ARTICLES BY ERIN HARRIS
Novartis’ T-Charge Platform Spotlights Auto CAR-Ts6/23/2022
You don’t have search long and hard for news in the CGT sector about companies concentrating on developing and manufacturing allogeneic, or off-the-shelf- therapies. Take Poseida Therapeutics for example. The broad platform technology company boasts auto and allo CAR T-cell therapies for oncology and beyond as well as in vivo and ex vivo gene therapies. In Episode 26 of Cell & Gene: The Podcast, Poseida Therapeutics’ CEO, Mark Gergen, and I talked about the clinical-stage biopharma’s plan to develop allogeneic versions of all of its hematological and solid tumor product candidates.
Precision Biosciences’ Race To Allo CAR-T Therapy Approval6/8/2022
Back in 2020, Precision Biosciences’ Brent Rice, PhD, Director, Manufacturing Science and Technology and Damien Hallet Senior Director, CMC Operations and Strategic Planning wrote an article for Cell & Gene about how successful manufacture of allogeneic CAR T therapies depends on a gene editing technology that maintains the integrity of the T cells and ensures routine, reliable, and consistent production of CAR T batches with the goal of generating 100 doses or more from a single leukopak.
Rocket Pharma’s Phase 2 For RP-L2015/31/2022
Rocket Pharma’s new positive long-term data from its Phase 1/2 trial of lentiviral-based gene therapy RP-L201 for the treatment of Leukocyte Adhesion Deficiency-I (LAD-I). RP-L201 is being developed for LAD-I, a rare genetic immune disorder that predisposes patients to recurrent and fatal infections, often resulting in death within the first two years of life.
CGT Delivery Challenges5/27/2022
Mass General Brigham recently revealed its “Disruptive Dozen,” 12 emerging cell and gene therapy (CGT) technologies with the greatest potential to impact health care in the next several years. The “Disruptive Dozen” results from interviews of 50 Mass General Brigham senior Harvard faculty followed by a rigorous selection process to identify the 12 most likely to have significant impact on patient care in the next few years.
XyloCor, Encoded, Ultragenyx, And SQZ Share News At ASGCT 25th Annual Meeting5/18/2022
The ASGCT 25TH Annual Meeting, May 16-19 in Washington, D.C., has delivered valuable insight to its attendees, who have the option to attend in person or virtually. Plenary sessions featured some of the most prominent luminaries in the field including AskBio’s Dr. Kathy High and NIH’s Dr. Francis Collins. As expected, CGT biotechs have brought their most recent and ground-breaking news to share with attendees. Here’s some of what’s been delivered at the event as well as Cell & Gene’s editorial coverage on the company’s sharing their exciting news.
Mass General Brigham’s 2022 World Medical Innovation Forum5/6/2022
Mass General Brigham’s 2022 World Medical Innovation Forum, in partnership with Bank of America, was held May 2-4 in Boston, and just as in previous years, this year’s event was chock full of valuable, innovative content. From the main stage panel discussions to “The Dr. Is In” lunch-and-learn-style sessions, luminaries from academia and industry shared valuable and innovative data on the future of cell and gene therapies from the future of CRISPR to safe and efficacious treatment of solid tumors to life-saving therapies for rare diseases.
Lineage Cell Therapeutics’ CEO Talks Clinical Trials, In-House Manufacturing4/29/2022
Lineage Cell Therapeutics’ CEO, Brian Culley, and I discussed the clinical-stage biotech’s existing plans for developing novel cell therapies for unmet medical needs. We discuss their cell therapy currently in clinical development for patients with acute spinal cord injuries as well as their cell therapy platform in development for cancer and why manufacturing large-scale amounts of whole cells consistently and within specifications is far more complex than manufacturing a single compound. Read on to get Culley’s take on the company’s near-term future.
Inside Mustang Bio’s Phase 1/2 Clinical Trial Of MB-1064/27/2022
Mustang Bio recently reported a favorable safety profile suitable for outpatient treatment and a high complete response rate, including patients with Waldenstrom macroglobulinemia and those who received prior CD19-directed CAR T therapy. The company expects to dose the first patient in a multicenter clinical trial this quarter. I caught up with Manuel Litchman, M.D., President & CEO, Knut Niss, Ph.D., CTO, Bruce Dezube, M.D., SVP & Head of Clinical Development at Mustang Bio to better understand their Phase 1/2 clinical trial of MB-106 as well as the company’s other ongoing clinical trials, and more.
Opus Genetics, Resilience Partner On Ocular Gene Therapy4/13/2022
Opus Genetics has partnered with Resilience for the development and manufacturing of AAV-based gene therapies for inherited retinal diseases. Opus’ first two programs in LCA target the LCA-5 and RDH12 genes and are licensed from the University of Pennsylvania and based on the work of Opus scientific cofounder, Jean Bennett, M.D., Ph.D., the F.M. Kirby Emeritus Professor of Ophthalmology at the Perelman School of Medicine. Opus’ third program is licensed from Massachusetts Eye and Ear / Harvard and is designed to address NMNAT1-associated retinal degeneration.
Innovation In CAR T Cell Therapy Manufacturing3/31/2022
Penn Medicine recently announced that a study led by Center for Cellular Immunotherapies researchers Michael C. Milone, M.D., Ph.D., an associate professor of Pathology and Laboratory Medicine and Saba Ghassemi, Ph.D., a research assistant professor of Pathology and Laboratory, published their pre-clinical study in Nature Biomedical Engineering on generating non-activated CAR-T cells in less than 24 hours.