Bedford biotech Stoke Therapeutics expects gross proceeds from its public offering of shares to reach $125 million. The offering is expected to close on or about April 2, 2024. Stoke is focused on addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines. Using its proprietary approach, the company is developing antisense […]

Orchard Therapeutics has received FDA approval for gene therapy Lenmeldy for the treatment of children with early-onset MLD, a rare and life-threatening inherited disease of the body’s metabolic system caused by a genetic mutation. The one-time, potentially life-saving treatment will carry a list price of $4.25 million, making the treatment the most-expensive ever approved according […]

AstraZeneca is buying Canadian clinical-stage biopharma Fusion Pharmaceuticals in a deal worth up to $2.4 billion. The acquisition of Fusion, a developer of radioconjugates (RCs) for cancer treatment, marks a major step forward in AstraZeneca’s goal to replace traditional cancer treatment regimens like chemotherapy and radiotherapy with more targeted treatments. RCs have emerged as a […]

Governor Healey has announced that she is reauthorizing the Life Sciences Initiative that has made Massachusetts a global leader in life sciences. The initiative proposes reauthorizing the Life Sciences Initiative at $1 billion for 10 years and will be included in the administration’s upcoming economic development bill, called the Mass Leads Act, which will require passage […]

Join the 28th annual Ride to End ALZ New England, a fully supported, one-day fundraising event at the beach offering 14 miles of coastal riding. Whether you’re a dedicated cyclist or a casual rider, the Ride to End ALZ® New England provides a picturesque New England route suitable for all. Every mile you ride will […]

Biogen has announced plans to reprioritize its resources in Alzheimer’s disease, a strategic therapeutic area expected to drive near and long-term growth. The company will continue to advance Leqembi, the first anti-amyloid beta treatment with FDA traditional approval in the United States, and will accelerate development of potential new treatment modalities, including its ASO targeting […]

Vertex has announced positive results from its Phase 3 program in the treatment of moderate-to-severe acute pain using its selective NaV1.8 inhibitor called VX-548. NaV1.8 plays a critical role in pain signaling in the peripheral nervous system. Vertex’s approach is to selectively inhibit NaV1.8 using small molecules with the objective of creating a new class […]

The FDA has approved the Vertex Pharmaceuticals CRISPR/Cas9 gene-edited cell therapy called Casgevy for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. “On the heels of the historic FDA approval of Casgevy for sickle cell disease [SCD], it is exciting to now secure approval for TDT well ahead of the […]

Vertex Pharmaceuticals’ Casgevy is a one-time therapy used to treat people with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The administration of Casgevy requires experience in stem cell transplantation thus Vertex is engaging with experienced hospitals to establish a network of authorized treatment centers throughout the U.S. to offer Casgevy to patients. Casgevy is made specifically for […]

As the first two programs signed under its collaboration with Flagship Pioneering, Novo Nordisk has entered into separate research collaborations with Omega Therapeutics and Cellarity. The companies, both founded by Flagship Pioneering in 2017, are based in Cambridge and Somerville, respectively. The Omega collaboration will leverage its proprietary platform technology to develop a potential new […]