Weekly reads: Gamida Cell, Siddhartha Mukherjee, CRISPR in viruses

Hematopoietic stem cells have many clinical applications and a company called Gamida Cell has an interesting product in this area called Omidubicel that may have several applications.  I’m going to start the weekly reads with news there.

Gamida Cell and the FDA

A recent piece discussed how Omidubicel is progressing with the FDA.

FDA extends deadline to decide on Gamida Cell’s stem cell transplant therapy by three months, Fierce Biotech. The product here is Omidubicel, which is also called NiCord. It’s an NAM-enabled cell therapy for blood and other cancers with potential utility in additional diseases. This is an allogeneic hematopoietic stem cell transplant hoped to lead to better reconstitution than a typical cord blood unit would. More specifically, I think that Omidubical is intended to spark more effective transplanted cell engraftment and recovery after chemo. The FDA extension here and request for more data will push things back a few months.

The Phase III trial paper in Blood on Omidubicel seems encouraging. From the study:

Omidubicel (Gamida Cell, Jerusalem, Israel) is a patient-specific cell product derived from a single banked UCB unit. It consists of an ex vivo expanded CD133⁺ fraction and a nonexpanded CD133^– fraction. Nicotinamide, the active agent in the culture system, inhibits differentiation and enhances functionality of cultured hematopoietic stem and progenitor cells. Preclinical studies demonstrated that when UCB-derived hematopoietic progenitor cells are cultured in the presence of nicotinamide and stimulatory hematopoietic cytokines, there is an outgrowth of phenotypically primitive CD34⁺CD38^– cells and a substantial increase in bone marrow (BM) homing and engraftment potential.

I found four trials using Ombidubical on Clinicaltrials.gov.

More regenerative medicine recommended reads

• How to Save a Life, NYT. This guest piece by Delia Ephron focuses on how a hematopoietic stem cell donor likely saved her life. She had AML, an often-fatal type of blood cancer, and needed a transplant. It’s an interesting read.
• In a first, a fatal enzyme deficiency is treated in the womb, STAT News. We are seeing the development of more cutting-edge in utero  It’s great to try to fix a problem during development so there’s less time for the condition to cause organ damage. In this case, the procedure prevented damage to the kiddo’s heart. I think we’ll see more gene editing therapies tested during pregnancy as well.
• A nice interview at STAT with Siddhartha Mukherjee on his work and his latest book on cells. I’m still trying to get a copy of this new book and can’t wait to read it.
• CRISPR is so popular even viruses may use it, Science. The research paper from Jennifer Doudna’s lab: Diverse virus-encoded CRISPR-Cas systems include streamlined genome editors, Cell.