C&G Editor in Chief, Erin Harris

1. What To Expect From Cell Therapy And Gene Therapy In 2022 And Beyond 12/2/2021

   From innovations in clinical development and manufacturing to the evaluation of investigational cell and gene therapies (CGTs), subject matter experts from Cellectis and Immatics share a brief look back on 2021 and a detailed glimpse into the near future of gene therapy and cell therapy respectively.

2. Inside Carisma Therapeutics’ CT-0508 11/23/2021

   Last year, I caught up with Michael Klichinsky, Ph.D. co-founder and VP of discovery and research at Carisma Therapeutics to discuss its then preclinical findings indicate that CAR-M therapy could overcome the key challenges that cell therapies have encountered with solid tumors — limited trafficking to the tumor site, an immunosuppressive tumor microenvironment, and the heterogeneous expression of tumor-associated antigens. In that article, among other things, Dr. Klichinsky and I discussed the challenges the cell and gene therapy sector faces when it comes to solid tumors. He explained that while it is clear that CAR-T cells are very good at killing hematologic malignancies (for example, B-cell acute lymphoid leukemia and B-cell non-Hodgkin lymphomas) in patients, they have a hard time against solid tumors.

3. Inside Novome Biotechnologies’ Phase 1 Study of NOV-001 11/22/2021

   Novome Biotechnologies recently announced positive results from a Phase 1 study of orally administered NOV-001 in healthy volunteers. The Phase 1 study demonstrated the ability to safely colonize the human gut with a therapeutically engineered microbe and control its abundance via once-daily dosing of a prebiotic control molecule. Based on these results, Novome intends to commence a Phase 2a study to evaluate preliminary efficacy in patients with enteric hyperoxaluria.

4. CGTs for Heart Disease 11/17/2021

   Although several conventional small-molecule treatments are available for common cardiovascular problems, cell and gene therapies are a potential treatment option for acquired and inherited cardiovascular diseases that remain with unmet clinical needs. 

5. Inside Canavan Disease With BridgeBio’s Dr. Eric David 10/29/2021

   Canavan disease is rare genetic neurological disorder characterized by the spongy degeneration of the white matter in the brain. Affected infants may appear normal at birth, but usually develop symptoms between 3-6 months of age. According to National Organization for Rare Diseases (NORD), most affected children develop life-threatening complications by 10 years of age. Canavan disease occurs because of mutations in the aspartoacylase (ASPA) gene that affects the breakdown (metabolism) of the N-acetylaspartic acid (NNA). It is inherited as an autosomal recessive condition.

6. Cell & Gene: The Podcast Breaks Down Patient Engagement With Novartis Gene Therapies 10/29/2021

   If you haven’t had the opportunity to listen to Episode 9 of Cell & Gene: The Podcast, be sure to do so, as Novartis Gene Therapies’ VP of U.S. Patient Advocacy and Government Affairs and Cell & Gene Editorial Advisory Board member, Dr. Amy Nicole Nayar, thoughtfully covers why Cell & Gene readers do what they do every single day — the patient.

7. Cellectis, Immatics On COVID-19’s Impact 10/26/2021

   In Life Science Leader’s upcoming annual Outlook issue, I contributed an article featuring Cellectis’ CEO André Choulika and Immatics’ Director, Global Site Operations, Michael Mehler about their take on the future of gene therapy and cell therapy respectively. In addition to the forward-looking insight included in that article, I also asked them how COVID-19 has impacted their companies, which I will share with you here. Here’s what they had to say about how the pandemic has affected their companies. And, watch your mailbox for the December 2021 issue of Life Science Leader magazine for the full-length outlook article featuring Choulika and Mehler.

8. Is A Fully Functioning Cell Therapy Global Logistics Network Achievable? 10/18/2021

   Expanding a GMP network, including a cold chain and adequate storage, is critical for cell and gene developers to reach previously unreached areas. 

9. A Look Into Cell & Gene: The Podcast Episode 7 Featuring Spark Therapeutics 9/30/2021

   A few months back, I wrote an article about Hemophilia A and how Spark Therapeutics’ administration of SPK-8011 in patients with hemophilia A resulted in an acceptable safety profile with no deaths and no FVIII inhibitor development with up to 4 years of follow-up.

10. Inside CARISMA Therapeutics’ Fast Track Designation By The FDA 9/26/2021

    Last year, I wrote an article about CARISMA Therapeutics’ foundational technology evaluating the potential of human chimeric antigen receptor macrophages (CAR-M) for cancer immunotherapy. CARISMA Therapeutics is making waves again with its recent news that it has been granted Fast Track Designation by the FDA to its CT-0508 for the treatment of patients with solid tumors. CARISMA’s Chief Medical Officer, Dr. Debora Barton, and I connected right after the announcement about the unmet needs of patients living with solid tumor cancer and how CARISMA’s CAR-Ms combine a unique set of characteristics, which they believe are the key to success in the treatment of solid tumors.