On Friday, the U.S. Food and Drug Administration placed Sarepta Therapeutics Inc.’s SRPT investigational gene therapy clinical trials for limb girdle muscular dystrophy (LGMD) on a clinical hold.The decision stemmed from serious safety concerns, including three patient deaths potentially linked to these products, indicating that study participants faced or would be exposed to an unreasonable and significant risk of illness or injury.The FDA also formally requested Sarepta to voluntarily halt all shipments o ...

Neuralink will do live-changing good for ultimately millions, maybe billions, of people.Imagine your loved one being able to walk again or your parent with dementia being able to recognize their child again.Neuralink (@neuralink):We successfully completed both P8 and P9 this weekend, our first time performing two surgeries in one day. Both participants are recovering well and in great spirits. We are looking forward to supporting them on their Neuralink journey. ...

Core Insights - Mesoblast Ltd's shares surged nearly 38% following the successful commercial launch of its FDA-approved treatment, Ryoncil®, for steroid-refractory acute graft-versus-host disease (SR-aGvHD) in children, with gross revenue of US$13.2 million reported for the first quarter post-launch [1][2] Financial Performance - The company reported a strong financial foundation with US$162 million (A$247 million) in cash at the end of the quarter, positioning it for continued expansion [3] - Mesoblast received US$1.6 million in royalties from the sale of TEMCELL® HS Inj. in Japan through its licensing agreement [3] Regulatory Exclusivity - Ryoncil® received seven years of orphan-drug exclusivity from the FDA for SR-aGvHD in pediatric patients, protecting it from competition in the US market during that period [4] - The company holds biologic exclusivity preventing competitors from referencing Ryoncil®'s biologic license application (BLA) until December 2036, effectively barring biosimilars from entering the market for more than a decade [4] Commercial Launch and Market Coverage - The company has onboarded more than 25 transplant centers, with plans to complete onboarding at 45 priority centers this quarter [6] - Ryoncil® is now insured for more than 250 million US lives through both commercial and government payers, including federal Medicaid, effective from July 1 [6] Future Expansion Plans - Mesoblast is focused on expanding the use of Ryoncil® in adults with SR-aGvHD and has met with the FDA to discuss a pivotal trial for this population [7] - The company is also advancing its second-generation allogeneic MSC therapy, rexlemestrocel-L, for chronic inflammatory diseases, with recent meetings aligning the company and regulators on the pathway to filing for biologics license approval (BLA) [8] Market Reaction - Despite a promising week, investor sentiment has moderated, with shares trading at A$2.32, down 3.73% from Friday highs [9] - The company's long-term prospects remain strong, supported by Ryoncil®'s commercial launch and a robust cash position as it enters the next growth phase [9][10]

ATLANTA, July 21, 2025 (GLOBE NEWSWIRE) -- A shareholder class action lawsuit has been filed against PepGen Inc. (“PepGen” or the “Company”) (NASDAQ: PEPG). The lawsuit alleges that Defendants made materially false and/or misleading statements and/or failed to disclose material adverse information about PepGen’s business, operations, and prospects, including allegations that: (i) PGN-EDO51 was less effective and safe than Defendants had led investors to believe; (ii) the CONNECT2 study was deficient for pur ...

CARBIOS has signed a multi-year commercial agreement with Indorama Ventures, the world leader in polyester production. CARBIOS will supply recycled monomers that will be transformed into filaments by Indorama Ventures, intended for use in tire reinforcement for Michelin.This new commercial agreement highlights the interest of a new market—industrial fabrics—for high-quality r-PET derived from enzymatic recycling. Clermont-Ferrand (France), 21 July 2025 (17h45 CEST) – CARBIOS (Euronext Growth Paris: ALCRB) ...

Investing in biotechnology stocks is similar to the drug development process itself. That is, investors frequently experience long periods of nothing much happening, interrupted by periods of sharp movement. This is particularly true of small-cap biotech stocks. In many cases, these are unprofitable companies with little to no revenue. However, these are also the companies that can provide the highest return for risk-tolerant investors. The risk with these stocks is that the company can deliver poor clinica ...

ATLANTA, July 21, 2025 (GLOBE NEWSWIRE) -- A shareholder class action lawsuit has been filed Rocket Pharmaceuticals, Inc. (“Rocket Pharmaceuticals” or the “Company”) (NASDAQ: RCKT). The lawsuit alleges that Defendants made materially false and/or misleading statements and/or failed to adequately disclose that Rocket Pharmaceuticals knew Serious Adverse Events (SAEs), including death of participants enrolled in the study, were a risk. If you purchased shares of Rocket Pharmaceuticals between February 27, 202 ...

Taking full advantage of the stock market and investing with confidence are common goals for new and old investors, and Zacks Premium offers many different ways to do both.The popular research service can help you become a smarter, more self-assured investor, giving you access to daily updates of the Zacks Rank and Zacks Industry Rank, the Zacks #1 Rank List, Equity Research reports, and Premium stock screens.It also includes access to the Zacks Style Scores. What are the Zacks Style Scores? Developed along ...

Key Takeaways VKTX to report Q2 results on July 23 with no revenue expected and a projected EPS loss of $0.44. Investor focus centers on VK2735's VANQUISH trial enrolment and oral dosing study progress. Updates are also expected on IND filing for DACRA obesity candidate and updates on NASH and X-ALD programs.We expect investors to focus on updates related to Viking Therapeutics’ (VKTX) pipeline when it reports second-quarter 2025 results on July 23, after market close. In the last reported quarter, the co ...

About this content About Angela Harmantas Angela Harmantas is an Editor at Proactive. She has over 15 years of experience covering the equity markets in North America, with a particular focus on junior resource stocks. Angela has reported from numerous countries around the world, including Canada, the US, Australia, Brazil, Ghana, and South Africa for leading trade publications. Previously, she worked in investor relations and led the foreign direct investment program in Canada for the Swedish government ...