Upcoming Events

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OTAT Town Hall: Clinical Development of Gene Therapy Products for Rare Diseases

The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) is hosting a virtual town hall to answer stakeholder questions related to the clinical development of gene therapy products for rare diseases (excluding hematology/oncology products). This event is part of a series to answer questions from stakeholders about a variety of topics on which OTAT has regulatory oversight.

FOCUS FOR THIS TOWN HALL: GENE THERAPY PRODUCTS INTENDED TO TREAT RARE DISEASES

There are around 7,000 known rare diseases affecting more than 30 million people in the United States. Many rare diseases are life-threatening, and only a fraction of rare diseases have an available treatment. Developing safe and effective gene therapy products to treat rare diseases can be challenging; despite these challenges, gene therapy-related research and development continue to grow rapidly. To support continued growth in the gene therapy field, FDA developed a guidance document in January 2020 to provide recommendations to sponsors developing gene therapy products intended to treat rare diseases.

MEETING LOGISTICS

  • Date: Tuesday, February 7, 2023
  • Time: 11:30 a.m. to 1:00 p.m. ET
  • Location: The webinar will be held via Zoom.
  • Registration: Registration is required. Please register for the event now by clicking the button below.

Register Here

2023 ASPET Guppy Tank Translational Science Pitch Competition

The “Guppy Tank” competition will showcase translational science pitches from four young scientists who will be coached by mentors with established experience in the biotech, pharma, and entrepreneurship realms. In addition, the Guppy Tank event will feature a keynote discussion by Dr. Trivia Frazier (President and CEO of Obatala Sciences) who will highlight the importance of a translational vision to scientific innovations and effective strategies for a successful science pitch. This session will be an exciting and essential educational opportunity for young scientists to hone their translational scientific communication skills while getting publicly recognized for their talents.

View Flyer Here

Please use the link below to fill out the application by January 6th.

www.aspet.org/guppytank2023

If you have any questions, please do not hesitate to reach out to Khalid Garman (khalid.garman@nih.gov) or Saranya Radhakrishnan (sara.radkrish@gmail.com)

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2023 ASPET Guppy Tank Translational Science Pitch Competition

The “Guppy Tank” competition will showcase translational science pitches from four young scientists who will be coached by mentors with established experience in the biotech, pharma, and entrepreneurship realms. In […]

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Clinical Development of Gene Therapy Products for Rare Diseases

The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) is hosting a virtual town hall to answer stakeholder questions related to the clinical […]

Find out more »

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