Cell and Gene Therapy
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Not developing potency assays and gaining knowledge about MOAs early in the drug development process not only can break ATMP success but can cause costs and delays that lead to company closures.
The payment scheme will tie gene therapy payments to improvements in health outcomes—and could potentially boost the uptake of these sickle cell disease treatments.
In this episode of Denatured, BioSpace’s Head of Insights Lori Ellis, Miguel Forte and Ali Pashazadeh discuss how a slow and steady pace is a continuation of the pattern we have seen throughout the last three years.
Not exactly known for its dealmaking, Sarepta Therapeutics has thrown down a massive wad of cash to work with Arrowhead Pharmaceuticals on RNAi-based medicines.
The cancers were diagnosed 19 to 92 months after Skysona treatment.
Emboldened by technological advances and a deeper knowledge of glioblastoma, Merck, Kazia Therapeutics, CorriXR Therapeutics and others are targeting the often-fatal brain tumor.
Despite the death, the FDA has allowed Neurogene to forge ahead with the Phase I/II Rett syndrome trial, but using only the lower 1E15 vg dose of its investigational gene therapy NGN-401.
Novartis’ up to $1.1 billion acquisition of gene therapy specialist Kate Therapeutics fits with the pharma’s plan to expand its new modality pipeline to ensure long-term business sustainability.
Investigational CAR T therapies stole the spotlight at the American College of Rheumatology Convergence as data presented by Bristol Myers Squibb, Kyverna Therapeutics and more highlighted their potential to effectively treat lupus.
After failing to receive the RMAT designation from the FDA for its early-stage Batten disease gene therapy, Neurogene tells investors that it’s evaluating options for the program.