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NGGT Progresses First US Gene Therapy Trials For Rare Conditions

Supported By China Data

 
• By Dexter Jie Yan

Next Generation Gene Therapeutics is fast-tracking clinical trials in the US for its gene therapy candidates for Bietti's crystalline dystrophy and classic phenylketonuria, supported by China data, the Chinese venture's CEO tells Scrip in an interview.

US China flags
NGGT leveraging China's large rare disorder patient pools to speed up US trials • Source: Shutterstock

For global developers of rare disease drugs, China and its 1.4 billion population may offer relatively large pools of patients with such disorders. Clinical data derived from trials in the country can also be used to help speed up clinical studies in the US or Europe.

This is a situation executives at US- and China-based Next Generation Gene Therapeutics (NGGT) believe they can leverage to make inroads into the

Key Takeaways

  • Next Generation Gene Therapeutics (NGGT) currently has two lead assets being evaluated in Phase I/II studies in the US and China.

  • NGGT-001 and NGGT-002 are being developed for the treatment of the rare diseases Bietti's crystalline dystrophy and classic phenylketonuria, respectively

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