Tag: cell types

CELEBRATION, Fla., July 28, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare diseases, announced the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the evaluation of arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC). NPC is an ultra-rare, neurological disease caused by genetic mutations that result in lipid accumulation in cells, leading to visceral, neurological, and psychiatric symptoms. Arimoclomol is the only treatment shown to directly target the underlying pathology of NPC by increasing gene expression for improved lipid clearance. Arimoclomol for the treatment of NPC has been designated as an Orphan Medicinal Product by the EMA. Arimoclomol is marketed in the U.S. under the brand name MIPLYFFA®.

Groundbreaking First-in-Human Study Establishes Potential to Treat Type 1 Diabetes by Transplanting Insulin-Secreting Cells Without Immunosuppression

Media Release

COPENHAGEN, Denmark; June 15, 2025

SAN FRANCISCO, June 13, 2025 (GLOBE NEWSWIRE) -- Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted protein degradation medicines, the next frontier in innovative drug design aimed at improving treatment options for patients with cancer and inflammatory diseases, today announced that on June 10, 2025, the company granted inducement awards to seventeen new employees. The grants were made pursuant to Nurix’s 2024 Equity Inducement Plan as an inducement material to the employees’ acceptance of employment with Nurix and were approved by the Compensation Committee of Nurix’s Board of Directors in accordance with Nasdaq Listing Rule 5635(c)(4).

MaxCyte and Ori Biotech successfully integrate their ExPERT™ and IRO® platforms to improve the yield of gene-edited T cells and shorten manufacturing timelines