Siren Biotechnology Inc. has unveiled its lead asset, SRN-101, for the treatment of high-grade gliomas. The FDA has granted orphan drug and rare pediatric disease designations to SRN-101 for high-grade gliomas and pediatric-type diffuse high-grade gliomas, respectively.
Neurogene Inc.’s stock sank 43% on news that its phase I/II gene therapy, NGN-401, resulted in a serious adverse event in a pediatric Rett syndrome patient receiving the highest dose.
PTC Therapeutics Inc.’s accelerated go-ahead from the U.S. FDA for Kebilidi (eladocagene exuparvovec-tneq), a recombinant adeno-associated virus serotype 2-based gene therapy to treat aromatic L-amino acid decarboxylase (AADC) deficiency, came with a priority review voucher (PRV) that the company intends to monetize.
