C&G Editor in Chief, Erin Harris

1. CGT Delivery Challenges 5/27/2022

   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.

2. XyloCor, Encoded, Ultragenyx, And SQZ Share News At ASGCT 25th Annual Meeting 5/18/2022

   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.

3. Mass General Brigham’s 2022 World Medical Innovation Forum 5/6/2022

   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.

4. Lineage Cell Therapeutics’ CEO Talks Clinical Trials, In-House Manufacturing 4/29/2022

   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. 

5. Inside Mustang Bio’s Phase 1/2 Clinical Trial Of MB-106 4/27/2022

   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.

6. Opus Genetics, Resilience Partner On Ocular Gene Therapy 4/13/2022

   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.

7. Innovation In CAR T Cell Therapy Manufacturing 3/31/2022

   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.

8. Carisma Therapeutics, Novartis, And Contract Manufacturing 3/31/2022

   Philadelphia-based Carisma Therapeutics, known for its novel CAR-M technology, which was developed by and licensed from scientists at the University of Pennsylvania, has partnered with Novartis for the manufacturing of the biopharma’s HER 2 targeted CAR-M cell therapy. Clinical manufacturing is planned to begin in 2023. Steven Kelly, President and CEO at Carisma Therapeutics and I sat down to talk more about Carisma’s HER2 targeted CAR-M therapy as well as the decision to ink a contract manufacturing partnership with Novartis.

9. RNA’s Delivery Challenges 3/16/2022

   Targeted delivery is a major hurdle for effective RNA Therapeutics, a hurdle that must be overcome to broaden the application of clinical translation of cell therapeutics. The mRNA-based therapeutic modality is new to emerging field of immuno-oncology, often referred to as immunotherapy.

10. How To Win The Fight For Talent In The CGT Sector 3/11/2022

    With the recent explosion of companies in the biotech space, talent has become highly coveted. Companies are clamoring for the same skilled employees, and poaching has been and still is a real problem. And yet, it is difficult to find talent with the right amount of experience in that while there are plenty of experienced professionals, they often lack specific CGT knowledge. Or, when you do find people with CGT expertise, they are junior to the processes. Indeed, there is an increasing level of complexity and new technologies are changing the requirements for new hires with more diverse technical and scientific skills required.