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$5 million raised towards gene therapies for muscular diseases

Drug Discovery World

This comes just as Myosana has appointed its new CEO, Dr Matthew Lumley, a physician scientist who has spent nearly two decades in academic medicine and drug development. The post $5 million raised towards gene therapies for muscular diseases appeared first on Drug Discovery World (DDW).

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Exa-Cel CRISPR Gene Therapy for Sickle Cell Disease Deemed Safe by FDA Advisory Panel

XTalks

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

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Viral vector provider opens new UK headquarters in Edinburgh

Drug Discovery World

Gene delivery technologies company VectorBuilder has opened a new office in Edinburgh, hoping to bolster its presence in the United Kingdom. The new location is part of Edinburgh Technopole, a Pioneer Group in Midlothian Science Zone.

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Verily – Janssen collaboration; Enhancing the response in pancreatic cancer; Avrobio gene therapy eradicates toxic substrate; Gut microbiome responsible for Multiple Sclerosis

Delveinsight

Immune-stimulating antibodies, which target the receptor CD40, are emerging as promising therapies for pancreatic cancer. The scientists analyzed samples from patients with the most common pancreatic cancer, pancreatic ductal adenocarcinoma (PDAC). Avrobio gene therapy eradicates toxic substrate in Fabry patient.

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Scribe and Sanofi partner to develop cell therapies for cancer

Pharmaceutical Technology

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Stem cells’ versatility in drug development  

Drug Discovery World

This pairing includes reprogrammed cells for both wild and mutation-specific Huntington’s Disease (H), Amyotrophic Lateral Sclerosis (ALS), and Alzheimer’s Disease (AD) and is expanding to include custom disease-relevant mutations. Disease relevant in vitro assays improve translation in vivo, leading to clinical success.

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Turning science into business: An optimised alternative to antibodies

Drug Discovery World

As the industry’s awareness of alternatives to traditional antibodies continues to grow, there is increasing demand for these alternatives, which we are seeing particularly within the therapeutic sector, to enable targeted therapeutics and enable the emerging gene therapy market. DS: The company now has clients across big pharma.