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On Day 1 of the BIO CEO and Investor Conference, CEOs and experts explained why it’s a great time to invest in innovations such as gene therapy and vaccines. Meanwhile, the I am BIO Podcast launches its new season with a look at the first approved CRISPR-based gene therapy and its impact on sickle cell patients. (780 words, 3 minutes, 54 seconds) |
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Is CRISPR the new sickle cell savior? |
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A CRISPR-based breakthrough has dramatically improved the outlook for sickle cell disease (SCD), but patients face challenges and a legacy of racism, say guests on today’s opener of the new season of the I am BIO Podcast. What is sickle cell disease? A genetic condition impacting nearly 100,000 Americans, most of African descent, can cause extreme pain, organ damage, and early death.
A breakthrough: Vertex’s CASGEVY—which became the first CRISPR-based medicine to obtain Food and Drug Administration (FDA) approval in December—prevented pain crises for 90% of clinical trial participants and may prevent further organ damage, says David Altshuler, Chief Scientific Officer of Vertex.
“We think of it as a one-time treatment with a potential for lifetime benefit,” says Altshuler. “CASGEVY can’t restore the function to an organ that was damaged. But the hope is that by addressing the underlying cause, the pain crises can be reduced or eliminated, the patient has improved quality of life.”
Vertex worked closely with patients—but more work is needed to ensure patients are heard by the healthcare community, explains Mapillar Dahn, a mother of three girls with SCD and founder of the MTS Sickle Cell Foundation.
Why it matters: “I think the neglect for innovation and science in sickle cell disease probably does relate to racism. I think it also relates to the belief that you could not make a good business if you were focused on sickle cell disease,” says BIO Board Chair and former CEO of Global Blood Therapeutics, Dr. Ted Love. "I wanted to prove that we could do both. We could make very innovative therapies to help these patients in a dramatic way, and we could build a valuable business while doing so.
Listen: You can listen at www.bio.org/podcast or wherever you get your podcasts! |
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How to follow the BIO CEO & Investor Conference |
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Now is the time to invest in gene therapy and vaccines, say CEOs and experts |
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On Day 1 of the BIO CEO and Investor Conference, we heard about innovations in gene therapy and vaccines, as well as the regulatory and investment outlook. The panel: CEOs developing cutting-edge gene therapies discussed challenges in development, investment, scaling manufacturing, and commercialization.
The key takeaway: The science of gene therapy is progressing at a remarkable pace, but for the medicines to reach patients, investment, scaling manufacturing, and regulatory processes need to keep up.
What they’re saying: “We’re seeing a number of different therapies that could be functional cures to diseases that are being developed and being approved today, and I think this is exactly what society wants from our industry—is functional cures to these diseases,” said Nolan Townsend, CEO of LEXEO. |
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The panel: CEOs and BIO experts discussed BIO’s report, The State of Innovation in Vaccines and Prophylactic Antibodies for Infectious Diseases, and opportunities and challenges to developing and commercializing much-needed vaccines.
The key takeaway: The vaccine pipeline boasts incredible innovation and breadth—from epidemic and pandemic pathogens to innovations in adult immunizations and mRNA for cancer—but lacks depth due to market hurdles.
What they’re saying: “Now is the time to invest in vaccines because we have these very exciting new modalities. We have platform technologies that can move from one disease to the other using the same platform—whether it’s prophylactic antibody approach or if it’s a mRNA, DNA vaccines,” said David Thomas, Senior Vice President of Industry Research and Analysis at BIO. “And the world needs these vaccines because there will be future pandemics.” |
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Rare Disease Week puts focus on ORPHAN Cures Act
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This year, Rare Disease Week is an important opportunity to advance the ORPHAN Cures Act, bipartisan legislation that will benefit rare disease patients and R&D.
Why it matters: The Inflation Reduction Act (IRA) exempts orphan drugs for rare diseases from price controls—but only if the drugs are approved for a single indication.
The fix: The ORPHAN Cures Act would change the IRA’s incentive structure to encourage follow-on development, which is how many orphan drugs are developed.
Learn more about ORPHAN Cures and why BIO and American voters support it. |
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President Biden’s Tuesday: Meeting with congressional leaders at the White House.
What’s Happening on Capitol Hill: With four days until a partial government shutdown, funding discussions continue. |
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