Exciting advancements are happening every month in the cell and gene therapy industry and July was no exception.
A novel gene therapy for a glycogen storage disease (GSD) was tested in humans for the first time late last month, UConn Today reports. The rare genetic disease causes dangerously low blood sugar levels, to the point that patients need to ingest glucose every few hours. Developed by the California biotech Ultragenyx, the investigational gene therapy aims to wean patients off the glucose regimen by delivering a functional copy of a gene crucial to regulating blood sugar levels. If it works in humans as it has in animal models, the therapy should jump-start the patient’s glucose control mechanisms and stabilize blood sugar levels.
Another innovative trial involves a gene therapy for Gaucher disease, a serious condition in which the body lacks a key enzyme to break down the lipid glucocerebroside, which researchers hope to cure in utero. A study published last month in Nature Medicine reported that scientists at University College London successfully transferred a corrective gene into a fetal mouse model of Gaucher disease. Treating the animals in utero yielded far greater benefit than treating them shortly after birth; in many respects, those treated in the womb were indistinguishable from healthy newborn mice, researchers reported.
There is more work to do in animal models, but scientists hope to eventually test in utero gene therapies in humans. The most likely scenario: Deploying the therapies to treat diseases so disabling that they cause irreversible organ damage during fetal development, rendering postnatal treatment ineffective.
An article in the national health publication STAT captured the growing excitement around engineering T regulatory cells, or Tregs, to treat autoimmune disease. “I think genetically engineered Tregs are likely to be a next wave in cellular immunotherapy,” Dr. Everett Meyer of Stanford Medical Center told STAT.
Last month gene therapy company Sangamo Therapeutics announced plans to buy a French company, TxCell, that focuses on engineering Tregs to treat autoimmune diseases. According to BioPharmaDive, the deal is worth roughly $84 million.
The U.S. FDA has issued a slew of draft guidance documents on human gene therapies. The documents are intended to shape industry standards in arenas such as outcomes data reporting on adverse events and conducting long-term observational studies. Additional documents offered more specific guidance for companies developing and testing therapies for hemophilia, rare disease and retinal disorders.
The FDA’s Center for Biologics Evaluation and Research is accepting comments on the guidance documents before finalizing them.