Sanofi recently provided a comprehensive update regarding its “Play to Win” strategy. This strategy continues to focus on the critical goals of executing transformative medicine and vaccines launches, driving agile and efficient resource deployment and enhancing R&D productivity. To that end, Sanofi announced plans to increase its R&D investments to fully realize its pipeline potential, […]

Biogen has reported new Phase 1b clinical data from the study of BIIB080, an investigational antisense oligonucleotide (ASO) therapy targeting tau, in mild Alzheimer’s disease (AD). Antisense therapies are designed to seek out, bind to and destroy a mRNA in a highly specific manner, so that the amount of disease-causing protein is dramatically decreased. The data […]

Verve Therapeutics has announced the lifting of the clinical hold and clearance of its Investigational New Drug (IND) application by the FDA to conduct a clinical trial evaluating Verve-101 for the treatment of heterozygous familial hypercholesterolemia (HeFH). Verve-101 is an investigational, in vivo base editing medicine designed to be a single-course treatment that inactivates the […]

Beam Therapeutics has announced portfolio priorities and plans to streamline its business operations including a reduction in headcount of approximately 100 employees, about 20% of its current workforce, which is anticipated to be completed in the fourth quarter of 2023. In related news, Beam also reported it expects to receive $250 million from Eli Lilly […]

The Advanced Research Projects Agency for Health (ARPA-H), an agency within the Department of Health and Human Services (HHS), is taking action to improve the nation’s ability to conduct clinical trials safely, quickly, and equitably and to improve clinical trial access for people in communities across the country as the first initiative within the recently […]

A group of international scientists have mapped the genetic, cellular, and structural makeup of the human brain and the nonhuman primate brain. This understanding of brain structure allows for a deeper knowledge of the cellular basis of brain function and dysfunction, helping pave the way for a new generation of precision therapeutics for people with […]

Three Vertex researchers have been awarded the 2024 Breakthrough Prize in Life Sciences “for developing life-transforming drug combinations that repair the defective chloride channel protein in patients with cystic fibrosis.” The award celebrates the work of Paul Negulescu, Ph.D., Fredrick Van Goor, Ph.D., and Sabine Hadida, Ph.D., who have worked together to lead cystic fibrosis […]

Sanofi and Teva Pharmaceuticals have entered into a collaboration to co-develop and co-commercialize TEV’574, currently in Phase 2b clinical trials for the treatment of ulcerative colitis and Crohn’s disease, two types of inflammatory bowel disease (IBD) characterized by chronic inflammation of the gastrointestinal tract. An estimated 10 million people worldwide live with IBD. According to […]

RNAi therapeutics company Alnylam Pharmaceuticals welcomed the positive outcome of the FDA advisory committee meeting. On Wednesday the Cardiovascular and Renal Drugs Advisory Committee (CRDAC) voted 9:3 that the benefits of RNAi therapeutic patisiran outweigh its risks for the treatment of the cardiomyopathy of ATTR amyloidosis. The CRDAC provides the FDA with independent, expert advice and recommendations […]

Biopharma BioLineRx, headquartered in Israel with a Waltham presence, has announced FDA approval for Aphexda, the company’s first approved therapeutic. The drug helps physicians harvest stem cells for the treatment of multiple myeloma, the second most-common hematologic malignancy. Autologous stem cell transplantation (ASCT) is part of the standard of care treatment for multiple myeloma and […]