Breaking Barriers in Rare Disease Research and Drug Development – Insights from IndoUSrare’s Rare Disease Day Event

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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.

NF2 BioSolutions Is Accelerating Research to Cure a Rare Tumor Disease

Collage of individuals with NF2 with text saying "I Need a Cure" and NF2 BioSolutions Logo
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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

REGENXBIO Logo
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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.

CRB Group Has Hand in Providing Space for Philadelphia’s Life Sciences Ecosystem

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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

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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.

Gain Raises $40 Million in IPO, Funds Will Advance Rare Disease Pipeline

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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.

The Children’s National Hospital Rare Disease Institute and Takeda Partner to Standardize Care for Patients With Rare Diseases

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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 [….]