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Armed with new gene therapy tools, a biotech startup promises a better way to target muscle diseases

BioPharma Drive: Drug Pricing

Two-year-old Kate Therapeutics is launching publicly with $51 million in funding and a licensing deal with Astellas Pharma for a neuromuscular disease gene therapy.

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Scaling Phage Therapy

Codon

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. alone, according to the Centers for Disease Control and Prevention.

Therapies 116
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Unlocking the power of stem cell therapy

Drug Target Review

What key findings about stem cell behaviour, differentiation and integration within host tissues impact the development of stem cell therapies? These characteristics position ADSCs as promising candidates for addressing a wide range of medical conditions, from degenerative diseases to traumatic injuries.

Therapies 105
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Novartis, Voyager Therapeutics reach license option agreement for next-generation gene therapy vectors for neurological diseases

The Pharma Data

Novartis today announced a license option agreement with Voyager Therapeutics, a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies, for three capsids to use in potential gene therapies for neurological diseases, with options to access capsids for two other targets.

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Advancing neurological therapies with Dr Bruce Leuchter

Drug Target Review

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? There are a lot of drug development challenges that are, in some cases, unique to neuroscience, including neurological diseases, psychiatric disorders, and rare diseases affecting the nervous system.

Therapies 104
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Gamma delta T cells: a rising star in cancer therapy

Drug Target Review

Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.

Therapies 105
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What Do Patients Have to Say about Gene Therapy Trials? An Upcoming FDA Public Meeting to Hear from Patients and Caregivers

FDA Law Blog: Drug Discovery

Knowing they could not hold PFDD meetings for every disease ( there are over 10,000 rare diseases alone ), they decided to turn PFDD meetings over to disease advocacy organizations, calling them Externally-Led Patient Focused Drug Development (EL-PFDD) meetings. There have been challenges in gene therapy development.