Gene - based medicines

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CRISPR Therapeutics AG (CRSP) William Blair's 45th Annual Growth Stock Conference (Transcript)
Seeking Alpha· 2025-06-03 20:07
Core Insights - CRISPR Therapeutics is focused on creating transformative gene-based medicines for serious diseases [3] - The company has made progress with its therapy CASGEVY, which is being commercialized in partnership with Vertex [4] - Several clinical trials are ongoing, with readouts expected in the next 6 to 12 months that will influence the company's strategic direction [4] - The company has multiple preclinical programs and aims to expand its pipeline significantly over the long term [5] Company Overview - Raju Prasad, the Chief Financial Officer, provided an overview of the company's goals and current projects [2] - The company is leveraging its gene-editing technology to develop additional products beyond CASGEVY [4] - Resource allocation is a consideration for the company as it seeks to grow its offerings sustainably [5]
CRISPR Therapeutics and Sirius Therapeutics Announce Multi-Target Collaboration to Develop Novel siRNA Therapies
Globenewswire· 2025-05-19 20:15
Core Insights - CRISPR Therapeutics and Sirius Therapeutics have formed a strategic partnership to co-develop and commercialize SRSD107, a long-acting Factor XI (FXI) small interfering RNA (siRNA) for thromboembolic disorders [1][2] - SRSD107 has shown promising results in Phase 1 clinical trials, achieving over 93% reduction in FXI activity and more than twofold increase in activated partial thromboplastin time (aPTT), with effects lasting up to 6 months post-dosing [1][3] - The collaboration includes an upfront payment of $25 million in cash and $70 million in equity from CRISPR to Sirius, with a 50-50 cost and profit-sharing structure for SRSD107 [1][8] Company Overview - CRISPR Therapeutics is focused on creating transformative gene-based medicines and has a diverse portfolio across various disease areas, including cardiovascular diseases [10] - Sirius Therapeutics is a clinical-stage biotech company developing innovative siRNA therapies, with SRSD107 being its most advanced product targeting thromboembolic disorders [17][18] Clinical Development - The clinical program for SRSD107 includes two Phase 1 trials demonstrating safety and robust pharmacodynamic effects, with a Phase 2 trial planned to evaluate its efficacy in preventing venous thromboembolism (VTE) in total knee arthroplasty patients [3][7] - SRSD107 is designed to selectively inhibit FXI, aiming to reduce thrombotic events while minimizing bleeding risks, offering a differentiated approach compared to existing therapies [2][16] Market Potential - Thromboembolic disorders are a significant global health issue, affecting millions and representing a substantial unmet medical need [2][15] - The addressable patient population for SRSD107 includes those with atrial fibrillation, venous thromboembolism, cancer-associated thrombosis, and other conditions where existing therapies pose bleeding risks [2][3]