Researchers have identified a new ALS gene called KIF5A. This important gene discovery, funded by The ALS Association through Ice Bucket Challenge donations, will help researchers understand ALS disease pathways and represents a potential new ALS therapeutic target. This was a large collaborative effort involving over 250 researchers, led by John Landers, Ph.D., Professor of […]

Biogen has announced an agreement to acquire from Pfizer a first-in-class, Phase 2b ready AMPA receptor potentiator for cognitive impairment associated with schizophrenia (CIAS). AMPA receptors mediate fast excitatory synaptic transmission in the central nervous system, a process which can be disrupted in a number of neurological and psychiatric diseases, including schizophrenia. The purchase will […]

Cambridge-based Rubius Therapeutics, a biotech company pioneering a new class of cellular therapies, today announced the successful completion of an oversubscribed $100 million crossover financing. In just under nine months, the company has now raised over $220 million. Rubius has developed the technology to grow, genetically engineer and mature long circulating Red Cell Therapeutics (RCT) […]

Vertex Pharmaceuticals has announced that the FDA approved Symdeko for treating the underlying cause of cystic fibrosis (CF) in certain patients ages 12 and older. Symdeko is Vertex’s third medicine approved to treat the underlying cause of CF. “Today is an exciting day for the CF community. The approval of Symdeko, our third disease-modifying CF […]

AstraZeneca and MedImmune, its global biologics research and development arm, today announced that the FDA has approved Imfinzi for the treatment of certain patients with non-small cell lung cancer (NSCLC), specifically those with unresectable Stage III NSCLC whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy. Stage III (locally advanced) NSCLC is […]

Sanofi’s acquisition of Ablynx in a deal valued at $4.8 billion complements the earlier acquisition of Biogen spin-off  Bioverativ for $11.6 billion. Both strengthen Sanofi’s position in rare blood disorders. Launched in 2017 following separation from Biogen, Bioverativ is focused on therapies for hemophilia and other rare blood disorders. With approximately $10 billion in annual […]

Leading RNAi therapeutics company Alnylam Pharmaceuticals recently announced its corporate goals for 2018. The goals focus on clinical and regulatory progress against its five mid- to late-stage programs, including patisiran, which is advancing toward potential global regulatory approvals and commercialization. “In the year ahead, we look forward to the potential global commercial launch of patisiran, […]

The FDA has expanded the approved use of AstraZeneca’s Lynparza to include the treatment of patients with certain types of breast cancer that have spread and whose tumors have a specific inherited genetic mutation. This makes Lynparza the first PARP inhibitor to be approved for breast cancer; and the first time any drug has been […]

[The following post has been excerpted from comments by FDA Commissioner Scott Gottlieb as published by the FDA on its website on January 03, 2018.] The FDA has announced additional steps to encourage generic competition as part of our continued implementation of the Drug Competition Action Plan. This plan has three main components: reducing gaming […]

The FDA has approved Luxturna, an innovative one-time gene therapy treatment from Spark Therapeutics used to improve vision in patients with vision loss due to inherited retinal diseases (IRD). As the first adeno-associated viral (AAV) vector product approved for clinical use in the United States, Luxturna represents a historic landmark, not only in the field […]