Dec. 19, 2024 -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA or the Agency) seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). “The path to get a treatment to the point of a BLA filing has been long and perilous for the Sanfilippo community. They have had to watch their children, once thriv

Trial is evaluating VIPER-101, a novel, gene edited, autologous dual-population CAR-T therapy in patients with T cell lymphoma VIPER-101 was developed with Vittoria’s proprietary Senza5 platform that features CD5 modulation and a proprietary 5-day manufacturing process Dec. 19, 2024 -- Vittoria Biotherapeutics, Inc., a clinical-stage immunotherapy company specializing in the development of innovative cell therapies for hard-to-treat diseases, today announced that in November, the first patient

Dec. 19, 2024 -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that YORVIPATH® (palopegteriparatide; developed as TransCon PTH) is now commercially available by prescription in the United States. YORVIPATH is a prodrug of parathyroid hormone (PTH [1-34]), administered once-daily, designed to provide continuous exposure to active PTH over the 24-hour dosing period. It is the first and only medicine approved by the U.S. Food & Drug Administration (FDA) for the treatment of hypoparathyroidis

APOLLO study investigating MT-601 in patients with relapsed lymphoma: 78% of patients achieved objective response rates, with 44.4% demonstrating complete response (CR) MT-601 was observed to be well tolerated in all study participants with no observation of immune-effector cell associated neurotoxicity syndrome (ICANS) Dec. 19, 2024 -- Marker Therapeutics, Inc. (Nasdaq: MRKR), a clinical-stage immuno-oncology company focusing on developing next-generation T cell-based immunotherapies for the t

Agreement Reached on Primary Endpoint and Phase 3 Trial Design Oral APX3330 is a Late-Stage Clinical Asset Available for Partnering Dec. 19, 2024 -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage ophthalmic biotechnology company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small-molecule drugs to treat other ophthalmologic disorders, today announced that it has reached agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol A

Dec. 18, 2024 -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced that the European Commission has adopted a positive decision for the designation of mitapivat, an oral, small molecule PK activator, as an orphan medicinal product (OMP) for the treatment of sickle cell disease. Earlier, in November 2020, the U.S. Food and Drug Administration (FDA) also granted orphan drug design

VG801 is the first FDA IND clearance for novel mRNA trans-splicing gene therapy, delivering the full-length functional ABCA4 gene to target the genetic root cause of retinal dystrophies associated with ABCA4 mutations VG801 IND clearance expands ViGeneron’s clinical-stage pipeline to two programs, utilizing its novel gene therapy delivery technology platforms vgAAV capsid and REVeRT Selected by FDA for the Rare Disease Endpoint Advancement (RDEA) Pilot Program Dec. 18, 2024 -- ViGeneron GmbH, a

Data from lowest dose level cohorts demonstrate a favorable safety and efficacy profile Data includes complete results from cohort 1 and initial results from cohort 2 Early exercise of common stock warrants from stockholder generates cash proceeds of approximately $12.7 million Company to host conference call and webcast today at 8:00 a.m. ET / 5:00 a.m. PT Dec. 18, 2024 -- Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, today announced interim data from

EYLEA HD demonstrated non-inferior vision gains with an every 8-week dosing regimen compared to EYLEA® (aflibercept) Injection 2 mg dosed every 4 weeks Safety data remains consistent with the known EYLEA HD and EYLEA safety profiles Supplementary biologics license application planned for submission to the U.S. Food and Drug Administration in the first quarter of 2025 Dec. 17, 2024 -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the primary endpoint was met in the Phase 3 QUASA

Duvakitug positive phase 2b results demonstrate best-in-class potential in ulcerative colitis and Crohn’s disease Primary endpoints met in ulcerative colitis (UC) and Crohn’s disease (CD), the most common forms of inflammatory bowel disease (IBD) Primary endpoint results in UC and CD for high dose represent the highest achieved with any TL1A monoclonal antibody Sanofi and Teva plan to initiate phase 3 development in IBD, pending regulatory discussions Program underscores Sanofi's Immunology lea