pharmaphorum

JANUARY 10, 2022

The German group is paying $40 million upfront to kick off the alliance, which is focusing on the development of up to five CRISPR drugs that can be delivered in vivo for liver-targeted diseases. – are around one-third the size of the more commonly used Cas9.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 52

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US. 14, 2020 10:00 UTC.

Business Development 40

Business Development Development Gene Editing In-Vivo 40

pharmaphorum

DECEMBER 6, 2020

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting. They are due to enrol 45 apiece, and are scheduled to readout next year.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Delveinsight

FEBRUARY 11, 2021

Researchers from the University of Pennsylvania’s Abramson Cancer Center contemplate they have discovered one key to the lack of response, and it may be targetable with different drug combinations, as per their announcement. Enhancing the response to immuno-oncology in pancreatic cancer. Median overall survival in those patients was 5.8

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

The Pharma Data

DECEMBER 27, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869). About Lysogene.

Gene Therapy 40

Gene Therapy Clinical Trials Clinical Trials In-Vivo 40

XTalks

OCTOBER 1, 2020

Xtalks spoke with WeiQi Lin, MD, PhD, executive vice president, research & development, and principal scientist at DURECT Corporation — a company focused on developing drugs and drug delivery methods in therapeutic areas with significant unmet need — about her transition from academia to industry. Key Moments.

Scientist 52

Scientist In-Vitro Research Life Science 52