of the LSP promoter).
Financial terms:
Latest news:
Is general: Yes
