Frontera Therapeutics Inc. raised $160 million in a series B funding round to develop its lead gene therapy product candidate for retinal disease, FT-001, for which INDs have been approved by the U.S. FDA and China NMPA.
Recently launched and relatively small Avista Therapeutics Inc. has just cut a deal with comparatively massive Roche Holding AG that could bring the new Pittsburgh-based company more than $1 billion. Avista’s single cell adeno-associated virus (AAV) platform will be used to develop intravitreal AAV capsids that match a Roche-defined capsid profile. Roche will evaluate and license Avista’s capsids and conduct the preclinical, clinical and commercialization work for the gene therapy programs.
Swanbio Therapeutics Inc. closed a $56 million series B round to take its lead gene therapy program, SBT-101, into clinical development later this year. The candidate, comprised of an adeno-associated virus type 9 vector encoding the ABCD1 peroxisomal ATP-binding cassette transporter, is in development for adrenomyeloneuropathy (AMN), an inherited disease that affects the central nervous system.
Although gene therapy is now “a clinical reality,” it still remains an early stage therapeutic modality. That’s the view of Caroline Man Xu, CEO and co-founder of Vigeneron GmbH, a German gene therapy company that has maintained a low profile while steadily staking out a promising position in gene therapies for inherited retinal disease.
Forcefield Therapeutics Ltd. has arrived on the scene with £5.5 million (US$7 million) in funding to advance development of naturally occurring proteins that have been shown to arrest the loss of cardiomyocytes in the immediate aftermath of myocardial infarction.
Novartis AG, an early and active player in bringing gene therapies to market, has agreed to pay Voyager Therapeutics Inc. $54 million up front and up to $1.7 billion in fees and milestone payments for options to license up to five next-generation adeno-associated virus (AAV) capsids to use as gene therapy vectors for neurological diseases.
LONDON – In the largest-ever series A for a Spanish biotech, Splicebio S.L. has raised €50 million (US$56.9 million) to apply its protein splicing technology to the delivery of large genes that do not fit into existing vectors. The company claims its approach will overcome the capacity constraints of adeno-associated viral vectors (AAVs), by splitting genes into parcels and reconstituting the proteins they express in vivo.
Combining Sarepta Therapeutics Inc.’s gene editing technology and Genedit Inc.’s Nanogalaxy platform to treat neuromuscular disorders shows promising potential, the companies reported. A year into the research collaboration, Genedit’s polymer nanoparticles have demonstrated the ability to deliver therapeutic cargo to specific muscle tissue following system administration of targeted genetic medicines.
Japan’s Astellas Pharma Inc. is continuing its investment in gene therapies, following up its $3 billion acquisition of Audentes Therapeutics Inc. with a technology licensing deal with Dyno Therapeutics Inc. potentially worth more than $1.6 billion. Central to the deal is Cambridge, Mass.-based Dyno’s adeno-associated virus (AAV) vector technology, which can be used to direct gene therapies to skeletal and cardiac muscle.
