C&G Editor in Chief, Erin Harris

1. More On SPEAR T-Cell Platform, Initial Responses In Four Solid Tumor Indications 1/13/2020

   At JPM Healthcare Conference, Adaptimmune reported two confirmed Partial Responses (PRs) — one in a patient with liver cancer and one in a patient with melanoma. The company also reported two unconfirmed PRs — one in a patient with gastro‑esophageal junction cancer and one in a patient with head and neck cancer. These data further confirm the potential of Adaptimmune’s SPEAR T-cell platform for patients with multiple solid tumors. Data were previously reported showing compelling efficacy with ADP-A2M4 in synovial sarcoma.

2. The State Of Genomic Testing In Blood Cancers 1/2/2020

   The Leukemia & Lymphoma Society’s Beat AML Master Trial found that patients treated with precision medicine had lower early death rates and improved overall survival compared to the standard of care. In this study, Foundation Medicine’s FoundationOne Heme test enabled the precision medicine approach that led to significantly longer overall survival rates in Beat AML-treated patients compared to those who elected standard therapy. I caught up with Jeffrey Venstrom, MD, SVP, Clinical Development at Foundation Medicine, to learn more about the current status of genomic testing in blood cancers. Here’s what he had to say. 

3. First-In-U.S. Trial Of CRISPR-Edited Immune Cells 12/10/2019

   According to last month’s announcement, the first-in-U.S. trial of CRISPR-edited immune cells for cancer patients suggest safety of approach. Genetically editing a cancer patient’s immune cells using CRISPR/Cas9 technology, then infusing those cells back into the patient appears safe and feasible based on early data from the first-ever clinical trial to test the approach in humans in the United States.

4. Challenges, Short-Term Outlook Facing the Cell and Gene Sector’s Regulatory Landscape 12/9/2019

   Here are the critical action items cell and gene therapy professionals should heed to keep pace with the sector’s ever-evolving regulations.

5. Takeaways from LSPA’s CRO & CMO Summit 11/27/2019

   Last week I attended Life Sciences Pennsylvania’s (LSPA) CRO & CMO Summit, which was held at West Pharmaceutical Services. For those of you unfamiliar with it, LSPA is the trade association for the Commonwealth’s life sciences community. LSPA represents biotechnology, medical device, diagnostic, pharmaceutical, research, and investment entities, along with many service providers that support the industry.

6. Part 2: Payment Over Time, Buy And Bill from Meeting On The Mesa 11/19/2019

   In Part 1 of our recap of last month’s Meeting on the Mesa’s plenary discussion moderated by Audentes’ Chairman and CEO, Matt Patterson, panelists offered insight on their companies’ path to commercialization. Dr. Vijay Chiruvolu, SVP Global Process Development – Cell Therapy at Kite; Rachelle Jacques, CEO of Enzyvant; Dr. Dave Lennon, President of AveXis; Ron Philip, Chief Commercial Officer at Spark Therapeutics; and Jeff Walsh, Chief Strategy Officer at bluebird bio participated in the discussion.

7. Pricing Reimbursement Recap From Meeting On The Mesa 11/14/2019

   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.

8. Keeping Pace With The Regulation Landscape 10/22/2019

   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.

9. Best Practices In Selecting A CRO 10/8/2019

   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.

10. Potential Solutions To Current Pricing Models For Cell And Gene Therapies 10/7/2019

    Curative cell & gene therapies are changing modern medicine as we know it, but are their high price tags fair to the patient?