At last month’s Meeting on the Mesa, ARM’s annual can’t-miss event, attendees were treated to insightful keynotes, panel discussions, and breakout sessions that covered all the major topics impacting the cell and gene sector. One such plenary discussion, moderated by Audentes’ Chairman and CEO, Matt Patterson, was particularly valuable in that the panelists offered insight on their companies’ path to commercialization.
I recently had an informative conversation with Dr. Jiwen Zhang, VP, Regulatory Affairs at Passage Bio to gain a better understanding of just how rapidly regulators must move to keep pace with the sector’s ever-evolving regulation landscape and what industry professionals should do to meet requirements. She shared with me the critical action items cell and gene therapy professionals should heed to keep pace with the sector’s ever-evolving regulations, which will be detailed in an upcoming article.
When it comes to partnering with a contract research organization (CRO), many pharmaceutical and biotech companies, particularly small ones, have no choice but to outsource. From crafting clearly defined policies and procedures to establishing non-negotiable requirements of a CRO partner, Sponsors select CROs meticulously and judiciously. I caught up with Bob Paccasassi, VP of Quality and Compliance operations, and Mandy Clyde, VP of Operations at BioRestorative Therapies for their insight on the best strategies for partnering with a CRO. Here’s what they had to say.
Curative cell & gene therapies are changing modern medicine as we know it, but are their high price tags fair to the patient?
Why patient advocacy is a crucial component in the on-going successful treatment of rare diseases.
Indeed, earlier this year at World Advanced Therapies & Regenerative Medicine (WATRM) Congress in London, it was predicted that manufacturing will be a major future differentiator between therapy companies. I spoke to Dr. Paul Lammers, MD, MSc, president, CEO, and Director of Triumvira Immunologics about the therapy pricing and manufacturing’s role in it.
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.
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