Antibody Gene Genetic Disease 
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.
pharmaphorum
OCTOBER 4, 2022
AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. The post AstraZeneca pays record 660% premium for gene editing company LogicBio appeared first on.
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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. was swooped up by Real Chemistry in January.
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. This design protects antibodies from the innate immune system.
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. Late in 2019, a Fabry patient in phase 2 clinical trial of ex vivo lentiviral gene therapy AVR-RD-01 became the first patient dosed using Plato.
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. CRISPR Therapeutics and Vertex are also in the running with their gene-editing candidate CTX001, in phase 1/2 trials which are due to generate final results later this year.
The Pharma Data
JULY 6, 2021
Data will include the final analysis from the phase IIIb STASEY study of Hemlibra® (emicizumab) and updated data from the phase I/II study of SPK-8011, an AAV-based gene therapy in development by Spark Therapeutics (a member of the Roche Group). Hemlibra is a bispecific factor IXa- and factor X-directed antibody.
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