Remove Gene Therapy Remove Genomics Remove Marketing Remove Regulation
article thumbnail

FDA releases draft guidance for cell and gene therapies

BioPharma Reporter

The FDA has released two draft guidance documents: for human gene therapy products incorporating human genome editing, and for CAR T cell products.

article thumbnail

BIO comments on FDA draft guidance for human gene therapy products incorporating human genome editing

BioPharma Reporter

BIO, the worldâs largest trade association for the biotech industry, has responded to the US Food and Drug Administration's draft guidance on developing human gene therapy products that incorporate genome editing (GE) of human somatic cells.

Insiders

Sign Up for our Newsletter

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

article thumbnail

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

XTalks

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

article thumbnail

Can vertical AI advance cell and gene therapies? 

Drug Discovery World

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. Here’s what this means for pharmaceutical leaders and patients.

article thumbnail

Can characterisation industrialise cell and gene therapy?  

Drug Discovery World

The cell and gene therapy (CGT) market is rapidly expanding, and projections suggest it will reach over $90B by 2023. Challenges of cell and gene therapy manufacturing Compared to traditional biologics, CGTs are living drugs, making their development and manufacturing substantially more complex.

article thumbnail

Urgent action needed to reduce high costs of gene therapies

BioPharma Reporter

The extremely high costs of gene therapies are unsustainable, and a global commitment to affordable, equitable access to these treatments is urgently needed, concluded the organising committee of a conference on human genome editing.

article thumbnail

Broken String Biosciences to advance ALS research with Francis Crick Institute

BioPharma Reporter

Broken String Biosciences, a genomics company driving cell and gene therapy developing, has entered a research collaboration with the Francis Crick Institute, a biomedical discovery institute dedicated to understanding the biology underlying health and disease.