Gene Therapy Pioneer Jim Wilson Comments on iECURE's Announcement of Complete Clinical Response for ECUR-506
"This illustrates the amazing power of modern day science and the importance of persistence."
PHILADELPHIA, Jan. 9, 2025 /PRNewswire/ -- Today, Dr. Jim Wilson, President and CEO of GEMMA Biotherapeutics ("GEMMABio") and co-founder of iECURE commented on the company's announcement of preliminary findings of complete clinical response for the first infant to receive treatment for Ornithine Transcarbamylase Deficiency (OTCD) with ECUR-506:
"Thirty-five years ago, the first grant I received from the National Institutes of Health (NIH) was to research gene therapy for children born with the lethal form of OTCD that affects neonates. After decades of work, we eventually developed a gene therapy product with Ultragenyx that in clinical trials has shown promise in treating adults with the less severe form of OTCD. However, preclinical models indicated that gene therapy was unlikely to work in the more severely affected forms of OTCD.
"Therefore 10 years ago we pivoted to genome editing which should allow durable gene engraftment in the dividing liver of the infant. We were able to advance our lead genome editing product into the clinic through iECURE, a biotechnology company that I founded in 2021.
"We were highly encouraged by the preliminary report of the first OTCD infant that received our gene editing product who appears to have had a complete clinical response. While there is much more that needs be done before this can be declared a true clinical success, this illustrates the amazing power of modern day science and the importance of persistence.
"My sincere gratitude to everyone who supported us over this 35 year journey and the many scientists and collaborators that contributed to this work. I also want to thank Joe Truitt and the team at iECURE for their outstanding efforts in bringing this product into the clinic. GEMMABio looks forward to supporting iECURE in the further development of this program and the application of this technology to other lethal liver metabolic diseases. "
About GEMMABio
GEMMABio is a therapeutics company focused on speeding the research of and global access to life-changing advanced therapies for those living with rare diseases. The company will provide research and product development functions to bring gene therapy discoveries from the bench to the bedside faster and affordably.
GEMMABio is led by gene therapy industry pioneer Jim Wilson and his team of experts, who previously conducted their work in academia. Wilson is also the Chairperson of Franklin Biolabs, a Contract Research Organization. The collective goal of the two affiliated companies is to translate innovative scientific work into clinical trials, to then commercialize and distribute the new therapies around the world to patients who need them most.
For more information, please visit gemmabiotx.com.
Contact: Kristen Love
klove@briancom.com
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SOURCE GEMMA Biotherapeutics