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Issue 200

The year 2023 was a difficult one for many in the sector. Funding has dried up and operating costs are higher than ever. But it never pays to dwell on the negatives, so instead let us look to the year ahead with excitement for new technologies and what they may bring to patients. CRISPR, AI, and new treatments for solid tumors are just some of the compelling topics I will be keeping my eyes on. 

I hope 2024 brings success to you and your companies – and better health (or even cures) to your patients. If you’d like to share your thoughts on what lies ahead for cell and gene in 2024, please get in touch: [email protected]

I’d also like to remind you all about nominations for The Medicine Maker 2024 Power List, which will close on January 12. The Power List aims to showcase inspirational and influential individuals in the pharmaceutical industry across three categories of drug development, including cell and gene. Nominating someone (or yourself – it’s OK, we won’t judge you!) is simple – just fill out the quick form on this page. The 2023 list included medicine makers from big pharma, medium pharma, biotechs, CDMOs, consultancy firms, AI specialists, and more! Check out the 2023 list here.

Until next week,

Stephanie Sutton | Group Editor


Essential Reading

Preventing ischemia-induced amputations
Researchers from Cincinnati Children’s Hospital Medical Center and Kanazawa University have demonstrated a new method to promote blood vessel growth to help prevent ischemia-induced amputations. Their work involved stem cells isolated from adipose tissue carrying the cell surface marker CD271, which were transplanted into mice with limb ischemia. In all of the treated mice, amputation was not required. The study was inspired by a small clinical trial in humans with foot ulcers. Patients were treated with a mix of stem cells. Only two patients had fully recovered limbs and both received high numbers of CD271-positive stem cells.

Understanding your cell culture media
How much do you understand about the role of cell culture media when it comes to gene therapy manufacturing? In this article, an expert from Thermo Fisher Scientific explains the logistical impact that cell culture media can have on development. “As workflows scale up and liquid media volumes increase, so too do the logistical and financial challenges of shipping and storage. Careful planning and forecasting are required so that media are available when needed and used before expiration. However, liquid media are heavy, which means they can be expensive to ship and challenging to move around facilities,” writes Elpidia Gamez. Read more


Worth Your Time...

Research

Scientists at Baylor College of Medicine describe RNA-based switch to regulate gene expression, according to patient’s specific needs.

Review article examines potential of hydrogel and nanofibrous scaffolds supplemented with stem cells for treating central nervous system.

Research from Japan shows how canine iPSCs can be created from urine-derived cells using painless method without need for feeder cells.


Business

FDA adds black box warning to CAR T therapy Carvykti for secondary hematological malignancies; myeloid neoplasms noted in 10 percent of patients treated.

Voyager Therapeutics and Novartis agree collaboration and license agreement for Voyager’s Tracer capsids; companies aim to advance gene therapy for Huntington’s disease.

AstraZeneca to acquire Gracell Biotechnologies, including clinical-stage autologous BCMA/CD19 CAR T and proprietary manufacturing platform.

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