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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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Merck and Orna partner for RNA technology-based vaccines and therapies

Pharmaceutical Technology

These molecules synthesised newly are packaged densely into custom lipid nanoparticles (LNPs), which Orna has made to act on the body’s crucial tissues. The rights to the oRNA-LNP technology platform of Orna will be retained by the company, which will also progress various other fully owned programmes in oncology and genetic disease areas.

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Novartis announces lift of partial clinical trial hold and plans to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with SMA

The Pharma Data

We are very pleased that our comprehensive nonclinical data package has addressed all issues identified related to DRG toxicity and the FDA has reached the decision that we may proceed with our OAV-101 IT clinical trial program and initiate the STEER trial,” said Shephard Mpofu, M.D., SVP, Chief Medical Officer, Novartis Gene Therapies. “We

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n-Lorem Foundation Preps First Doses to Treat Ultra-Rare Disease Patients for Free

The Pharma Data

n-Lorem Foundation has taken on a challenge that many nations consider too great: treating patients with ultra-rare diseases (which affect 30 or fewer people) for free, for life. Genetic diseases are vastly more common and more complex than we used to think.”. Patient dosing is expected to begin later this year. “No