article thumbnail

C4XD and AstraZeneca enter $402m deal to develop respiratory disease therapy

Pharmaceutical Technology

According to the deal, C4XD is entitled to get up to $16m in pre-clinical milestone payments before the commencement of the first clinical trial. . on meeting clinical development and commercial milestones in the future, in addition to tiered royalty payments, upon the marketing of the product.

article thumbnail

Gene Therapy and Pharmacokinetics

Camargo

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. PK Planning for Gene Therapy Development Programs. Gene Expression Considerations.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Another Astellas gene therapy hits a safety hurdle

pharmaphorum

The delivery vector – an AAV8 capsid – is designed to allow gene expression directly in target tissues like skeletal muscle. AT845 uses the same adeno-associated virus (AAV) vector platform as AT132, but so far there have been no reports of liver toxicity with the Pompe disease candidate.

article thumbnail

Prominent Reasons Behind The Failure Of NAMPT inhibitors And Key Strategies Adopted By Developers To Overcome Such Challenges

Roots Analysis

NAD + is involved in a wide range of cellular processes, including energy metabolism, DNA repair, and gene expression. Despite their potential therapeutic benefits, NAMPT inhibitors have faced several challenges in clinical development.

article thumbnail

Symvivo’s Oral COVID-19 Vaccine Enters Clinical Trials

XTalks

We are exceptionally pleased to commence dosing of our oral DNA vaccine for COVID-19 as we continue scale-up and manufacturing activities for future clinical development,” Symvivo’s chief medical officer Eric Sievers said in a press release from the company. Related: Red Meat Allergy Test Gets FDA Clearance. “We

article thumbnail

Fulcrum Therapeutics Announces Pricing of Public Offering of Common Stock – Jan 20, 2021

The Pharma Data

Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum’s proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression.

article thumbnail

2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences

XTalks

Omer maintains that the solution to this challenge is something that’s already in practice by developers of the mRNA vaccines. What you do is you insist that a proper clinical development program is followed and that has been followed with trial data of tens of thousands of people,” says Dr. Omer.