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.

Gene Therapy 264

Gene Therapy Gene Development Genetics 264

XTalks

APRIL 1, 2025

Hemophilia A and B are rare genetic bleeding disorders caused by a deficiency or dysfunction of clotting factors VIII (FVIII) and IX (FIX), respectively. In hemophilia patients, inhibitors are antibodies that interfere with the effectiveness of clotting factor replacement therapies. Among these are three gene therapies.

FDA Approval 59

FDA Approval RNA Clinical Trials Clinical Trials 59

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

JANUARY 12, 2021

It is an IgM antibody targeting Death Receptor 5 (DR5), and IGM is hoping to test Birinapant with IGM-8444 for the treatment of solid cancers later this year. AvantGen Enters into a Licensing Agreement for its Anti-SARS-CoV-2 Antibodies with IGM Biosciences. million after Birinapant successfully becomes a part of the Phase I trials.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

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.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

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

Gene Editing 64

Gene Editing Gene Genomics Genome 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.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52