The US National Institutes of Health (NIH) has signed a cooperative research and development agreement (CRADA) with life sciences company MaxCyte to develop new treatments for sickle cell disease (SCD).
Under the agreement, NIH’s National Heart, Lung, and Blood Institute (NHLBI) will carry out pre-clinical research to investigate the safety and effectiveness of CRISPR/Cas9-based single-nucleotide correction to treat SCD.
The institute will correct the faulty haemoglobin gene responsible for SCD and target DNA mutations in non-corrected cells associated with the disease.
MaxCyte president and CEO Doug Doerfler said: “We are delighted to continue our collaboration with NHLBI, one of the world’s leading disease institutes, which is leveraging MaxCyte’s expertise in developing a new generation of potential treatments for SCD.
“We believe that this work will further validate our platform for developing gene-editing therapies for a broad range of diseases while enabling rapid, development and commercial manufacturing of new therapies for patients where there is an extremely high unmet medical need.”
MaxCyte is responsible for the supply of mRNA molecules and will use its Flow Electroporation Technology to create effective methods to generate clinically meaningful corrections in mutated gene sequences.
SCD represents a group of inherited red blood cell disorders that are characterised by abnormal haemoglobin levels.
Current statistics indicate that about 300,000 infants are born with the disease per year globally and the incidence is anticipated to rise rapidly.
In June last year, MaxCyte signed a separate CRADA with NIH’s National Institute of Allergy and Infectious Diseases for the development of X-linked chronic granulomatous disease therapies.