Shares of Sera Therapeutics up by about 36% in the pre-market. The company says that it will resume shipping Alvdus, that's a muscular disorder gene therapy to patients who can walk. Last week, the FDA recommended a pause in shipments because of serious safety concerns after three deaths of patients who were on this who were on serene therapies. This past weekend, there were reports of a death of another child on Avdus, but the death, the FDA now says, was unrelated to the gene therapy. In a statement, Surr ...

HC Wainwright assumed coverage on Ultragenyx Pharmaceuticals, Inc. RARE on Monday, representing an attractive opportunity at current levels.Analyst Raghuram Selvaraju notes that Ultragenyx stock is down 35% year to date, compared to a 2.7% decline in the benchmark XBI index.“Intriguing buying opportunity in one of biopharma’s most diversified rare disease-focused companies,” the analyst said.HC Wainwright assumed coverage at a Buy rating with a price target of $80.The analyst says Ultragenyx shares have pri ...

The FDA won’t sign off on Sarepta bringing its controversial gene therapy back to the market until the company can persuade US regulators that it won’t cause more deaths, according to an official familiar with the situation https://t.co/7gpkWSFdzG ...

There were warnings to the FDA about Sarepta before US regulators asked the company to halt shipments of its gene therapy https://t.co/ZZC0xgoUpS ...

uniQure to host earnings call on Tuesday, July 29, 2025 at 8:30 a.m. ETLEXINGTON, Mass. and AMSTERDAM, July 22, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs will report second quarter 2025 financial results before market open on Tuesday, July 29, 2025. Management will then host a conference call at 8:30 a.m. ET. The event will be webcast under the Events & Presentations section of uniQure’s webs ...

Children’s Hospital Los Angeles paused the use of Sarepta’s gene therapy for Duchenne muscular dystrophy in the wake of rising safety concerns about liver toxicity, adding to mounting challenges for the drugmaker https://t.co/HBmRIgaNhA ...

Children’s Hospital Los Angeles paused the use of Sarepta’s gene therapy for Duchenne muscular dystrophy in the wake of rising safety concerns about liver toxicity, adding to mounting challenges for the drugmaker https://t.co/WOhmDsWdFS ...

Welcome back to Squawk on the Street. Healthc care you might know the worst performing sector of the year so far. Just one of just three still in the red since January after we got weakness in one part of the market.The health insurers names like Centine, UNH, Molina, Eleance all have had bad news last couple of weeks. Down double digits on the year and more than cut in half off of their 52- week highs. It's been a non-stop stream of bad news from almost all of those names.Yeah, brutal. Also pharma as well. ...

MALVERN, Pa., July 21, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced updates to its Retina Scientific Advisory Board (SAB) and Executive Leadership Team to enhance external guidance from key opinion leaders and strengthen the Company’s internal expertise in critical functions and as it pursues its goal of three BLAs in the next three years. “As our novel modifier gene therapy programs ...

Company Overview - Ocugen, Inc. is a pioneering biotechnology leader focused on gene therapies for blindness diseases, utilizing a breakthrough modifier gene therapy platform to address significant unmet medical needs for large patient populations [3] Upcoming Events - The company will host a conference call and live webcast to discuss its second quarter 2025 financial results and provide a business update on August 1, 2025, at 8:30 a.m. ET [1] - A pre-market earnings announcement will be issued on the same day, with dial-in numbers provided for U.S. and international callers [2] Business Focus - Ocugen's modifier gene therapies aim to address complex diseases potentially caused by imbalances in multiple gene networks, with current programs in development for inherited retinal diseases and blindness diseases affecting millions globally, including retinitis pigmentosa, Stargardt disease, and geographic atrophy [3]