Tag: gene

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®.

SAN DIEGO, July 25, 2025 (GLOBE NEWSWIRE) -- Calidi Biotherapeutics, Inc. (“Calidi” or the “Company”) (NYSE American: CLDI), a clinical-stage biotechnology company pioneering the development of targeted therapies with the potential to deliver genetic medicines to distal sites of disease, announced today that it will effect a reverse stock split of its outstanding shares of common stock at a ratio of 1-for-12 (the “Reverse Stock Split”). The Company’s common stock is expected to commence trading on a split-adjusted basis when the NYSE American market opens on August 5, 2025 under the existing symbol of “CLDI.” The new CUSIP number for the Company’s common stock following the Reverse Stock Split will be 320703 408.

A Natural Supplement Trend in 2025, Menovelle Helps Women Support Hormonal Balance and Metabolic Energy Without Hormone Therapy or Stimulants

VYJUVEK approved for the treatment of DEB from birth with flexible administration options including home dosing and the option for administration by the patient or their family

[220+ Pages Latest Report] According to a market research study published by Custom Market Insights, the demand analysis of Global CRISPR Gene Editing Market size & share revenue was valued at approximately USD 3.65 Billion in 2024 and is expected to reach USD 4.10 Billion in 2025 and is expected to reach around USD 12.6 Billion by 2034, at a CAGR of 13.01% between 2025 and 2034. The key market players listed in the report with their sales, revenues and strategies are CRISPR Therapeutics AG, Agilent Technologies Inc., Thermo Fisher Scientific Inc., GeneCopoeia Inc., Synthego Corporation, System Biosciences LLC, ToolGen Inc., Rockland Immunochemicals Inc., Horizon Discovery Group PLC, Abcam Inc., Applied StemCell Inc., Cellecta Inc and others.

MN-166 COMBAT ALS trial down to single digit required enrollment while MN-001 hypertriglyceridemia trial in type 2 diabetes patients requires final two patients

SAN FRANCISCO, July 23, 2025 (GLOBE NEWSWIRE) -- When, on May 27, 2025, Rocket Pharmaceuticals (NASDAQ: RCKT) revealed that the FDA put a clinical hold on its Phase 2 pivotal trial for RP-A501, an experimental therapy intended to treat patients with a rare gene disorder called Danon disease, investors in the company’s shares saw their investment crash over 60% that day.