Japanese specialty global pharma Kyowa Kirin Co. Ltd. has agreed to buy out Orchard Therapeutics plc in a $387.4 million cash takeover that could jump $90 million to reach $477.6 million, contingent on the pending U.S. FDA approval of its EU-approved gene therapy, Libmeldy (atidarsagene autotemcel).
Japanese specialty global pharma Kyowa Kirin Co. Ltd. has agreed to buy out Orchard Therapeutics plc in a $387.4 million cash takeover that could jump $90 million to reach $477.6 million, contingent on the pending U.S. FDA approval of its EU-approved gene therapy, Libmeldy (atidarsagene autotemcel).
Though data won’t be available for a few years, the disclosure in mid-July that Grifols SA completed enrollment in the phase III study called Sparta caused some ears to perk up in the alpha-1-antitrypsin deficiency (AATD) space, where a number of contenders are busy.
The combination of new U.S. FDA phase II study guidance and a $175 million underwritten public offering sent gene therapy developer Rocket Pharmaceuticals Inc.’s stock soaring on Sept 13. Shares (NASADQ:RCKT) closed 38.8% upward to $21.23 each on Sept. 13.
In its second big pharma deal to date, Shape Therapeutics Inc. drew Otsuka Pharmaceutical Co. Ltd. to the table in a potential $1.5 billion-plus collaboration initially aimed at developing gene therapies for ocular diseases. The multitarget agreement, which includes options for additional targets and tissue types, will combine Shape’s AI-driven adeno-associated virus (AAV) platform and Otsuka’s expertise in ophthalmology to develop intravitreally delivered AAV therapies.
Uniqure NV has received FDA clearance of its IND application for AMT-260, the company’s gene therapy candidate that represents a potential one-time administered approach to treating refractory mesial temporal lobe epilepsy (MTLE).
Ferring Pharmaceuticals A/S has struck a deal with Royalty Pharma for $500 million, capital it plans to plough into achieving a successful launch of its approved bladder cancer gene therapy, Adstiladrin (nadofaragene firadenovec), in the U.S., as well as enabling it to upgrade and expand its manufacturing sites.
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.
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.
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