12:44 , Mar 21, 2019 |  BC Innovations  |  Emerging Company Profile

A Passage to optimal CNS vectors

Passage is arming itself with AAV vectors, gene therapies and non-human primate studies from gene therapy pioneer James Wilson at UPenn to develop disease-modifying treatments for rare monogenic diseases affecting the CNS. The company launched...
19:29 , Dec 21, 2018 |  BC Week In Review  |  Company News

Axovant gets rights to gene therapies from UMass medical school

The University of Massachusetts Medical School (Worcester, Mass.) granted Axovant exclusive, worldwide rights to develop and commercialize two gene therapies to treat GM1 and GM2 gangliosidoses. The school received $10 million up front and is...
19:22 , Jul 28, 2017 |  BioCentury  |  Regulation

Ganging up in rare disease

A joint proposal from EMA and FDA that encourages companies to join multidrug platform trials for pediatric Gaucher’s disease came too late to serve its original purpose. But the proposal could provide a template for...
23:06 , Feb 8, 2017 |  BC Extra  |  Financial News

Lysogene raises EUR 22.6M in IPO

Lysogene S.A. (Euronext:LYS) raised EUR 22.6 million ($24.3 million) through the sale of 3.3 million shares at EUR 6.80 in an IPO underwritten by Societe General and Gilbert Dupont. The offering values the company at...
22:41 , Jan 20, 2017 |  BC Week In Review  |  Clinical News

LYS-GM101 regulatory update

Lysogene said FDA granted rare pediatric disease designation to LYS-GM101 to treat GM1 gangliosidosis. The company plans to begin a Phase I/II trial of LYS-GM101 in 1H18. The product is an adeno-associated virus (AAV) serotype...
07:00 , May 8, 2014 |  BC Innovations  |  Distillery Therapeutics

Indication: Endocrine/metabolic disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Endocrine/metabolic disease Glycosphingolipid storage disorders Galactosidase b1 (GLB1); ganglioside GM1 (GM1) Feline studies suggest intracranial delivery of adeno-associated viral (AAV) vectors encoding GLB1 could help treat...
07:00 , Apr 21, 2014 |  BioCentury  |  Emerging Company Profile

Minoryx: Mediating rare metabolism

Minoryx Therapeutics S.L. has two complementary strategies for developing therapies to treat inherited metabolic diseases involving enzyme deficiencies: identifying small molecule chaperones that can restore mutant enzyme function, and repurposing existing drugs to treat the...