Pharmaceutical Technology

OCTOBER 25, 2022

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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Gene Therapy Gene Development Genetics 264

Delveinsight

AUGUST 26, 2021

Shape Therapeutics inks gene therapy deal with Roche worth up to USD 3 Billion. Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease.

Vaccine 98

Vaccine Vaccination Gene Therapy RNA 98

Delveinsight

FEBRUARY 11, 2021

Immune-stimulating antibodies, which target the receptor CD40, are emerging as promising therapies for pancreatic cancer. Avrobio gene therapy eradicates toxic substrate in Fabry patient. Moreover, those that did become ill showed relatively mild disease, from which they recovered quickly.

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Gene Therapy Gene Immune Response In-Vivo 52

Pharmaceutical Technology

APRIL 24, 2023

Conde says the use of nanoparticle delivery for gene therapies has become more common over recent years. Nanoparticles can be tailored to target specific cells or tissues, release gene therapies in a regulated manner, reduce toxicity, and increase stability,” he added. These could feature in the next four to five years.

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Drug Delivery Vaccine Vaccination Research 147

pharmaphorum

OCTOBER 4, 2022

US-based LogicBio is a clinical-stage genetic medicine company focused on genome editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood. As per the agreement, Alexion will initiate a cash tender offer through a subsidiary to acquire all outstanding shares of LogicBio for $2.07

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Gene Editing Gene Genome Genomics 64

The Pharma Data

JANUARY 9, 2021

(NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants.

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Gene Therapy Gene Trials Clinical Trials 52

pharmaphorum

FEBRUARY 17, 2022

The drug – which costs $125,000 at US list prices – can be used either alone or in combination with hydroxyurea, a well-established therapy for the disease.

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Drugs Gene Therapy Gene Editing Gene 59