Pharmaceutical Technology

JUNE 19, 2023

Although only a small number of gene therapies have reached the market thus far, the industry is poised to grow quickly over the next few years. According to GlobalData’s clinical trials database, there are currently 1,231 planned and ongoing trials for gene therapies and gene-modified cell therapies alone.

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Pharmaceutical Technology

JULY 29, 2022

However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. Their platform identifies which regRNAs control the specific gene of interest.

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The Pharma Data

DECEMBER 13, 2020

.–( BUSINESS WIRE )– EdiGene, Inc. which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., as Head of Business Development. Both will report to Dr. Dong Wei, CEO of EdiGene.

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The Pharma Data

DECEMBER 17, 2020

Most recently, Cadavid served as senior vice president and Head of Clinical Development at Fulcrum Therapeutics where he led the development of multiple small molecules for the treatment of genetically defined rare diseases. Prior to SpringWorks, Smith was the executive vice president of Gene Therapy at Bamboo Therapeutics.

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XTalks

MAY 17, 2024

Prior to joining Calliditas Therapeutics as CMO, he held roles at GlaxoSmithKline (GSK) and Takeda, focusing on clinical and business development in rare diseases. I’ve had a great passion for drug development in rare diseases,” says Dr. Philipson, underscoring a commitment that would shape his career’s trajectory.

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The Pharma Data

NOVEMBER 3, 2020

Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, announced today the U.S. LogicBio Therapeutics is dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms. LEXINGTON, Mass., About LogicBio Therapeutics.

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Pharmaceutical Technology

MARCH 20, 2023

Research – and money – going into even more advanced gene therapies is ramping up. In APAC, for example, industry commentators are predicting that the pharmaceutical sector’s historic focus on genetics and manufacturing will shift towards cutting-edge technology. Source: GlobalData. Source: GlobalData.

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