One-year KYSA-8 and KYSA-6 data showed durable miv-cel benefit in SPS and gMG, supporting Kyverna's rolling BLA submission targeted for Q4 2026.
|Articles|October 10, 2018
Sharing Insight on Myeloma, CAR T, and More in San Francisco
Author(s)Unknown Author
We visited San Francisco, California for a State of the Science Summit on Hematologic Malignancies. The meeting covered novel agents and combinations in lymphoma, updates in acute and chronic leukemias, and modalities in the management of multiple myeloma.
Advertisement
We visited San Francisco, California for a State of the Science SummitTM on Hematologic Malignancies. The meeting covered novel agents and combinations in lymphoma, updates in acute and chronic leukemias, and modalities in the management of multiple myeloma.
Advertisement
Related to this article
FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time AAV9 gene therapy, for pediatric patients with Sanfilippo syndrome type A, based on cognitive data from an open-label trial in children 2 to 5 years old.
Ocugen dosed the first patient in ArMaDa3, a global phase 3 trial of the one-time gene therapy OCU410 for geographic atrophy, following FDA RMAT designation and phase 2 data showing a 31% reduction in lesion growth versus control.
Novartis suspended several autoimmune and neurology trials of rap-cel after 3 patient deaths linked to immune effector cell-associated hemophagocytic syndrome, and BMS separately paused zola-cel studies after observing inflammatory safety events.
The FDA placed a new clinical hold on REGENXBIO's RGX-121 for Hunter syndrome after asymptomatic spine MRI findings were identified in 5 trial participants, the gene therapy's second regulatory setback this year.
The FDA extended its review of deramiocel for Duchenne muscular dystrophy to November 22, 2026, after accepting additional HOPE-3 data as a major BLA amendment.
PTC Therapeutics wins Sangamo's BLA-stage Fabry disease gene therapy ST-920 for $111 million in a bankruptcy auction, with STAAR data supporting accelerated approval.
FDA granted accelerated approval to pariglasgene brecaparvovec-opnr, a one-time liver-directed AAV8 gene therapy, based on a 31% reduction in cornstarch intake versus placebo.
Advertisement
Advertisement