Cartesian, BD and REGENXBIO’s Ken Mills Take Home Top Honors at MTC’s Industry Awards Celebration

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The Maryland Tech Council, the largest technology and life science trade association in the state, announced the winners of its 2022 Industry Awards Thursday at the Bethesda North Marriott Hotel & Conference Center. The event, which honored companies, executives, students, and educators in 15 categories, marked the first in-person gathering for the Industry Awards Celebration since 2019. 

The Promise of Gene Therapy Offers More Hope for Rare Disease Cures in 2022

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While the field of gene therapy is still relatively new, it’s moving at a breakneck pace. In fact, 1,745 gene therapies are currently in development around the world! Technological advancements in gene editing, modulation, and replacement technologies have helped to advance this new breed of therapies, raising hope for cures for many of these rare diseases in the near future.

REGENXBIO Presents Positive Initial Data from Phase I/II Trial of RGX-111 for the Treatment of Severe MPS I at 18th Annual WORLDSymposium™ 2022

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REGENXBIO Inc. (Nasdaq: RGNX) today announced positive interim data are being presented at the 18th Annual WORLDSymposium™ from five patients in the ongoing Phase I/II trial and one patient from a single-patient Investigational New Drug (IND) application of RGX-111 for the treatment of severe Mucopolysaccharidosis Type I (MPS I).  

REGENXBIO Starts 2022 by Moving DMD Gene Therapy into the Clinic and Advancing AbbVie Partnership

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Rare disease-focused REGENXBIO hit the ground running in 2022 with its developmental programs for Duchenne Muscular Dystrophy and wet age-related macular degeneration. The Maryland-based company received clearance from the U.S. Food and Drug Administration to initiate a first-in-human study of its gene therapy for the rare form of muscular dystrophy.

REGENXBIO Announces FDA Clearance of IND for Clinical Trial of RGX-202, a Novel Gene Therapy Candidate for Duchenne Muscular Dystrophy

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REGENXBIO Inc. (Nasdaq: RGNX) today announced the clearance of its Investigational New Drug (IND) application by the United States Food and Drug Administration (FDA) to evaluate RGX-202, a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy (Duchenne) in a first-in-human clinical trial. RGX-202 is designed to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin. RGX-202 uses REGENXBIO’s proprietary NAV® AAV8 vector. REGENXBIO plans to initiate the trial in the first half of 2022.

REGENXBIO Closes $370M+ Gene Therapy Eye Care Collaboration Agreement with AbbVie for Wet AMD and Diabetic Retinopathy

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REGENXBIO Inc. (Nasdaq: RGNX) today announced the closing of its Collaboration and License Agreement with AbbVie to develop and commercialize RGX-314, a potential one-time gene therapy for the treatment of wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR) and other chronic retinal diseases.

REGENXBIO and Solid Biosciences Take Aim at Rare Diseases with AAV Gene Therapies via New Pathway Development Consortium

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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.

A Unique Talent Pool is Propelling Philly to become a Top Biotech Cluster

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As some are now calling it, the greater Philadelphia region, or ‘Cellecon Valley, is garnering a lot of attention these days as a rapidly emerging Biopharma cluster, earning a defining spot among the top 10 clusters in the US.