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.
At the virtual Cell & Gene Meeting on the Mesa, panelists talked about the challenges and benefits of developing cell and gene therapies to treat chronic conditions.
As with many conferences, the Cleveland Clinic’s 2020 Medical Innovation Summit went virtual this year. Still, the event featured the hotly anticipated top 10 list of innovations for 2021 that saw a range of therapies. Ranked in order of expected importance, the list was led by gene therapy for hemoglobinopathies. The top three innovations, including a novel drug for primary-progressive multiple sclerosis and smartphone-connected pacemaker devices, were highlighted in a special presentation.
DUBLIN – Versant Ventures, the founding investor of Crispr Therapeutics AG, is teaming up with one of the academic founders of that company, Stanford University’s Matt Porteus, to start a new gene editing firm, Graphite Bio Inc., which is focused on targeted integration of DNA to achieve a therapeutic benefit.
DUBLIN – Mina Therapeutics Ltd. raised £23 million (US$29.9 million) in a series A round to take forward a pipeline and a platform based on a novel regulatory RNA species, small activating RNAs, which selectively activate gene expression.
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