XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

pharmaphorum

APRIL 23, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . For example, adding a small-molecule might stabilise the medicine, allowing levels of the target protein to increase.

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

Camargo

JULY 27, 2021

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 is a new therapeutic approach in which genes are used to treat or prevent diseases.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

Pharmaceutical Technology

MARCH 1, 2023

Nedisertib is under clinical development by Merck and currently in Phase I for Neuroendocrine Tumors. The drug candidate targets DNA-dependent protein kinase (DNA-PK). It was also under development for small cell lung cancer, rectal cancer and chronic lymphocytic leukemia (CLL). It is a new chemical entity.

Gene Editing 100

Gene Editing DNA Cosmetics Life Science 100

Pharmaceutical Technology

MARCH 1, 2023

Nedisertib is under clinical development by Merck and currently in Phase I for Head And Neck Cancer Squamous Cell Carcinoma. The drug candidate targets DNA-dependent protein kinase (DNA-PK). It was also under development for small cell lung cancer, rectal cancer and chronic lymphocytic leukemia (CLL).

Gene Editing 100

Gene Editing DNA Cosmetics Life Science 100

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics. Financial details were not disclosed.

Gene Therapy 40

Gene Therapy Gene Gene Editing In-Vivo 40

pharmaphorum

SEPTEMBER 18, 2020

Vertex is already the top player in CF with a quartet of exon-skipping medicines that tackle the mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that underlies the disease, but there are still a sizeable group of patients with no treatment options.

Gene Editing 105

Gene Editing Gene Vaccination Vaccine 105