article thumbnail

Forge Biologics joins BGTC for new AAV gene therapies development

Pharmaceutical Technology

The AMP programme is a public-private partnership between a number of biopharmaceutical and life sciences companies, the US Food and Drug Administration (FDA) and the National Institutes of Health (NIH), along with non-profit and other organisations.

article thumbnail

New White Paper on Advanced Therapies Sets up Developers for Success

XTalks

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. The Xtalks White Paper, The Intersection of Rare Disease and Advanced Therapies: What it Means for Clinical Development , is available for free download. “They called him ‘The Boy.’

Insiders

Sign Up for our Newsletter

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

article thumbnail

How Our Future Health will impact drug discovery 

Drug Discovery World

Researchers working across the entire life sciences community, including academia, charities, industry and the NHS will be able to apply to use this information to discover more effective ways to prevent, detect and treat diseases and conditions such as cancer, Alzheimer’s, heart disease, arthritis, diabetes and stroke. .

Drugs 52
article thumbnail

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

article thumbnail

Biopharma Money on the Move: December 2 – 8

The Pharma Data

December is bringing frost in the North and plenty of cold hard cash for these life sciences companies. . European venture capital firm Forbion rounded up $545 million for its fifth life sciences fund. The impact of CNS diseases extends beyond patients—to their families and society as well.”

RNA 52
article thumbnail

Patient centricity and the changing pharmaceutical vista

pharmaphorum

Developing medicines – for people living with disease Dr Mullen’s current role involves providing medical monitoring support, as well as safety, pharmacovigilance, scientific, and medical advice across a range of client projects, including advanced therapies and orphan drugs. I still feel that it is very important,” Dr Mullen said. “We’ve

article thumbnail

Transcending expectations for cell & gene therapy development

Drug Discovery World

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. There are over 7,000 genetic diseases that could potentially be cured using gene therapy 10. Conclusion. About the author.