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Breakthrough: Rare Gene Mutation Offers Clues to Preventing Type 1 Diabetes

AuroBlog - Aurous Healthcare Clinical Trials blog

A unique genetic mutation in two siblings – that has never been seen in anyone else – has been discovered by UK researchers at the University of Exeter, pointing the way towards new treatment options for type 1 diabetes. The mutation is in the gene for a protein called programmed death-ligand 1 (PD-L1), and a […]

Gene 184
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Sickle cell pipeline narrows as gene therapy developers rethink research plans

Bio Pharma Dive

Graphite Bio and Sangamo are stopping work on their respective sickle cell gene therapies, while Intellia revealed partner Novartis ended development of its genetic treatment for the blood disease.

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Leveraging Genetic Testing for Enrolling Rare Disease Trials

Worldwide Clinical Trials

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.

Genetics 168
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Yet another gene therapy developer turns to layoffs

Bio Pharma Dive

Taysha Gene Therapies is narrowing its research efforts and cutting around 35% of its workforce, the latest in a series of layoffs announced by makers of genetic medicines.

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Astellas to support development of Taysha’s gene therapy programmes

Pharmaceutical Technology

Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha Gene Therapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Lilly agrees to acquire gene therapy developer Akouos for $610m

Pharmaceutical Technology

Eli Lilly and Company has signed a definitive agreement for the acquisition of all outstanding shares of precision genetic medicine firm Akouos for a total deal value of up to nearly $610m or up to $15.50 Akouos focuses on developing adeno-associated viral gene therapies to treat inner ear conditions, including sensorineural hearing loss.

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Gene Therapy and Pharmacokinetics

Camargo

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.