This author has yet to write their bio.Meanwhile lets just say that we are proud old admin contributed a whooping 84 entries.
HIGH IMPACT – CSL/uniQure’s Hemgenix (etranacogene dezaparvovec, Etranadez) was approved by the FDA for hemophilia B. Key Takeaways Hemgenix was approved for adults with hemophilia B who currently use FIX prophylaxis, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes Hemgenix is the first hemophilia B gene therapy approved and the […]
HIGH IMPACT – Surface Oncology provided a corporate update, including the decision to pause internal clinical development of SRF617 (CD39 mAb) and early clinical data for SRF388 monotherapy in NSCLC. The company intends to open a SRF388 + pembrolizumab study for 2-4L NSCLC. Pipeline updates: SRF617 (CD39 mAb) development will be paused, with a corresponding reduction […]
HIGH IMPACT – Travere hosted a call to discuss the recent regulatory update for sparsentan Travere’s sparsentan was previously assigned a PDUFA target action date of November 17, 2022 for its NDA under accelerated approval for the treatment of IgAN, which has been extended by 3 months following late-cycle review interactions with the FDA. The […]
HIGH IMPACT – Wave Life Sciences hosted an investor call titled “Towards the Clinic: Spotlight on RNA Editing for AATD” to discuss their technology, preclinical results, and path forward for WVE-006, their mRNA editing treatment for patients with alpha-1 anti-trypsin deficiency (A1ATD). On the call: Dr. Paul Bolno (CEO), Dr. Paloma Giangrande (VP, WVE-006 Program […]
HIGH IMPACT – In an SEC filing, BioMarin disclosed that they were notified on August 19th, 2022 that a participant in the Roctavian Phase 3 trial was diagnosed with B-cell acute lymphoblastic leukemia (B-ALL). Based on BioMarin’s assessment of the case to date, including initial genetic testing of the leukemic cells, BioMarin believes at this […]
