Adaptimmune’s SPEAR (Specific Peptide Enhanced Affinity Receptor) T-cell platform enables the engineering of T-cells to target and destroy cancer across multiple solid tumors. The FDA has granted Orphan Drug Designation (ODD) to SPEAR T-cells targeting MAGE-A4 (Adaptimmune’s ADP-A2M4 program) for the treatment of soft tissue sarcomas. Orphan Designation by FDA was created to encourage the development of drugs for rare diseases, such as sarcomas. I caught up with Dr. Elliot Norry, interim Chief Medical Officer at Adaptimmune, to learn more about the designation’s impact on the treatment of soft tissue sarcomas.
Patient advocacy plays a tremendously crucial role in the cell and gene sector. Jamie Ring, Head of Patient Advocacy at Spark Therapeutics and Cell & Gene Editorial Advisory Board member shared with me valuable information about role of patient advocacy in industry (i.e. what it is; what it’s not; the difference between patient advocacy in healthcare overall versus patient advocacy in gene therapy), which we will cover in detail in the coming weeks. To start, Ring lists and explains the three pillars of the role of patient advocacy in industry.
Dilated cardiomyopathy (DCM) is a condition that affects more than 3 million patients in the U.S. Recently subpopulations have been identified that develop DCM due to mutations in specific genes that have been shown to result in the development of DCM. One of these specific genes is the Bcl2-associated athanogene 3 (BAG3) gene.
I reached out to Dr. Jude Samulski, co-founder and chief scientific officer of AskBio, to learn more AskBio's recent acquisition of Synpromics and to better understand just how the end-to-end AAV gene therapy platform will affect and improve gene therapy in the United States.
Allogeneic cell therapy products are displaying encouraging clinical and pre-clinical results. Precision BioSciences — a genome editing company known for its proprietary ARCUS genome editing platform, recently announced the opening of its Manufacturing Center for Advanced Therapeutics (MCAT), the first in-house current Good Manufacturing Process (cGMP) compliant manufacturing facility in the United States dedicated to genome-edited, off-the-shelf CAR-T cell therapy products. I caught up with Matt Kane, Chief Executive Officer and co-founder of Precision BioSciences to learn more about the manufacturing center and what that means for commercial launch.
In Part 1 of our two-part series with Adaptimmune, CEO James Noble, who has announced his retirement since the publication of this article, provided thoughtful insight on the status of combatting solid tumors as well as the ongoing pricing issue affecting the cell and gene therapy sector. Here in Part 2, along with John Lunger, SVP, Manufacturing and Supply Chain, and Mark Dudley, SVP, Product Development at Adaptimmune, we dig deeper into the topics discussed in Part 1 — we shed granular light on how pricing will affect manufacturing; we talk outsourced versus in-house manufacturing and more.
On the heels of our sister print publication, Life Science Leader’s cover story on the whirlwind rise of Orchard Therapeutics, I had the opportunity to talk to company’s Co-Founder and Chief Scientific Officer, Dr. Bobby Gaspar about how the company has developed innovative gene therapies to treat CNS diseases, such as metachromatic leukodystrophy (MLD), tackle ADA-SCID, and more.
Minovia Therapeutics recently announced dosing of the first patient in a Phase I/II clinical trial of the company’s Mitochondrial Augmentation Therapy (MAT) for the treatment of Pearson syndrome. As stem cell transplantations and cellular therapies continue to gain momentum rapidly, this is an example of trial that could pave the way for the development of treatments for all mitochondrial diseases.
Erin Harris, Editor-In-Chief, Cell & Gene conferred with several SMEs in the cell and gene sector to get their take on the challenges for both industry and patient associated with cell and gene therapy prices, and what can be done, realistically, to bring costs down over time.
James Noble, CEO of Adaptimmune, explains where the industry is with attacking solid tumors and sheds light on the ever-present pricing issue.