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The I am BIO Podcast explores the next frontier for CRISPR: more effective delivery. Plus, nominations are open for BIO’s George Washington Carver and Rosalind Franklin Awards. (476 words, 2 minutes, 22 seconds) P.S. BIO’s Education Alliance Partner, UCSF, is offering a 10-week virtual master class direct from Silicon Valley, “Entrepreneurship for Life Science/Healthcare,” offered Fall 2024. Learn more at an info session TODAY, April 23, at 2 PM ET. |
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I am BIO Podcast explores the next CRISPR frontier |
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We can achieve more gene editing cures if we can more effectively deliver CRISPR-Cas9 technology inside the human body, explores today’s new episode of the I am BIO Podcast.
The current situation: CRISPR-Cas9 enables a revolutionary sickle cell treatment that requires a bone marrow transplant, complete blood replacement, and weeks in the hospital. Although daunting, this ex vivo procedure is a one-time treatment for a lifetime benefit.
What’s next: In vivo treatment that requires only hours in a clinic would expand the horizons of what's possible for patients. Delivery is key to enabling CRISPR-Cas9 to fight against more diseases. New technology targeting hepatocytes—cells comprising 80% of the liver’s mass—enables in-vivo gene editing delivered in a few hours by intravenous drip. Regeneron SVP Christos Kyratsous, Ph.D., and Intellia CEO John Leonard, M.D., explain how their companies collaborate on this technology.
How it works: Lipid nanoparticles from Regeneron and Intellia contain fat molecules, which are naturally attracted to hepatocytes. The nanoparticles carry a CRISPR-Cas9 gene editing tool to address diseases expressed in the liver.
How well it’s working: The collaborators are developing a treatment for transthyretin amyloidosis (TTR) mutations, a genetic condition caused by a protein from the liver and impacting the heart or nervous system.
What they’re saying: The Phase III trial shows they can “suppress the levels of TTR expression in the body by about 90%,” says Kyratsous. “That’s exactly the promise of genome editing. It’s a one-and-done therapy.”
What’s next: Using the liver as a “biofactory,” the partners are working on a hemophilia treatment. They hope to confirm the possibility of addressing several other diseases through gene editing tools delivered to the liver.
Listen at bio.org/podcast or wherever you get your podcasts. |
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Nominations now open for BIO’s agriculture and environment awards |
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At the BIO Agriculture & Environment Summit, BIO officially opened nominations for the George Washington Carver and Rosalind Franklin Awards, recognizing biotech innovators.
The Rosalind Franklin Award recognizes women leaders who have pioneered innovations in agriculture, food systems, energy, and biobased manufacturing, thereby advancing the bioeconomy. The award is named for Rosalind Franklin, whose work advanced the discovery of DNA’s structure. Last year’s winner: Dr. Pam Marrone of Invasive Species Corp., which implements biobased, environmentally friendly solutions to protect biological diversity and curb climate change impacts.
The George Washington Carver Award recognizes innovators in agriculture, food systems, energy, and biobased manufacturing whose accomplishments advance the bioeconomy. George Washington Carver pioneered sustainable biobased products and energy derived from agricultural feedstock.
Last year’s winner: Dr. Hank Harris, whose Genvax Technologies focuses on the development of new mRNA/nanoparticle livestock vaccines.
Nominations are due by May 3 at 5pm EDT. The winners will be announced at the June 3-6 BIO International Convention.
Click here to nominate a leader for the George Washington Carver Award or Rosalind Franklin Award.
Read about the inaugural Rosalind Franklin Award winner, in 2014, who spoke to Bio.news about what the award still means to her a decade later.
Recap: Watch the highlights from the BIO Agriculture & Environment Summit, and read more about what policymakers and regulators said at the event. |
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