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.
LONDON – Bayer AG is making a major move into gene therapy with the $4 billion acquisition of one of the pioneers of the field, Asklepios Biopharmaceutical Inc. The deal will give the German pharma access to an adeno-associated viral vector platform that has generated multiple commercial and clinical stage assets across a broad range of indications from rare diseases to chronic conditions. The in-house portfolio includes treatments for Pompe and Parkinson’s diseases and congestive heart failure. Asklepios (Askbio) also has spun out programs in hemophilia and Duchenne muscular dystrophy.
Solid Biosciences Inc. already has its own Duchenne muscular dystrophy program, but with some new investment money it is plunging into a collaboration with Ultragenyx Pharmaceutical Inc. to co-create another program. The two will collaborate on developing and commercializing new gene therapies for treating Duchenne muscular dystrophy as Novato, Calif.-based Ultragenyx is investing $40 million in Solid.
Aavantibio Inc., with the help of a large check from Sarepta Therapeutics Inc., has launched with a $107 million series A to pursue gene therapies for treating rare genetic diseases. The lead program is in Friedreich’s ataxia (FA), a rare genetic disease that typically begins in childhood and causes progressive damage to the nervous system.
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