New Technologies for Gene Targeting + Gene Correction with David Segal, PhD
May 7, 2024
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David Segal, PhD, an abstract session co-chair at the ASGCT Annual Meeting, sheds light on groundbreaking advancements in gene targeting and correction. He discusses the transition of academic innovations like CRISPR from labs to industry, aiming to benefit rare disease patients. Segal highlights the growing influence of biotech in genetic therapies and the crucial challenges of effective delivery systems. The conversation also addresses the FDA's impactful approval of the first gene editing therapy, signaling exciting times ahead for genetic medicine.
Significant advancements in gene editing technologies, particularly CRISPR and epigenetic methods, are crucial for developing new RNA therapies and gene correction.
The collaboration between academia and biotech industries is enhancing the translation of research into effective treatments for various genetic diseases.
Deep dives
Advancements in Gene Editing Technologies
Recent discussions highlight significant advancements in gene editing technologies, particularly focusing on CRISPR and epigenetic editing methods. These innovations are paving the way for new RNA therapies and the application of artificial intelligence to facilitate gene correction. Notably, the FDA's approval of the first gene editing gene therapy, known as Cass Gevi, marks a pivotal moment in the field, showcasing the potential therapeutic benefits of these technologies. The increasing variety and sophistication of these approaches reflect the vibrant research landscape aimed at addressing genetic diseases, ranging from common conditions like cancer to rare genetic disorders.
Industry's Role in Translating Research into Therapies
The growing representation of industry at scientific meetings demonstrates a shift towards collaborative efforts between academia and biotech companies in developing therapeutics. A significant number of research presentations originate from industry, indicating a flourishing sector that is actively translating academic innovations into clinically relevant treatments. This trend is notable as graduates from academic programs increasingly seek employment in the biotechnology field, further bridging the gap between research and practical application. The emphasis on translating foundational research into therapies is crucial for addressing the diverse range of genetic diseases that require innovative solutions.
In this episode, host Lynnea Olivarez talks with David Segal, PhD, an abstract session co-chair at the ASGCT Annual Meeting. Dr. Segal discusses new technologies that he says have moved from academia to industry, and, hopefully soon, to rare disease patients.
Miss a session? Catch it on demand on the virtual platform for 30 days following the conclusion of the meeting!