The gene editing alliance between Sanofi and Sangamo Therapeutics has produced an experimental sickle cell disease treatment, which recently released encouraging early clinical data, but the pharmaceutical giant has decided to abandon the treatment and termination The entire agreement is part of its overall genomic drug strategy reform.
The right to cooperate with sickle cell therapy SAR445136 will be returned to Sangamo at the end of the alliance in the middle of this year. Sangamo, based in Brisbane, California, said Thursday that it plans to explore options for the plan, including finding new partners.
Sanofi inherited the Sangamo alliance after acquiring Bioverativ in 2018. The initial transaction paid Sangamo US$20 million in advance, and an additional US$276.3 million was associated with the milestone. The focus of the partnership is to use Sangamo’s zinc finger nuclease gene editing technology to develop new therapies for inherited hemoglobin diseases.
The sickle cell project is the most advanced project in the partnership. Sickle cell disease is an inherited disease that causes hemoglobin (the blood protein that carries oxygen) to appear crescent-shaped. Treatment includes blood transfusions. Sangamo’s SAR445136 is made by editing the patient’s stem cells to produce fetal hemoglobin, which may provide the patient with functional hemoglobin.
Sanofi’s termination notice to Sangamo was issued a few weeks after encouraging early data came up with At the annual meeting of the American Society of Hematology. So far, of the four patients treated, no one needs a blood transfusion, and total hemoglobin has stabilized in the 26th week after treatment. In addition, all four patients had an average increase in fetal hemoglobin. No adverse events related to the experimental treatment were reported.
However, competition in the field of genomic drugs for sickle cell disease is becoming increasingly fierce. Partners Vertex Pharmaceuticals and CRISPR Therapeutics are also adopting gene editing methods for this disease. last year, Vertex paid $900 million for greater responsibility and potential economic windfall Of cooperative therapy. Graphite Bio also uses CRISPR technology in its gene editing method for sickle cell disease; A clinical trial started at the end of last year. Beam Therapeutics is preparing to test its Base editing method After receiving a green light from the FDA recently, clinical trials for the disease began.
The methods used by Vertex, Graphite, and Beam are all autologous-cells are taken from the patient, edited in the laboratory, and then reinjected into the patient. Although Sangamo uses different editing techniques, cooperative therapy is also autologous. Sanofi may have seen that the field of autologous sickle cell therapy is too crowded, resulting in the decision to terminate the alliance with Sangamo despite the encouraging data so far.in a Regulatory filing, Sangamo stated that the pharmaceutical giant’s December 30 notice “shows that its termination is related to Sanofi’s change of strategic direction, focusing on allogeneic generic genomic medicine methods, rather than autologous personalized cell therapy.” Allogeneic therapy is Those can be produced and readily available, ready-made therapies.
According to the document, the termination will take effect on June 28. From now to then, Sanofi will transmit data, information and regulatory materials to Sangamo. The biotech company said that the phase 1/2 sickle cell study should be completed as planned, and the patient will eventually be given the drug in the third quarter of this year. Before the agreement officially ends in June, Sangamo said that Sanofi is expected to continue to pay for the research. Sanofi will withdraw from the alliance before it is responsible for making any large payments to Sangamo. The biotech company stated in the document that Sanofi has so far paid it a milestone payment of $13.5 million.
Sangamo’s pipeline includes other genomic drugs, some of which are collaborative. Under the alliance with Pfizer, gene therapy for hemophilia A is currently in an advanced stage of development. Projects targeting a range of conditions are undergoing preclinical development with partners including Novartis, Takeda Pharmaceuticals, Biogen and Gilead Sciences.
Image: Meletios Verras, Getty Images
