Bloomsbury Genetic Therapies Ltd. announced that the FDA granted orphan drug designation for its investigational liver-targeted gene therapy, BGT-OTCD, for the treatment of ornithine transcarbamylase deficiency (OTCD).
Modalis Therapeutics Corp. – previously Edigene Corp. – has regained full rights to two gene therapy candidates for muscle disorders, MDL-201 and MDL-202, that were co-developed with Astellas Pharmaceutical Inc. since 2019.
Researchers at City of Hope were awarded $32.3 million from the California Institute for Regenerative Medicine (CIRM) to support three novel phase I clinical trials evaluating innovative cell and gene therapy treatments for patients with HIV, acute myeloid leukemia (AML) and severe aplastic anemia.
Six years of collaboration is ending in a buyout, as Decibel Therapeutics Inc. agreed to be acquired by Regeneron Pharmaceuticals Inc. in a deal valued at up to $213 million, including $109 million in equity, with additional payments via contingent value rights linked to clinical and regulatory milestones for DB-OTO, the lead gene therapy program targeting hearing loss.
Researchers from Interius Biotherapeutics Inc. presented the development and preclinical evaluation of a novel gene therapy candidate, INT-2104, as potential candidate for the treatment of B-cell malignancies.
Alexion, Astrazeneca Rare Disease, part of Astrazeneca plc, has entered a definitive purchase and license agreement for a portfolio of preclinical gene therapy programs and enabling technologies from Pfizer Inc.
EG 427 SAS has added a further a further €5 million (US$5.6 million) to its series A, closing the round at €18 million and setting the stage for the first clinical trial of a gene therapy in the treatment of a chronic disorder.
Tenpoint Therapeutics Ltd. raised $70 million in a series A funding round to pursue ambitious plans to reverse vision loss using both ex vivo cell engineering and in vivo cell reprogramming approaches.
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