Outsmarting cancer with RNA, 'genome-tuning' drugs and other gene-altering therapies With first-in-human trial results, Intellia shows the world that gene editing has arrived Blackstone, Cellex and Intellia form $250M CAR-T startup Sponsored: Balancing Quality Grade Plasmids and Expedited Timelines for Advanced Therapies Gilead's Kite makes off-the-shelf play with Shoreline Biosciences CAR-NK deal worth as much as $2.3B Bluebird bio gets FDA green light to restart sickle cell gene therapy trials after rocky few months Esco raises $200M to set up Boston cell and gene therapy hub Adaptimmune’s cell therapy shrinks 39% of rare sarcomas Otonomy eyes clinical trial of hearing loss gene therapy with promising animal data Umoja banks $210M to bring next-gen tumor tagging immunotherapies into the clinic J&J's anti-BCMA CAR-T pads its case ahead of speedy review and Bristol Myers showdown Featured Story | By Arlene Weintraub Startups and academics are working on novel treatments designed to turn the expression of certain genes up or down to achieve an anti-cancer effect. It’s not gene therapy in the traditional sense, because they’re not replacing disease-causing genes, but if they succeed, they could offer new ways to conquer "undruggable" cancer targets. read more |
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Top Stories By Annalee Armstrong Interim results are in for Intellia and partner Regeneron’s in vivo CRISPR/Cas9 genome editing candidate, NTLA-2001, in patients with transthyretin (ATTR) amyloidosis: and the numbers look good. This is the first time gene editing has been proven to work in humans, which “opens up a whole new area of therapies for patients that wasn't there.” read more By Nick Paul Taylor Blackstone Life Sciences, Cellex Cell Professionals and Intellia Therapeutics have teamed to create a CAR-T therapy startup. The biotech begins life with $250 million from Blackstone, universal CAR-T platforms from Cellex and CRISPR/Cas9 genome editing technology from Intellia. read more Sponsored By Catalent Cell & Gene Therapy The key to success in developing and bringing mRNA-, DNA-, viral vector- and cell-based therapies and vaccines to market is having a partner like Catalent with a global reach and multiple solutions for clients. The Catalent team works with its partners to look for ways to streamline the development path to help meet expedited timelines. read more By Amirah Al Idrus Gilead’s Kite unit made its name developing autologous CAR-T treatments made from a patient’s own cells. Now, the subsidiary is making another big play in off-the-shelf treatments that can be developed more quickly in a deal with Shoreline Biosciences that could exceed $2.3 billion. The partners will initially work on CAR targets for natural killer cell therapies for blood cancers. read more By Amirah Al Idrus The FDA lifted a clinical hold it placed on bluebird bio’s sickle cell gene therapy in February, making way for the company to get a phase 1/2 trial and phase 3 study of the treatment back on track. Bluebird paused the trials in mid-February after two patients in the phase 1/2 study developed blood cancer after treatment. read more By Nick Paul Taylor Esco Lifesciences has raised $200 million in a series A and crossover round co-led by Novo Holdings and Vivo Capital. The Singapore-based company will use the cash to establish a hub for cell and gene therapy tools and technologies in Boston while expanding in China and making bolt-on acquisitions. read more By Amirah Al Idrus CAR-T and other cell therapies have seen success in blood cancers but have shown limited effect in solid tumors. Adaptimmune is taking an engineered T-cell receptor approach to change that and its first phase 2 data, from patients with rare soft-tissue sarcomas, look promising. The MAGE-A4-targeting treatment, afamitresgene autolecel, shrank tumors in 39.3% of the patients. read more By Angus Liu Otonomy may have hit a snag with development of its lead ear disorder candidate Otividex, but the biotech is trumpeting an early win for a gene therapy designed for congenital hearing loss caused by mutations in the GJB2 gene. The treatment reduced hearing loss and repaired structural damage in the inner ear in two mouse models. read more By Amirah Al Idrus Umoja Biopharma emerged last November with $53 million and a mission to push past the limits of today’s immunotherapies. Now, the company is raising another $210 million to get its lead programs in bone cancer and blood cancer into the clinic. read more By Amirah Al Idrus Just days after moving their multiple myeloma CAR-T into the FDA’s fast lane, Johnson & Johnson and Legend Biotech are unveiling new data to pad its case. The treatment shrank tumors in 98% of very sick patients with multiple myeloma and eliminated them in 80% of patients. read more Resources Sponsored by: Catalent Learn how product characteristics, formulation, and patient needs impact device selection and manufacturing processes – and how experienced partners can help. Sponsored by: Thermo Fisher Scientific Learn how the diagnostics industry is being fundamentally transformed with point-of-care molecular testing. Sponsored by: Clinical Ink For faster deployment, improved patient engagement, and better-quality data — go where your patients are. GO BYOD with Lunexis™ from Clinical Ink. 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 SDG Group Discover how Ashfield Healthcare uses the power of data and analytics to enable pre-call planning, inform sales rep actions and drive higher market share for its target pharmaceutical products. Sponsored by: BBK Worldwide Create a more diverse and inclusive clinical trial enrollment and engagement environment by learning the barriers to participation and the strategies to overcome them. 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. Sponsored by: Thermo Fisher Scientific Autologous cell and gene therapy workflows involve isolating cells from an individual, engineering the cells, expanding and concentrating them, and infusing them back into the patient. Optimized automation of certain steps of the workflow may decrease hands-on time and the cost of the cell manufacturing process. Sponsored By: Oracle Health Sciences New research indicates an accelerated adoption of decentralized clinical trials due to COVID-19. Diversity, Equity & Inclusion Week August 9-11, 2021 | Virtual Event Clinical Quality Oversight Forum September 27-29, 2021 | Philadelphia, PA | 