Acquisition bolsters Eton’s commercial pediatric endocrinology portfolio Product is now available through AnovoRx, a specialty pharmacy dedicated to serving patients with rare and chronic conditions Dec. 20, 2024 -- Eton Pharmaceuticals, Inc (“Eton” or “the Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced that it has completed its previously announced asset purchase of Increlex® (mecasermin inj

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

Dec. 19, 2024 -- Certa Therapeutics (Certa), a biotechnology company developing innovative precision therapies for patients with fibrotic diseases, today announces the acquisition of OccuRx, a Melbourne-based biotechnology company with highly complementary assets, resources and expertise, and a shared focus on targeting GPR68, a master switch of fibrosis. The acquisition combines the assets and capabilities of two companies founded by a leader in the GPR68 field, Professor Darren Kelly, consoli

Dec. 19, 2024 -- Arbor Biotechnologies™, a biotechnology company discovering and developing the next generation of genetic medicines, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for ABO-101, a novel gene editing therapeutic designed to address primary hyperoxaluria type 1 (PH1). The redePHine Phase 1/2 study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficac

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

Partnership leverages proprietary PHICS™ technology to bring together a kinase and target of interest to modulate protein function, in this case an undisclosed cardiometabolic target Total potential deal value up to $186M in upfront, development and commercial milestone payments, in addition to R&D funding and tiered royalty payments on commercial sales Dec. 18, 2024 -- Photys Therapeutics, Inc., a proximity-based therapeutics company, today announced a multi-year collaboration and license agre

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