22:30 , Feb 2, 2018 |  BioCentury  |  Product Development

AA Viral meme

Gene therapy pioneer James Wilson’s revelation of two new types of toxicity in animals receiving high systemic doses of adeno-associated viral vectors is unlikely to herald a major disruption of the AAV space, though it...
08:00 , Nov 17, 2014 |  BC Week In Review  |  Company News

Evotec, Jain Foundation deal

Evotec and Jain extended and expanded their 2012 deal to develop a cell-based high throughput screening assay using dysferlin ( DYSF )-deficient cells to identify compounds to treat dysferlinopathy. Under the extension, Evotec began screening...
07:00 , Sep 11, 2014 |  BC Innovations  |  Distillery Therapeutics

Indication: Musculoskeletal disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Musculoskeletal disease Muscular dystrophy Dysferlin (DYSF) Studies in patients suggest proteasome inhibitors could help treat muscular dystrophies caused by DYSF mutations. In three patients with muscular...
08:00 , Dec 9, 2013 |  BC Week In Review  |  Company News

Cellular Dynamics, Jain Foundation deal

Cellular Dynamics signed a master service agreement with the foundation and will create five induced pluripotent stem cell (iPSC) lines from patients with muscular dystrophies caused by dysferlin (DYSF) deficiency. Cellular Dynamics will use its...
07:00 , Sep 2, 2013 |  BC Week In Review  |  Company News

Evotec, Jain Foundation deal

Evotec and the foundation extended and expanded their 2012 deal to develop a cell-based high throughput screening assay using dysferlin ( DYSF )-deficient cells to identify compounds to treat dysferlinopathy. The partners declined to disclose...
08:00 , Mar 1, 2012 |  BC Innovations  |  Distillery Therapeutics

Indication: Musculoskeletal disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Musculoskeletal disease Muscular dystrophy Dysferlin (DYSF); proteasome Cell culture studies suggest inhibiting proteasome-mediated degradation of DYSF could help treat muscular dystrophy. In human myoblasts with missense...
08:00 , Dec 2, 2010 |  BC Innovations  |  Distillery Therapeutics

Indication: Musculoskeletal disease

This week in therapeutics Indication Target/marker/pathway Summary Licensing status Publication and contact information Musculoskeletal disease Muscular dystrophy Complement 3 (C3) Mouse studies suggest that inhibiting the complement factor system in skeletal muscle could help treat...