Regulatory
GPH101 is an investigational next-generation gene-edited autologous hematopoietic stem cell (HSC) therapy.
The FDA placed a clinical hold on the program due to a determination that there is insufficient information to support dose escalation with the product.
The FDA is quickly catching up after COVID-19 disrupted typical review timelines and planned inspections and Monday morning held bad news for Coherus, Junshi and Axsome.
As May gets off to a slow start for the FDA calendar, a few companies have important target action dates.
China’s proposed guidelines for bioethics say that organizations outside of China cannot collect and preserve Chinese human genetic resources inside China or take them out of the country.
The Q1 results for Voxzogo gave BioMarin the confidence to raise its 2022 full-year guidance to between $100 million to $125 million (net product revenue) for the drug.
Editas Medicine snagged Rare Pediatric Disease designation for its experimental beta thalassemia gene therapy, while VBL received Fast Track designation for an ofra-vec combination in ovarian cancer.
Gamida Cell reported that the FDA had lifted its clinical hold on its cryopreserved formulation of GDA-201. It expects to launch a Phase I/II drug trial for those indications this year.
The DSMB for Ocugen’s Phase I/II trial of OCU400, a gene therapy in development for Retinitis Pigmentosa (RP), recommended the study proceed with enrolling more patients.
Shares of Axsome Therapeutics have fallen more than 20% in trading this morning after the company announced the FDA is unlikely to approve its acute migraine treatment AXS-07 due to unresolved quality control issues.
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