Tag: gene therapy

January 20, 2022

Scientists from the University of Virginia (UVA) School of Medicine describe in a study involving mouse models how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, and potentially prolong survival for people with the […]

January 20, 2022

The Center for Breakthrough Medicines (CBM) and the University of Pennsylvania’s Gene Therapy Program (GTP) have signed a landmark collaboration agreement, to combine Penn’s knowledge and expertise in the development of Gene therapies with CBM’s manufacturing capacity and singular focus […]

January 18, 2022

Ori Biotech, which is on the cusp of launching an innovative process for manufacturing cell and gene therapies (CGTs), tallied more than $100 million in its latest funding round. The company will use the money to add key personnel in […]

January 10, 2022

Last summer, Mikael Dolsten, head of research and development at Pfizer and the company’s top scientist, was thinking of new uses for the messenger RNA technology the pharmaceutical giant had developed so effectively for its coronavirus vaccine. The drugmaker already […]

January 4, 2022

First approved in late 2016, Spinraza has become one of the most important medicines in Biogen’s arsenal, accounting for about a fifth of the company’s total product revenue. Recently, though, the Spinraza franchise hasn’t fared as well due to competitive […]

January 4, 2022

Fabry is one of several dozen diseases known collectively as lysosomal storage disorders, all caused by genetic breakdowns in how the body’s cells process complex fats or sugars. Treatment, when it’s available, usually involves infusions of the digestive proteins that […]

December 15, 2021

The FDA has kicked off a priority review of Opzelura (ruxolitinib), a JAK1/JAK2 inhibitor it approved to treat atopic dermatitis in September, and aims to decide on the vitiligo indication by 18 April next year. If approved, Opzelura would provide […]

December 8, 2021

Sotio Biotech, a clinical stage immuno-oncology company owned by PPF Group, has entered into a clinical trial collaboration and Supply Agreement with MSD, a tradename of Merck & Co., Inc., Kenilworth, NJ., through its subsidiaries, to evaluate the combination of […]

December 6, 2021

Astellas Pharma and Dyno Therapeutics have announced an option and license agreement to develop next-generation adeno-associated virus (AAV) vectors for gene therapy directed to skeletal and cardiac muscle using Dyno’s CapsidMap™ platform. Dyno’s CapsidMap platform represents a new approach applying […]

December 2, 2021

The Japanese drugmaker is paying $18 million upfront to exercise an option on the AAV platform – which my help avoid safety issues that have emerged with other AAV-based gene therapies – with milestone and royalty payments that could exceed […]

November 19, 2021

The race between Moderna and Pfizer to gain FDA nods for their COVID-19 vaccines as boosters in all adults has ended in a dead heat. On Friday morning, the FDA simultaneously expanded emergency use authorizations for both mRNA vaccines to […]

November 18, 2021

Gene therapy shows promise in reducing, and even halting, potentially life-threatening bleeding events in people with hemophilia, researchers report. Hemophilia A is the most common inherited bleeding disorder, affecting one in 5,000 males worldwide. It’s caused by a missing coagulation […]