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The United States gene editing technology market is expected to experience significant growth in the coming years, according to the biotechnology research firm BioIntel360. The report predicts that the market will record a compound annual growth rate (CAGR) of 28.2% during the period from 2023 to 2027, which will result in the market size reaching US$8,493.9 million by 2027. This growth will be driven by increasing demand for gene editing technologies in the healthcare sector, particularly for the development of personalized medicines and the treatment of genetic disorders.
A genetic blood ailment called sickle cell disease is brought on by a beta-globin gene mutation that causes sickle haemoglobin (HbS) to polymerize. Red blood cells typically have a disc shape, but in sickle cell disease, they are distorted into a sickle shape. Anaemia, pain crises, organ failure, and early death are all brought on by the red blood cells' irregular shape, which also shortens their lifespan and prevents blood flow. According to estimates, 100,000 Americans today suffer from sickle cell disease. Higher amounts of foetal haemoglobin (HbF) prevent HbS polymerization and hence lessen sickling symptoms.
Recently on April 27, 2023, the FDA designated the investigational gene-editing drug EDIT-301 for the treatment of sickle cell disease as an orphan drug, according to a statement from the genome editing company Editas Medicine.
EDIT-301 comprises patient-derived CD34+ hematopoietic stem and progenitor cells altered by a highly effective and unique AsCas12a nuclease at the gamma-globin gene (HBG1 and HBG2) promoters, which are home to naturally occurring foetal haemoglobin (HbF) generating mutations. For persons with severe SCD and TDT, red blood cells made from EDIT-301 CD34+ cells show a sustained increase in foetal haemoglobin synthesis, which may offer a one-time, long-lasting therapeutic effect.
In a recent press release, Editas stated that the therapy was well-tolerated, that both patients were effectively engrafted, and that they had not experienced any vaso-occlusive events throughout the follow-up. The RUBY study included two patients, who were reported in a December 2022 update to have achieved promising results. After five months, the first patient had a total haemoglobin level of 16.4 g/dL and a foetal haemoglobin content of 45.4%. By the end of this year, Editas intends to dose 20 people for this trial.
The FDA's Orphan Drug Designation program grants orphan designation to drugs or biologics created for the management, detection, or prevention of disorders that affect less than 200,000 individuals in the United States. Sponsors of drugs with Orphan Drug Designation are entitled to several benefits, including tax deductions for appropriate clinical trials, exemptions from prescription drug user fees, and a possible seven-year marketing exclusivity period upon FDA approval.
Patients with acute sickle cell disease (RUBY trial, NCT04853576) and transfusion-dependent beta-thalassemia (EDITHAL trial, NCT#05444894) are the subjects of a clinical study looking at EDIT-301. Two times this year, at the halfway point and the conclusion of the year, Editas Medicine will give clinical data updates from the RUBY experiment. Additionally, by year's end, the Company expects to have dosed 20 patients in the RUBY trial.
The company has previously declared a focus on in vivo research and a shift towards creating therapies for hemoglobinopathies such as sickle cell disease and beta-thalassemia. In a press release on January 9, Editas said that the company would stop investing in its medicines for inherited retinal illnesses and lay off about 20% of its personnel.
This specifically means that the business discontinued funding the development of EDIT-101 for the treatment of Leber Congenital Amaurosis and EDIT-103 for the treatment of rhodopsin-associated autosomal recessive retinitis pigmentosa. In addition, Editas has halted its investments in the development of its NK cell program intended for treating solid tumors, which includes potential candidates such as EDIT-202. This decision was made by the company and is reflected in its recent activities. Furthermore, on January 19, 2023 Editas announced that it has signed a binding agreement to license its natural killer cell program to Shoreline Biosciences, indicating a shift in focus for the company's research and development efforts. This Agreement included the acquisition of EDIT-202 by Shoreline.
