Pharmaceutical Technology

JULY 29, 2022

CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year. Ultragenyx Pharmaceuticals has an orthinine transcarbamylase (OTC) activator drug in Phase III trials. Future Possibilities.

RNA 245

RNA Gene Expression Gene Gene Therapy 245

Pharmaceutical Technology

JUNE 19, 2023

Pharmaceutical companies are putting their trust in the immense potential this new generation of medicine has for treating individuals with rare genetic diseases, which currently affect an estimated 280 million patients worldwide. The majority of these are in Phase I (68%) and Phase II (24%), with just 8% in Phase III.

Gene Therapy 130

Gene Therapy Gene Packaging Packaging 130

Drug Discovery World

JANUARY 30, 2024

“Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said. Streamlining trial designs and regulatory pathways can help. Optimising these processes and exploring alternative delivery methods can bring down costs.

Gene Therapy 71

Gene Therapy Manufacturing Gene Clinical Trials 71

Drug Discovery World

AUGUST 28, 2023

DDW’s Diana Spencer speaks to Peter Hamley , Chief Scientific Officer of Samsara Therapeutics, to understand the role autophagy plays in neurodegenerative diseases and how boosting this process could increase healthspan. DS: Samsara’s autophagy inducing candidate SAM001 will be the first agent of its class to enter clinical trials.

Genetics 52

Genetics Protein Genetic Disease Clinical Trials 52

The Pharma Data

DECEMBER 8, 2020

The ImmunoTAC platform pairs proprietary payloads that modulate key disease modifying pathways with monoclonal antibodies directed at specific disease sites. Silverback’s lead candidate is currently in a Phase I trial in adults with HER2-expressing solid tumors. Nuance Pharma . Sigilon Therapeutics .

RNA 52

RNA Antibody Life Science Protein 52

The Pharma Data

OCTOBER 15, 2020

The committee is tasked with supporting BeyondSpring’s business development activities related to its lead asset, Plinabulin, and other pipeline assets. Mukul Agarwal, former vice president of Corporate Development, at Forty Seven, Inc., BeyondSpring – BeyondSpring Inc. Sabbagh will replace David Thompson who is retiring.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

XTalks

OCTOBER 1, 2020

Dr. Lin has made several transitions throughout her career, from being a clinician at an academic center, to academic research, to heading business development in industry. Working on finding therapies for genetic diseases at NeuBase, Dr. Sooter says, “Everyday, we get up and that’s our goal and it really drives everyone.

Scientist 52

Scientist In-Vitro Research Life Science 52