Rare diseases impact populations across the globe, but despite that wide swath of known patients, the majority of clinical work is conducted in the United States and the European Union. There is a lack of diversity within the patient populations being tested for potential therapies, including cutting-edge gene therapies.
Neurofibromatosis type II, also known as NF2, is a rare disease caused by a mutation in the NF2 gene, whose protein product, “Merlin”, plays a key role in managing cell proliferation. In Merlin’s absence, cells will grow unchecked, forming benign tumors throughout the body.
REGENXBIO and Solid Biosciences Take Aim at Rare Diseases with AAV Gene Therapies via New Pathway Development Consortium
Rare disease specialist REGENXBIO is partnering with Solid Biosciences to advance AAV gene therapies for rare diseases through an accelerated approval pathway with the U.S. Food and Drug Administration.
The Amicus facility encompasses 75,000 square feet of the top three floors of a new building located on Market Street in Philadelphia. The space includes office space, as well as state-of-the-art laboratories. It’s expected to provide space for approximately 200 researchers and drug developers focused exclusively on gene therapies.
The Discovery Labs Signs Foundational Lease with the University of Pennsylvania Gene Therapy Program as Anchor Tenant
The Discovery Labs has signed a foundational lease with the University of Pennsylvania Gene Therapy Program (GTP), which will use Discovery Labs’ suburban campus for a portion of its expanding research operations focused on the development of genetic medicines for rare and orphan diseases, as well as acquired and pandemic infectious diseases, such as COVID-19.
Code Biotherapeutics, a gene therapy company taking aim at rare diseases such as Duchenne Muscular Dystrophy, launched into the Greater Philadelphia gene therapy epicenter with $10 million in seed financing.
Gain Therapeutics made its debut on the Nasdaq Thursday with a $40 million initial public offering. The company will use the funds to advance the development of its pipeline of therapeutics for rare genetic diseases characterized by protein misfolding.
Gain Therapeutics Announces Pricing of Initial Public Offering of Common Stock BETHESDA, Md., March 18, 2021 (GLOBE NEWSWIRE) — Gain Therapeutics, Inc. (“Gain”) today announced the pricing of its initial [….]
The Children’s National Hospital Rare Disease Institute and Takeda Partner to Standardize Care for Patients With Rare Diseases
The Children’s National Hospital Rare Disease Institute and Takeda Partner to Standardize Care for Patients With Rare Diseases – A Five-year Commitment From Takeda Will Help Children’s National Launch a [….]
The National Economic Burden of Rare Disease Study conducted on behalf of the EveryLife Foundation for Rare Diseases took a first-of-its-kind look at the economic burden of rare diseases in the United States for the year 2019.