June was a major month for IPO filings, with more than 15 biotech companies filing in the last two weeks of the month alone. Several companies in the cell and gene therapy field were among them, including AVROBIO, a lentiviral gene therapy company looking to treat lysosomal storage disorders; Unum Therapeutics, which is developing an antibody-coupled T cell receptor for immuno-oncology use; and Magenta Therapeutics, which is looking to improve on bone marrow transplant therapy. Neon Therapeutics, which is working on neoantigen-targeted therapies including T cells, also filed an IPO in June. As Bruce Booth writes for Forbes, the industry has reached a tipping point. Signs of success — and enthusiasm for treatments — are increasing steadily.
As momentum grows, safety remains a top priority. Earlier in June, researchers at French biotech Cellectis described a new technology under development called CubiCAR. The method would enable easier purification of CAR-T cells in the laboratory, and would also build in a stopgap capability to eradicate the cells if an adverse treatment event occurs. CubiCAR is not the only research effort aimed at adding safeguards to CAR-T: FierceBiotech reports that Bellicum, Gilead and Boston University are all working on similar ideas.
June was also an exciting month for gene therapy. Bluebird Bio released positive data from clinical trials of LentiGlobin, which aims to treat beta thalassemia and sickle cell disease. And, two companies working on gene therapy treatments for Duchenne Muscular Dystrophy (DMD) gained momentum last month. Sarepta Therapeuticsreleased promising preliminary data on a small trial of three children that found lower levels of creatine kinase — an enzyme responsible for muscle damage — after treatment with AAVrh74.MHCK7.micro-Dystrophin, the Wall Street Journal reports. The FDA also lifted its clinical hold on Solid Biosciences’ experimental gene therapy after the first patient treated began showing positive results.
Finally, a new study in animal models generated buzz when it showed how gene therapy could be used to address spinal cord injuries. According to FierceBiotech, the treatment results in production of the enzyme chondroitinase, which helps restore connections between damaged nerves. Rats treated with the drug regained the use of their paws after two months — a promising step, the researchers say, toward using gene therapy to address an area of dramatically unmet medical need.