FDA Clears Way for Trial of Gene Editing Therapy for Severe SCD – Sickle Cell Anemia News

The U.S. Food and Drug Administration (FDA) has cleared the initiation of a Phase 1/2 trial investigatingEDIT-301, Editas Medicines experimental gene editing cell therapy for sickle cell disease (SCD).

The planned open-label study, to be called RUBY, will assess the safety and efficacy of a single dose of EDIT-301 in patients with severe SCD.

The FDAs clearance for initiation for our EDIT-301 clinical trial is an exciting moment for us and the patients we hope to serve. We look forward to bringing this potentially best-in-class, one-time, durable medicine into the clinic and to patients, Cindy Collins, president and CEO of Editas Medicine, said in a press release.

The FDA clearance, however, applies only to the therapy being testing in the studys safety phase.

An improved potency assay must be developed by the company and submitted to the U.S. agency for review before the trial can begin enrolling patients into its efficacy phase. Usually, treatment effectiveness is investigated once safety is shown in a first patient group.

Potency assays are tests, often requested by regulatory health authorities, to evaluate the ability a treatment to induce a specific effect when given at a particular dose.

Editas reported that it has started preparations to launch RUBY, including identifying a principal investigator to lead the study and engaged a clinical research organization to support its regulatory procedures.

EDIT-301 is an experimental one-time gene editing cell therapy that modifies a patients blood cell precursors (hematopoietic stem cells) to increase the production of fetal hemoglobin in red blood cells. Fetal hemoglobin,a version of hemoglobin found in newborns, is more efficient at transporting oxygen than its adult counterpart.

This experimental therapy, which edits cells taken from a patient before being returned to them, is the first to use the gene editing tool CRISPR/Cas12a (also known as CRISPR-Cpf1) and targets two regions in the genome, theHBG1andHBG2 promoters.

By increasing the production of fetal hemoglobin, EDIT-301 may provide long-term benefits to those with SCD, including improved blood flow and fewer pain crises.

We know patients are counting on us, and we believe EDIT-301 has the potential to transform the lives of people living with sickle cell disease, addressing a significant unmet need, Collins said.

The FDA granted rare pediatric disease designation to EDIT-301, a status given to investigative therapies for serious or life-threatening conditions affecting fewer than 200,000 people in the U.S., and mainly those under age 18.

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that made up the lining of blood vessels found in the umbilical cord of newborns.

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Ins holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Cincias e Tecnologias and Instituto Gulbenkian de Cincia.Ins currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.

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FDA Clears Way for Trial of Gene Editing Therapy for Severe SCD - Sickle Cell Anemia News