From The Editor | February 23, 2023

Cell & Gene: The Podcast Covers CGT Companies in the News


By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1


One of the best parts of hosting Cell & Gene: The Podcast is that I have the opportunity to talk to academic and industry leaders who, along with their colleagues, continue to make exceptional strides in bringing life-saving therapies to the patients who need them. So, when my expert guests and their respective companies are in the news for evidence of clinical benefit, launching a startup, commercial success, etc., I check back in to their episodes to see just how far they’ve progressed.

Sangamo Therapeutics

Sangamo recently announced evidence of clinical benefit in its Phase 1/2 STAAR study in Fabry disease. The Brisbane, CA-based biotech stated that data showed sustained, elevated expression of alpha-galactosidase A (α-Gal A) activity observed in 13 patients for over two years for the longest treated patient as of cutoff date. They achieved 78% globotriaosylceramide (Gb3) substrate clearance at 6-months and 77% reduction in urine podocyte loss in one of the first kidney biopsies. All dose escalation patients had been withdrawn from enzyme replacement therapy (ERT) and remain off ERT today.

I reached out to Nathalie Dubois-Stringfellow, Chief Development Officer at Sangamo Therapeutics for her take and for more information on the study. "Sangamo’s Phase 1/2 STAAR study is evaluating isaralgagene civaparvovec, or ST-920, for the treatment of Fabry disease,” she states. "Updated preliminary data being presented at the WORLDSymposium in Orlando Florida this week show promising results from biopsy to clinical benefit and with continued safety and efficacy across the thirteen patients in the dose escalation and dose expansion phases. Of particular note, we revealed the first kidney biopsy data from this study showing significant Gb3 substrate reduction, along with highly encouraging SF-36 general health score findings. We believe we have a potential best-in-class gene therapy, and we’re excited to continue to evaluate the treatment so we can deliver it to patients as quickly as possible.

We are actively preparing for a potential Phase 3 trial, which we expect to begin in the second half of 2023. Dosing of the first patient could happen as early as the first part of 2024, depending on regulatory interactions. The expansion phase of the Phase 1/2 study – for which we expect to complete dosing in 2023 – is not expected to be a gating factor for the commencement of Phase 3.”

The news comes on the heels of our most recent episode of Cell & Gene: The Podcast featuring Sangamo’s Andy Ramelmeier, EVP Head of Technical Operations. During the episode, which focused on the company’s decision to build its own Phase 1/2 cGMP AAV in-house facility, Ramelmeier explained the why behind that decision and the benefits the company has experienced as a result of the build. He also spoke broadly about manufacturing cell and gene therapies and the potential and realistic solutions to needed to drive down manufacturing costs.

Mustang Bio

Back in December, Mustang Bio, a clinical-stage biopharma focused on potential cures for hematologic cancers, solid tumors, and rare genetic diseases, announced its CAR-T cell therapy portfolio updates and provided anticipated milestones for 2023. President and CEO Dr. Manuel Litchman stated that in 2023 the company anticipates filing an IND for its combination therapy MB-109 to treat glioblastoma and that it will continue to collaborate with the Mayo Clinic to progress its exclusively licensed novel in vivo CAR-T technology platform and anticipate the publication of proof-of-concept research in a murine tumor model.

In January 2023, we featured Mustang Bio’s Robert Sexton, VP Program and Alliance Leadership, on episode 41 of Cell & Gene: The Podcast. During the episode, Sexton explained the company’s pipeline covered in greater detail its plans for MB-109 and in vivo CAR-T technology. He also explained the why behind the need for the VP Program and Alliance Leadership role in any CGT company. Having worked for Novartis, Sanofi, Legend Biotech, and more, with responsibilities having included quality, operations, and order management, Sexton is currently responsible for aspects of CMC. As such, he also addressed the industry's top CMC challenges as well as sound advice for future scale up.

Cartesian Therapeutics

Late last month, Gaithersburg, MD-based Cartesian Therapeutics announced its first placebo-controlled study of an engineered cell therapy and dosed the first participant in its Phase 2b randomized controlled trial (RCT) for generalized myasthenia gravis (MG), an autoimmune disorder that causes muscle weakness and fatigue. The RCT will evaluate the efficacy and safety of the company’s lead asset, Descartes-08, an RNA-engineered chimeric antigen receptor T-cell therapy (rCAR-T). Descartes-08 is administered over 6 weekly outpatient visits and requires no preconditioning chemotherapy.

This news brings me to episode 25, which featured Dr. Metin Kurtoglu, CEO at Cartesian. During our discussion, Dr. Kurtoglu explained the clinical-stage biotech’s autologous RNA cell therapies, Descartes-08 for Myasthenia Gravis and Descartes-11 for multiple myeloma. He also explained Descartes-25, their allogeneic RNA cell therapy and the reasons for the move to "off-the-shelf," and why the future of RNA cell therapy is not confined to rare and fatal diseases. We also discussed post-care for patients having received infusions. Listen in to the details that brought Cartesian to where it is today with Descartes-08.

Dr. Carl June

Dr. June is the Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine in the University of Pennsylvania’s Perelman School of Medicine and director of the Center for Cellular Immunotherapies in the Abramson Cancer Center and director of the Parker Institute for Cancer Immunotherapy at Penn. You may have heard that during Advanced Therapies Week in Miami last month, he was awarded the inaugural Phacilitate Lifetime Achievement Award for his pioneering work in the field of immunotherapy and CAR-T cell therapy.

Last year, I had the honor of featuring Dr. June on Cell & Gene: The Podcast on episode 28 to discuss the current state of CAR-T therapy, what's on the short-term horizon for immunotherapy, on-going manufacturing capacity issues, an update on Emily Whitehead, the first child to receive CAR-T cell therapy on an experimental basis, and more. Listen in; you’ll understand right away why Dr. June received the inaugural Phacilitate Lifetime Achievement Award.

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