Increased payload capacity for gene therapies, off-the-shelf genome-engineered allogeneic cell therapies, reduced cost of goods and faster bioprocesses, are promised by “big DNA” specialist Replay Holdings LLC. The newco arrived with a $55 million seed round and having assembled a portfolio of technologies for writing and delivering large pieces of DNA.
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.
VBL Therapeutics Inc.’s phase III study of its gene therapy, ofranergene obadenovec, in treating ovarian cancer missed its primary endpoints, prompting the company to discontinue the trial and investors to pull way back. Shares of the Tel Aviv, Israel, and New York-based company’s stock (NASDAQ:VBLT) plunged 79% on July 20 to close at 43 cents per share. The $1.62 per share drop in value made it a penny stock. VBL shares hit their high on Nov. 8, closing at $2.53 each. Top-line data for ofra-vec, also known as VB-111, showed no statistically significant improvement in progression-free survival or overall survival.
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.
The U.S. FDA has placed a clinical hold on Astellas Pharma Inc.’s Fortis phase I/II trial evaluating AT-845 following a serious adverse event of peripheral sensory neuropathy in one of the trial participants. AT-845 is an adeno-associated virus (AAV) gene replacement therapy being studied in adults with late-onset Pompe disease.
The U.S. FDA has placed a clinical hold on Astellas Pharma Inc.’s Fortis phase I/II trial evaluating AT-845 following a serious adverse event of peripheral sensory neuropathy in one of the trial participants. AT-845 is an adeno-associated virus (AAV) gene replacement therapy being studied in adults with late-onset Pompe disease.
Uniqure NV rolled out stock-pleasing safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing phase I/II trial with AMT-130 for the treatment of Huntington’s disease (HD), but investors must wait for details on MRI scans. “We have communicated that we will discuss the MRI findings in the middle of next year,” CEO Matt Kapusta told investors during a conference call. “That’s all I can say.”
In keeping with the 21st Century Cures Act, the U.S. FDA issued a draft guidance describing a standards recognition program for regenerative medicine therapies at the Center for Biologics Evaluation and Research that’s intended to facilitate the development of the therapies and reduce the regulatory burden.
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