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Biotech Blueprint

Katerina Roznik
Biotech Blueprint
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  • FDA Shake Up, Sarepta Rebounds, and Trump Targets Drug Prices — This Week in #Biotech 63
    Subscribe to Biotech Blueprint for free HERE and visit Biotech Blueprint website for more.
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  • Sarepta, Elevidys, and the Future of AAV Gene Therapy
    Subscribe to Biotech Blueprint HERE and to Biotech Capital Compass HERE.In this episode of the Biotech Blueprint podcast, I teamed up with the Biotech Capital Compass to break down Sarepta Therapeutics’ recent safety crisis, and what the FDA’s partial green light means for Elevidys, investor confidence, and the broader AAV field. We dig into the science, regulatory dynamics, and what might come next.
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  • Sarepta’s Gene Therapy Crisis Deepens — This Week in Biotech #62
    Subscribe to Biotech Blueprint HERE.
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  • CRISPR in 2025: Clinical Use, Regulatory Pressure, and Future Promise
    Welcome back to Biotech Blueprint!In this week’s special episode, I teamed up with the Biotech Capital Compass to examine where gene editing stands today, scientifically, commercially, and clinically. This marks the start of a multi-part series on gene therapies, viewed through both scientific and investment lenses.Subscribe to Biotech Blueprint and Biotech Capital Compass.
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  • Sarepta’s $400M Pivot, MiNK’s 500% Surge, and AML Breakthroughs — This Week in Biotech #61
    Subscribe to Biotech Blueprint for free HERE.In this episode: Layoffs, CRLs, and standout trial data from Sarepta, MiNK, SELLAS, DiaMedica, and more (July 11–17, 2025).THIS WEEK’S KEY TAKEAWAYS 🔑Sarepta led the week with sweeping changes: the company laid off 500 employees and pivoted toward its siRNA platform after safety concerns with its Duchenne gene therapy, Elevidys, triggered an FDA-mandated black box warning. While painful, the restructuring was met with investor optimism, driving the stock up nearly 40%.On the clinical front, SELLAS reported standout phase 2 data for its CDK9 inhibitor in relapsed AML, with response rates more than double historical expectations and a move into front-line trials now underway. Similarly, DiaMedica impressed with early data in preeclampsia, a space with no approved drugs, showing significant blood pressure reductions without crossing the placenta.MiNK Therapeutics soared 500% after publishing a striking case study: a full remission in metastatic testicular cancer using its off-the-shelf iNKT cell therapy. While the data is from a single patient, the durability and safety profile suggest meaningful potential for solid tumors.Not all news was positive. Capricor’s Duchenne therapy was rejected by the FDA despite priority review, sending shares tumbling over 50%. Ultragenyx also received a CRL for its Sanfilippo gene therapy, though the delay was attributed to manufacturing issues, not clinical concerns.Elsewhere, AstraZeneca’s AL amyloidosis antibody failed its phase 3 primary endpoint but showed signs of efficacy in a subset of patients, setting up a potential niche strategy. And in a surprise twist, 23andMe was acquired by Anne Wojcicki’s nonprofit TTAM Research Institute, edging out Regeneron in a bidding war that reignited debate over the future of consumer genomics.
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Connecting with biotech, pharma, and academic professionals to explore the latest in science, startups, market trends, and breakthroughs. For more content, please subscribe to biotechblueprint.substack.com.
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