| Amicus spins off gene therapy unit in $600M SPAC deal as CEO becomes chief at Caritas Editas' gene-editing data has company touting prime time based on early vision improvement signals but questions remain AstraZeneca moves deeper into rare diseases, spending $150M on late-phase hematology prospect Sponsored: Navigating the complexities of oncology clinical trials in an increasingly competitive landscape I-Mab hits the deal table with biopharma majors to talk partnerships and investments: report Sponsored: A New Reference Model for Clinical Trial Recruitment, Enrollment and Retention Success Pfizer tightens DMD patient criteria after serious adverse events crop up in phase 3 gene therapy trial Iterum will give UTI antibiotic another go with new trial after meeting with FDA Novartis, Sanofi-backed Expansion snags $80M for neurodegenerative RNA assets to rival Dyne, Locanabio FDA's Marks takes over vaccines office as agency prepares to part ways with 2 key officials Eli Lilly taps Reify Health's Care Access to diversify trials of Verzenio Medtronic's newly acquired radial artery catheters go global with European approval In vivo gene editing grabs the spotlight after Intellia triumph, but challenges loom Amazon taps VR, voice assistant startups and 8 others for inaugural digital health accelerator Featured Story | By Ben Adams Amicus has endured a tough few years of pipeline flops and a roller coaster stock ride, but now it’s riding the SPAC wave, spinning off its gene therapy unit in a $600 million deal. read more |
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Top Stories By Annalee Armstrong Editas has confirmed safety—and its concept—in a small group of patients who have received the gene-editing therapy EDIT-101. The small amount of data also suggests that the middle dose group is starting to see some signals that their vision may be improving. read more By Nick Paul Taylor AstraZeneca is continuing to build out its rare disease pipeline, pulling the trigger on an option to buy Caelum Biosciences that it picked up in its takeover of Alexion. The buyout gives AstraZeneca control of a phase 3 treatment for the rare hematology disease light chain amyloidosis. read more Sponsored by: Premier Research With the shift from traditional chemotherapy agents to innovative immuno-oncology drugs and cell and gene therapies, oncology clinical trials have increased in complexity. read more By Nick Paul Taylor I-Mab has had a big 12 months, landing an $180 million upfront from AbbVie and delivering a series of readouts from clinical trials. Now, the Chinese biotech is reportedly seeking to build on its progress by talking to global biopharma companies about partnerships and investments. read more Sponsored by: IPM.ai Rare diseases demand a modern recruitment approach grounded in uncovering the ideal, medically-eligible patient population with the highest propensity for study enrollment and retention. read more By Annalee Armstrong Pfizer is tightening up the criteria for Duchenne muscular dystrophy patients to participate in a phase 3 clinical trial for an experimental gene therapy after three serious adverse events were seen in ongoing studies. read more By Kyle LaHucik The FDA denied Iterum Therapeutics' antibiotic approval in July, but, after meeting with the regulator last week, the biotech is ready to move forward on another phase 3 trial in uncomplicated urinary tract infections. A trial design could come as early as third-quarter earnings, RBC Capital Markets analysts estimated. read more By Kyle LaHucik Already backed by Sanofi and Novartis' venture arms, Expansion Therapeutics now has another $80 million to bankroll preclinical studies in neurodegenerative diseases. The biotech aims to have the studies done in the coming three years in myotonic dystrophy type 1, ALS and frontotemporal dementia. read more By Eric Sagonowsky Amid the heated debate over COVID-19 booster shots, two key FDA vaccine experts said they'd be leaving the agency last month. In an effort to smooth over the transition, Peter Marks, director of the agency's Center for Biologics Evaluation and Research, has officially taken over the FDA's vaccines office on an acting basis. read more By Kyle LaHucik Having already partnered on trials for Eli Lilly's COVID-19 treatment bamlanivimab, the Indy Big Pharma and Care Access are once again working together on decentralized clinical trials. This time around, they'll work to diversify a phase 3 trial in patients with breast cancer. read more By Andrea Park Barely a year after the Rist radial access catheter technology made its operating room debut in Chicago last September, Medtronic has progressively expanded its availability across the U.S. and now, with a freshly minted CE mark, will also bring the devices overseas. read more By Arlene Weintraub Investors are enthusiastic about the next generation of gene-focused treatments, most notably in vivo gene editing, which recently got a major boost from Intellia's positive first-in-human data. But all of these companies are advancing their gene therapies at a time when safety concerns are dogging developers of first-generation products. read more By Heather Landi For its first healthcare accelerator program, Amazon's cloud division tapped a diverse group of startups working in virtual reality, voice assistant technology, remote monitoring and social determinants of health. read more Resources Sponsored by: WCG Every biopharma sponsor faces the challenge of initiating, running and closing trials on time, but oncology studies pose several unique hurdles. Sponsored By: Unlearn AI Read Unlearn’s whitepaper to learn about how novel trial designs with Digital Twins enable more efficient clinical trials, with higher power and smaller required sample sizes. Sponsored by: Patheon by Thermo Fisher Scientific Choose the right AAV platform for your viral vector-based therapy Sponsored by: XIFIN inc. Download the Value of Precision Medicine Informatics (PMI) Initiatives report to learn about the technology, data types, and the value derived from more than 100 PMI initiatives. Sponsored by: EVERSANA Today’s economics do not allow manufactures to keep making decisions in an antiquated way. They need to challenge tradition & deliver faster, cheaper, successful launches. Learn how. Sponsored by: Thermo Fisher Scientific Thermo Fisher Scientific introduces a new NK cell culture medium designed to support feeder-free, high-yield growth for cell therapy applications. Sponsored by: Cascade Chemistry by Aceto Read four customer success stories and four success factors that helped move pharmaceutical customers of Cascade Chemistry by Aceto quickly and effectively to clinic with trial-ready APIs. Sponsored by: Box Read Get to Know Cloud Content Management for Life Sciences and learn how to eliminate silos across all workflows, from R&D to commercialization. Sponsored by: Catalent Learn about drug product process development challenges and risks, the difference between innovator and biosimilar drug product process development, and more during the drug product process development phase. Sponsored by: Catalent Get insights on key considerations for a successful tech transfer process for manufacturing a biologic drug product, including a case study on overcoming challenges in a process transfer for a sterile diluent. 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