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News
RARE Stock Gains on FDA Nod for Rare Disease Gene Therapy Genglycos
3+ week, 2+ day ago (276+ words) Shares of Ultragenyx Pharmaceutical RARE are rising in premarket trading today following the FDA's accelerated approval of DTX-401, an AAV8 gene therapy for glycogen storage disease type Ia (GSDIa). Year to date, shares of Ultragenyx have gained 14.1% compared with the industry's…...
EDIT Q2 Earnings Beat Estimates, Revenues Rise, Pipeline in Focus
1+ mon, 6+ day ago (771+ words) Editas Medicine EDIT incurred a loss of 15 cents per share in the second quarter of 2026, narrower than the Zacks Consensus Estimate of a loss of 30 cents. The company had reported a loss of 63 cents per share in the year-ago quarter....
BEAM Stock Gains 5% as FDA Clears BEAM-304 IND for Phenylketonuria
2+ mon, 3+ week ago (398+ words) Shares of Beam Therapeutics BEAM rose 5% on Thursday after the FDA cleared the investigational new drug (IND) application for BEAM-304, the company’s investigational gene-editing therapy for the treatment of phenylketonuria (PKU). The clearance marks an important milestone for Beam Therapeutics…...
Vedanta Biosciences Showcases Innovative Work on its Microbiome-based Therapeutics at the European Society of Clinical Microbiology and Infectious Diseases (ESCMID) 2026 Global Congress
5+ mon, 3+ day ago (225+ words) VE303 for Recurrent CDI Prevention VE303 is an eight-strain bacterial consortium that demonstrated a greater than 80% reduction in the odds of a CDI recurrence when compared with placebo in a Phase 2 clinical study. Title: Impact of a Defined Bacterial Consortium on Host…...
Rocket Pharmaceuticals (RCKT) Is Down 8.9% After FDA Accelerated Approval Of KRESLADI Gene Therapy
5+ mon, 2+ week ago (269+ words) The approval, which is supported by increased neutrophil CD18 and CD11a expression and includes a Rare Pediatric Disease Priority Review Voucher, underscores a significant regulatory milestone in treating an ultra-rare, life-threatening pediatric immunodeficiency. We will now examine how the accelerated approval of…...
Ocugen Inc. (OCGN): One of the Popular Penny Stocks on Robinhood
5+ mon, 2+ week ago (281+ words) Ocugen Inc. (NASDAQ:OCGN) is one of the popular penny stocks on Robinhood to buy. On March 2, Ocugen, Inc. (NASDAQ:OCGN) announced the completion of patient enrollment for its Phase 3 liMeliGhT clinical trial. The trial is for evaluating OCU400, an investigational…...
Ocugen Index Addition Highlights Rising Attention And Ongoing Gene Therapy Risks
5+ mon, 2+ week ago (469+ words) Ocugen, Inc. (NasdaqCM:OCGN) has been added to the S&P Biotechnology Select Industry Index. This index inclusion marks a new milestone for the company within the biotechnology sector. Ocugen focuses on biotechnology, with programs that seek to address eye…...
Apellis Pharmaceuticals Details SYFOVRE, EMPAVELI Growth Plans and FcRn Gene-Editing Push at TD Cowen Conf.
6+ mon, 1+ week ago (859+ words) Apellis laid out a three‑pillar plan focused on a “durable” SYFOVRE franchise in geographic atrophy, an expanding EMPAVELI launch in nephrology, and a planned second‑half clinic entry for a Beam collaboration on an FcRn gene‑editing “once‑and…...
Intellia Therapeutics to Hold Conference Call to Discuss Fourth Quarter and Full-Year 2025 Financial Results and Business Updates
6+ mon, 3+ week ago (244+ words) CAMBRIDGE, Mass., Feb. 19, 2026 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (Nasdaq: NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that the company will host a conference call on February 26, 2026, at 8 a.m. ET to discuss its…...
Bionano Announces Publication from Sanford Burnham Prebys Medical Discovery Institute Describing use of OGM to Detect Genomic Alterations Introduced by Gene Editing Technologies
7+ mon, 1+ week ago (578+ words) SAN DIEGO, Feb. 04, 2026 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (Nasdaq: BNGO) announced a peer-reviewed study published in Molecular Therapy - Methods & Clinical Development, describing the application of optical genome mapping (OGM) to detect genomic alterations introduced by different gene editing technologies, including…...