Pharmacologists at LMU have developed gene-based agents for the treatment of congenital eye diseases. The first of these is now undergoing a phase-I clinical trial in color-blind patients at the University Medical Center in Tübingen. Is this approach translatable in principle to other visual disorders? Michalakis: About 200 genes have been identified which, when mutated, lead to monogenetic …
gene therapy
Avalanche Biotechnologies Closes Transaction With Annapurna Therapeutics
Avalanche Biotechnologies, Inc. (AAVL) today announced the closing of the previously announced transaction with Annapurna Therapeutics. “The new management team is focused on applying our extensive clinical development expertise to advance the company’s gene therapy programs into the clinic. We intend on dosing our first patients for A1AT deficiency by the end of this year and for HAE in 2017 …
Advancing Therapeutic Strategies for Inherited Retinal Degeneration: Recommendations From the Monaciano Symposium
Over the past 3 decades, global research efforts have unveiled the genetic complexity of the group of rare disorders collectively referred to as retinal dystrophies. Causative mutations for dozens of retinal dystrophies have now been identified, resulting in the discovery of multiple new genes, biological pathways necessary for photoreceptor cell function, and pathogenetic mechanisms involved in …
Spark Surges in IPO as Prices Fuel Gene Therapy
Spark Therapeutics Inc. hit a $1.2 billion valuation on the biotechnology firm’s first day on the market, reflecting growing investor enthusiasm for the once-beleaguered field of gene therapy, a field that has produced the first $1 million drug. The treatments focus on diseases where a single mutation in the genetic code is known to be the cause. Fix or replace the genetic code, and, …