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CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea. The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. Future Possibilities.

RNA 245
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Why demand is rising for secure and climate-controlled gene therapy services

Pharmaceutical Technology

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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EdiGene Expands Management Team by Appointment of Head of US Subsidiary Dr. Bo Zhang and Head of Business Development Dr. Kehua Fan

The Pharma Data

.–( 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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LogicBio Therapeutics Receives FDA Fast Track Designation for LB-001 for the Treatment of Methylmalonic Acidemia (MMA)

The Pharma Data

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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Advances in Rare Disease Drug Development: Disease Biology, Regulatory Support, Building Relationships and the Patient Voice

XTalks

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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BioSpace Movers & Shakers, Dec. 18

The Pharma Data

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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Investment fuels AI-driven development of breakthrough genomic medicines

pharmaphorum

Now, he leads Whitelab Genomics as its artificial intelligence (AI) platform powers the development of genomic therapies – an emerging field in which genetic sequences are injected into cells to target and repair damaged genes. and France to accelerate the development of genomic therapies.

Genome 105