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Three Gene Therapies, Moderna Metabolic Drug In Inaugural START Class; CBER Oversubscribes

 
• By Kate Rawson

Seven rare disease programs make up the first round of participants in the US FDA’s ‘Operation Warp Speed’-style program. Sponsors will benefit from ‘rapid, ad hoc’ communication with the agency.

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FDA to seven rare disease sponsors: Let's Go. • Source: Shutterstock

The US Food & Drug Administration’s inaugural class of applications for its START rare disease pilot program includes three early-stage gene therapy programs intended to treat pediatric diseases, including Rett syndrome (Neurogene), NGLY1 deficiency (Grace Science), and Canavan disease (Myrtelle).

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Pipeline To Product – What Is Driving Cell And Gene Therapy Progress In India?

 
• By Vibha Ravi

US-based Colossal Labs has claimed to bring back the dire wolf from extinction. While the science is not as dramatic in India yet, cell and gene therapies are making progress as treatments for cancer and other diseases. Pink Sheet takes a look at what is driving CGT success and growth

Gene Therapy ‘Survival Of The Fittest’ – Why Companies Need To Understand Health Systems

 
• By Eliza Slawther

Cell and gene therapy manufacturers must consider the practicalities of their product within the context of a health care system before it comes onto the market to be successful, experts from Novartis, AstraZeneca and England’s National Health Service say.

Device-Like System Proposed For Low-Risk Human Cell Therapies, Tissue-Based Products At US FDA

 
• By Kate Rawson

A risk-based approach to human cell therapies and tissue-based products could incentivize development and prevent bad actors from taking advantage of the current FDA system.

US Approach To Cell And Gene Therapy Regulations ‘Less Strict’ Than EU

 
• By Eliza Slawther

Experts working in the advanced therapy space say the US has less strict criteria for regulatory pathways for cell and gene therapies than the EU, particularly for products in early development.

More from Advanced Technologies

Device-Like System Proposed For Low-Risk Human Cell Therapies, Tissue-Based Products At US FDA

 
• By Kate Rawson

A risk-based approach to human cell therapies and tissue-based products could incentivize development and prevent bad actors from taking advantage of the current FDA system.

US Approach To Cell And Gene Therapy Regulations ‘Less Strict’ Than EU

 
• By Eliza Slawther

Experts working in the advanced therapy space say the US has less strict criteria for regulatory pathways for cell and gene therapies than the EU, particularly for products in early development.

US FDA Under Makary: MAHA With A Lighter Touch

 
• By Michael McCaughan

US FDA Commissioner nominee Martin Makary is being embraced by industry, and Senate Democrats, as a more traditional pick than other Trump Administration nominees, but the Make America Healthy Again agenda still is clearly coming to the agency.