Avrobio Inc. said Feb. 8 that the first person dosed with AVR-RD-01, an investigational ex vivo lentiviral gene therapy from its upgraded manufacturing platform, Plato, experienced a complete clearance of the toxic substrate lyso-Gb3 in a kidney biopsy.
While it’s too late to save the contingent value rights connected with the acquisition of Celgene Inc., Bristol Myers Squibb Co.’s CD19-targeted CAR T therapy, lisocabtagene maraleucel, for treating certain types of relapsed or refractory large B-cell lymphoma patients who have received at least two prior therapies, won FDA approval.
At the recent 39th J.P. Morgan Healthcare Conference, Biomarin Pharmaceutical Inc. popped the lid off top-line results from its ongoing phase III GENEr8-1 study with valoctocogene roxaparvovec – also known as valrox, now commonly called Roctavian. Data, though encouraging, may not have quelled controversy around the prospect.
LONDON – A remodeled Rexgenero Ltd. has launched as an integrated cell and gene therapy specialist, following integration of a French biotech acquired last year and a commitment from the company’s two shareholders to invest £40 million (US$54 million).
Lexeo Therapeutics Inc., a New York-based startup initially advancing clinical and near-clinical stage candidates for Friedreich’s ataxia, CLN2 and Alzheimer's diseases, has raised an $85 million series A financing round led by Longitude Capital and Omega Funds. Founded by gene therapy development veteran Ronald Crystal, the company is led by CEO Nolan Townsend, the former head of Pfizer Inc.'s rare disease efforts in North America.
Shares of Voyager Therapeutics Inc. and its partner, Neurocrine Biosciences Inc., fell in early trading Dec. 23 after Voyager announced an FDA clinical hold on the phase II Restore-1 trial of VY-AADC, an adeno-associated virus-based gene therapy Neurocrine is developing as NBIb-1817 for the treatment of Parkinson's disease. The trial had been paused since at least November as its data safety monitoring board reviewed MRI abnormalities in some study participants.
Prevail Therapeutics Inc. won't be prevailing on its own. Eli Lilly and Co. is acquiring the gene therapy company for $880 million up front plus an earn-out of up to $160 million if Lilly can gain regulatory approval for at least one of Prevail's drugs.
Precision Biosciences Inc. CEO Matthew Kane said the company is in “active discussions around additional partnerships in vivo and in other areas across our organization,” after scoring a deal with Eli Lilly and Co. centered on the firm’s Arcus genome-editing platform. “There’s no conceivable way in the near term that we’re going to advance all of the possibilities of Arcus on our own,” he said.
Metagenomi Inc. has raised a $65 million series A financing to expand its gene editing abilities, advance its research and validate its pipeline in preclinical studies. The company’s CRISPR-based systems use algorithms for screening thousands of genomes from microorganisms to advance therapies for use in oncology, genetic diseases and possibly much more.
Shares of Bluebird Bio Inc. (NASDAQ:BLUE) sank 16.6%, or $9.72, to close at $48.83 as Wall Street reacted to news that the U.S. regulatory filing for Lentiglobin in sickle cell disease (SCD) will be delayed. Previously expected in the second half of next year, the filing won’t happen until late 2022.
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