A foreign drugmaker has thrown a gauntlet to the Korean rare disease treatment market. Italy-based Recordati is the company. On Rare Disease Day Feb. 28, the company launched Recordati Korea, its Korean offshoot and Asia-Pacific headquarters, declaring its official advance to the Korean and regional market. Unlike most other pharma...
treatment News
EMERYVILLE, Calif. — 4D Molecular Therapeutics (Nasdaq: FDMT), a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases, today announced an update on its regulatory interactions and development path for 4D-710, an aerosolized genetic medicine for the treatment of CF...
EMERYVILLE, Calif. — 4D Molecular Therapeutics, a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases in ophthalmology and pulmonology, today announced that the U.S. Food and Drug Administration (FDA) has granted the RMAT designation for the investigational genetic medicine...
EMERYVILLE, Calif. — 4D Molecular Therapeutics, a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for the Company’s product candidate aerosolized 4D-710...
Washington DC – As of Dec. 22, the FDA had approved at least 88 novel treatments in 2023, for indications ranging from breast and colorectal cancer to Duchenne muscular dystrophy to sickle cell disease. Rare diseases such as Rett syndrome and Friedreich’s ataxia saw their first approvals. Some decisions, like Sarepta’s...
Washington, DC – 2023 was a banner year for cell and gene therapy, with seven FDA approvals, according to the Alliance for Regenerative Medicine. And the pace doesn’t appear to be slowing at all as the second quarter of this year gets underway. The advocacy group is anticipating an even...
WASHINGTON, DC — 60 Degrees Pharmaceuticals, Inc. , a pharmaceutical company focused on developing new medicines for infectious diseases, announced today the approval of an Investigational Review Board (IRB) sanctioned Phase IIA clinical study. The study aims to investigate the efficacy and safety of the ARAKODA® regimen of tafenoquine in...
SAN FRANCISCO, Calif. — 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardiometabolic diseases, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) status to pegozafermin in patients with MASH. The...
SAN DIEGO, Calif. – SYNC-T, an investigational therapy that combines a device-induced vaccination at the tumor site with intratumoral infusion of a multitarget biologic drug led to numerous clinical responses in patients with metastatic castration-resistant prostate cancer (mCRPC), according to results reported at the American Association for Cancer Research (AACR)...
Toruń, Poland – Boys suffering from Barth syndrome, a genetic determined disease with a very high mortality rate, finally have hope for a cure. What has contributed to this finding is research of Dr. habil Karolina Mikulska-Ruminska, Nicolaus Copernicus University (Torun, Poland) CU Professor from the Institute of Physics. The...