CRISPR Therapeutics’ Post

Comings and Goings: Exagamglogene autotemcel (exa-cel) non-viral cell therapy for treatment of Sickle Cell Disease (SCD). Findings: A phase 3, single-group, open-label study was conducted in patients 12 - 35 years of age with SCD (n=44) who had had at least 2 severe vaso-occlusive crises (VOC's) in each of the 2 years prior to screening. CD34+ HSPCs were edited with the use of CRISPR-Cas9 at the erythroid-specific enhancer region of BCL11A to reactive fetal hemoglobin synthesis. Before exa-cel infusion, patients underwent myeloablative conditioning with PK dose-adjusted busulfan. The primary end point was absence of severe VOC's for at least 12 consecutive months. A secondary end point was lack of hospitalization for severe VOC's crises for at least 12 consecutive months. Median follow-up was 19.3 months (range, 0.8 to 48.1). Safety was also assessed. Of the 30 patients who had sufficient follow-up to be evaluated, 29 (97%; 95% confidence interval [CI], 83 to 100) were free from VOC's for at least 12 consecutive months, and all 30 (100%; 95% CI, 88 to 100) were free from hospitalizations for VOC's for at least 12 consecutive months (P<0.001 for both comparisons vs a null hypothesis of a 50% response). The safety profile was generally consistent with that of myeloablative conditioning and autologous HSPC transplantation. No cancers were observed. (CLIMB SCD-121; ClinicalTrials.gov number, NCT03745287.)

ATMPs and cancer medicines will go through a Joint Clinical Assessment (JCA) at the EU level starting in 2025. The JCA can potentially reduce duplicative reviews in EU Member States and accelerate access to transformative ATMPs. But the EU JCA must adopt innovative methodologies that reflect the unique nature of ATMPs, rather than the approaches designed for yesterday’s pharmaceuticals. This includes providing guidance on developing evidence outside randomized control trials and leveraging real-world evidence to confirm durability. We urge the HTA Coordination Group to develop approaches that won’t leave rare disease patients behind: https://lnkd.in/eZu5W6WS Want to explore this topic more? Be sure to join us tomorrow for our event co-hosted with ATMP Sweden on European Competitiveness in Advanced Therapies. For in-person attendance - https://lnkd.in/gUMMU9Vb For virtual attendance registration - https://lnkd.in/e72DSYjF #EUHTA #ATMP #HospitalExemption

Here is an invitation to the EFPIA - European Federation of Pharmaceutical Industries and Associations European Brain Council virtual event on the Rethinking of Alzheimer's disease detection and diagnosis pathway in Sweden. 26 September 2023 - 9h00/11h00 In the continuation of the publication 'Rethinking Alzheimer's Disease' White Paper https://lnkd.in/edQgFa8F and the Ageing Brain article https://lnkd.in/eUBf2D2A #alzheimer #detection #diagnosis #innovation

Verve Therapeutics granted Eli Lilly and Company exclusive, worldwide rights for five years to develop and commercialize Verve's lipoprotein(a) therapy for the treatment of atherosclerotic cardiovascular disease (ASCVD), ischemic stroke, thrombosis, and aortic stenosis. Verve will be responsible for developing lipoprotein(a) therapy through Phase I while Lilly will be responsible for the related costs and further activities with an option to expand the collaboration for an additional one year. Additionally, Verve has the option to share 40% of the development cost and profits. Verve will receive $30M up front in cash and $30M in equity by granting 1552795 shares of Verve's common stock at $19.32 per share and is eligible for up to $465M in research, development, and commercial milestones, plus high single and low-double-digit royalties. See this and similar full deal profiles when you become a DealForma customer. #pharma #biopharma #biopharmadatabase #vervetherapeutics #elililly

I'm excited to attend The C-Path Rare and Orphan Disease Conference to work on drug development solutions for rare diseases. I'll be sharing my experience in two sessions on mitochondrial and metabolic drug development and informed consent and data sharing. I'm looking forward to collaborating with experts in the field and making a significant impact... #rareandorphan #RareDisease #DrugDevelopment #Collaboration #InformedConsent #DataSharing #CPath

Less than 2 weeks away from our big Rare and Orphan Disease Conference in DC. With over 250 registrants thus far, and more than half representing bio-pharm/bio-tech and nonprofits! I'll be presenting/co-presenting the following sessions: September 11: RDCA-DAP Task Forces: Mitochondrial and metabolic drug development - addressing unmet needs through collaboration Co-Lead: Alexandre Bétourné, PhD, PharmD, PMP at Critical Path Institute (C-Path), PMP Danielle B. at CUREMITO, and Dima Martini-Drew MD Astellas Pharma US September 12: Rare Infections: New endpoints to inform trial design to treat virus BK infections Co-Lead: Juergen Beck at Memo Therapeutics AG September 13: Applying propensity methods to the U.S. transplant registry for external real-world evidence control arms for 5-year survival in the BENEFIT study On behalf of the #TransplantTherapeuticsConsortium American Society of Transplant Surgeons American Society of Transplantation Did you know? Critical Path Institute (C-Path) has the MOST #publicprivatepartnerships with FDA, across many therapeutics and populations Klaus Romero Daniel Jorgensen, MD, MPH, MBA Kristen Swingle Register here: https://lnkd.in/gEBYeHVa #raredisease #drugdevelopment #datasharing #collaboration

📅 Save the dates and register now for C-Path's Rare and Orphan Disease Conference, Sept. 11-13, in Washington. Patients, providers, researchers, clinicians, biopharmaceutical companies, regulatory reviewers and scientists will leave this 2.5-day conference with an understanding of: -C-Path’s Rare and Orphan Disease Program’s current and future impact on drug development in rare diseases -The Critical Path for Rare Neurodegenerative Diseases (CP-RND) initiative’s first year impact and highlights -How RDCA-DAP has been enhanced in the last year, and how the platform informs rare disease drug development -Transversal solutions to address bottlenecks in drug development Full details and register here: https://lnkd.in/gTS3GAnX Klaus Romero, Collin Hovinga, Terina N. Martinez, Ph.D., Alexandre Bétourné, PhD, PharmD, PMP, Sorin Fedeles, PhD, MBA, Ramona Belfiore-Oshan, Ramona Walls, Sergey Rakhilin, Amanda Klein, PharmD, CDCES (she-her) #CPath #RareandOrphan #RDCADAP #drugdevelopment #datasharing #register #Duchenne #ataxia #sicklecell #neurodegenerativediseases #pkd #transplanttherapeutics #huntingtonsdisease #globalhealth #collaboration

Looking forward to this series, especially as the first-ever treatment for this disease was approved by the FDA earlier this year. Please share this learning event and help us spread the word to the families who might benefit from tuning in!

📅 There's still time to register for C-Path's Rare and Orphan Disease Conference, Sept. 11-13, in Washington. Patients, providers, researchers, clinicians, biopharmaceutical companies, regulatory reviewers and scientists will leave this 2.5-day conference with an understanding of: -C-Path’s Rare and Orphan Disease Program’s current and future impact on drug development in rare diseases -The Critical Path for Rare Neurodegenerative Diseases (CP-RND) initiative’s first year impact and highlights -How RDCA-DAP has been enhanced in the last year, and how the platform informs rare disease drug development -Transversal solutions to address bottlenecks in drug development Full details and register here: https://lnkd.in/gTS3GAnX Klaus Romero, Collin Hovinga, Terina N. Martinez, Ph.D., Alexandre Bétourné, PhD, PharmD, PMP, Sorin Fedeles, PhD, MBA, Ramona Belfiore-Oshan, Ramona Walls, Sergey Rakhilin, Amanda Klein, PharmD, CDCES (she-her) #CPath #RareandOrphan #RDCADAP #drugdevelopment #datasharing #register #Duchenne #ataxia #sicklecell #neurodegenerativediseases #pkd #transplanttherpeutics #huntingtonsdisease #globalhealth #collaboration