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 [….]

Jeeva Informatics Hosts Rare Disease Panel on Decentralized, Patient-Focused Clinical Trials

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In recognition of Rare Disease Day 2021, Jeeva Informatics (Jeeva) brought together a panel of experts to discuss the importance of transforming the clinical trial process for rare diseases. The panel included Annie Kennedy, Chief of Policy and Advocacy, EveryLife Foundation for Rare Diseases; Thomas Lester, Former Executive Director, Audentes Therapeutics; Scott Schliebner, Senior Vice President, Scientific Affairs & Therapeutic Expertise, PRA Health Sciences; and Dr. Eric Sid, Program Director, National Center for Advancing Translational Science, Office of Rare Disease Research, (NCATS)/NIH.

Vigene Biosciences Sees Growth Through a Commitment to Gene Therapy and Rare Diseases

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About a year ago, BioBuzz attended the grand opening of Vigene Biosciences (Vigene) brand new headquarters in Rockville, Maryland. It was a fantastic day for Vigene, the BioHealth Capital Region (BHCR), and for the patients and families of people suffering from cancer and rare genetic diseases.

10 Philadelphia Biotech Companies Targeting Rare Diseases

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Philadelphia, the birthplace of cell and gene therapy, is home to a number of companies focused on developing multiple treatment options for rare diseases, those that affect less than 200,000 people in the United States.

Rare Disease Challenge, REGENXBIO Tackles Duchenne Muscular Dystrophy

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Duchenne Muscular Dystrophy is a severe degenerative muscle disease that impacts about one in 5,000 boys per year across the globe. The disease causes a progressive loss of muscle strength attributable to a loss of a protein called dystrophin, which normally protects muscle fibers from breaking down.