Thinly traded Magenta Therapeutics (MGTA +14.7%) is up more than double normal volume, albeit on turnover of only 211K shares, on the heels of encouraging preclinical data on CD117-ADC, its most advanced program for the preparation of patients undergoing stem cell transplantation or gene therapy. The results were presented yesterday at ASH in Orlando.
An NIH scientist presented data that showed the first-ever successful transplant of gene-modified cells in non-human primates without chemo or radiation preconditioning via the use of a targeted single-agent antibody-drug conjugate (ADC). Chemo and radiation are typically used to kill disease-causing cells before treatment, a paradigm saddled with severe side effects like toxicity and infertility.
A single dose of CD117-ADC achieved the same level of depletion as four doses of chemo agent busulfan, enabling the successful engraftment and persistence of stem cells with the β-globin gene, mutations of which cause sickle cell disease and β-thalassemia, without the toxic side effects. Vector copy number was stable beyond three months.
The single dose of CD117-ADC depleted hematopoietic stem cells in the animals while sparing immune cells.
#ASH19
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