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Slate Medicines and Fulcrum Therapeutics have reached a reverse merger agreement. Fulcrum is seeking strategic options after terminating its sickle cell disease drug pociredir, while Slate focuses on developing new migraine treatments. After the merger, Fulcrum shareholders will hold 5% of the shares, and Slate CEO Gregory Oakes will lead the new company. The transaction is expected to close by the end of 2026.
PTC Therapeutics has agreed to acquire Sangamo Therapeutics' experimental gene therapy ST-920 for Fabry disease for up to $211 million. The deal, part of Sangamo's bankruptcy proceedings, gives PTC an advanced asset already in a rolling FDA submission. ST-920 delivers a functional copy of the GLA gene in a one-time infusion and has shown promising results. PTC plans to complete the application in Q4 and potentially launch the therapy in 2027. Analysts view the acquisition favorably due to the ongoing regulatory process and synergies with PTC's rare disease business, though risks remain around FDA's stance on gene therapies.
U.S. President Trump signed an executive order on Monday named the "Golden Standard Childhood Vaccine Recommendations," attempting to make major changes to the U.S. childhood immunization program that has been in place for decades. The order plans to reduce the number of universally recommended vaccines from 17 to 11 and requires the measles-mumps-rubella (MMR) vaccine to be split into three separate injections. This move has been criticized by professional organizations such as the American College of Physicians for bypassing existing scientific review procedures. Additionally, the legal effect and scope of implementation of the executive order remain unclear, as vaccine mandate policies are primarily determined by individual states.
Jazz Pharmaceuticals announced it will acquire Actio Biosciences for an upfront payment of $820 million plus up to $500 million in milestone payments. The latter focuses on treatments for genetic epilepsy. The transaction is expected to close within the year, and Actio will simultaneously spin off an early-stage asset to form a new company.
Gene editing company Scribe Therapeutics has gone public in the first IPO for a gene editing company in more than two years. CEO Benjamin Oakes speaks with BioPharma Dive about convincing investors to back Scribe's scalable CRISPR-based medicines, its first program that switches off a gene involved in high cholesterol rather than permanently altering it, and the human behavior challenges in cardiovascular treatment. Oakes explains the 'MAYA' design principle, why Scribe raised less than other recent IPOs, and how the company aims to make preventive genetic medicine accessible to millions.
Gene-editing company Scribe Therapeutics, based at a former naval shipyard in California, is attempting to bring CRISPR technology to the masses. In an interview, CEO Benjamin Oakes explains its epigenetic silencing strategy, views on the competitive landscape, and why it opted for an IPO a year before clinical data is released.
The U.S. FDA approved Replimune's advanced melanoma therapy Tudriqev (formerly RP1) on Thursday, after two prior rejections. This is an accelerated approval requiring a subsequent Phase III trial to confirm efficacy. Data showed tumor responses in about a quarter of patients, with a median duration exceeding 14 months.
Tarsus Pharmaceuticals announced on Thursday the acquisition of Alkeus Pharmaceuticals, with a total transaction value of up to $800 million, including $270 million in cash and $180 million in stock, plus milestone payments of up to $350 million. Alkeus's core asset is gildeuretinol, an experimental drug for treating Stargardt disease, which has entered Phase III clinical trials. This acquisition marks the end of Alkeus, a startup long operated by an individual.
The U.S. Food and Drug Administration (FDA) approved Takeda Pharmaceutical's Orzeyful (generic name: oveporexton) for the treatment of type 1 narcolepsy, making it the first orexin receptor agonist targeting the root cause of the disease. Analysts predict its peak annual sales could reach $2 billion. Companies such as Eisai, Eli Lilly, and Alkermes are also racing to develop similar drugs, indicating significant market potential.
The U.S. Senate confirmed Erica Schwartz as CDC director on Wednesday, ending the agency's nearly year-long period without a permanent leader. During her confirmation hearing, Schwartz pledged to 'never betray science,' but some Democratic lawmakers expressed doubts about her ability to withstand political interference. The vote largely followed party lines, with the only Democrat voting in favor being Senator Tim Kaine of Virginia.