Recent Articles in Cell & Gene Therapy

A siRNA therapy developed by Shanghai Refreshgene Therapeutics enables children with genetic deafness to hear. In a China-US trial, six children aged one to seven were able to hear for the first time after a single dosing of an siRNA therapy that used an adeno-associated virus (AAV) to deliver a functioning copy of the human otoferlin gene to previously deaf children. The therapy was effective in five out of the six patients. In the five patients who responded, all were able to hear after a few months and also learned how to speak. The trial results were published in the Lancet on Wednesday. More details....

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SineuGene, a Beijing startup that is developing an RNA therapy for ALS, reported that its lead candidate, SNUG01, slowed the progression of ALS to a “plateau stage” in the first person dosed with the therapy. In addition, the patient’s lower limbs responded to electric stimulation, an indication that a nerve has at least partly rejuvenated. SineuGene is led by Yichang Jia, PhD, a Professor at Tsinghua University School of Medicine. The company has not disclosed many details about its research, but its lead candidate stems from his research into RNA metabolism abnormalities that cause ALS. SineuGene believes its technology may also be effective for two other brain maladies, Alzheimer’s and Parkinson’s. More details....

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Shanghai CARsgen Therapeutics was cleared to start a China trial of CT011, an autologous CAR T-cell candidate against Glypican-3 (GPC3) for hepatocellular carcinoma. Patients must have GPC3-positive stage IIIa disease with a high risk of recurrence following surgical resection. CT011 is an autologous GPC3 CAR T-cell product that was originally cleared by China’s NMPA in 2019 to treat patients with GPC3-positive solid tumors, the first China CAR T-cell therapy clearance for a solid tumor indication. CARsgen has completed enrollment of a China Phase I trial for the candidate. More details....

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Shanghai IASO Biotherapeutics announced an extensive collaboration with Seattle’s Umoja Biopharma to develop off-the-shelf, CAR T-cell gene therapies. IASO Bio will use Umoja’s rapamycin-activated cytokine receptor (RACR™) platform to develop two ex vivo iPSC-derived chimeric antigen receptor (CAR)-bearing cell therapies. IASO will be responsible for worldwide development and commercialization of both products. In exchange, Umoja will receive rights to CAR cassettes for two targets to advance two in vivo product candidates. Umoja will be responsible for product development outside of Greater China, and IASO for the Greater China territory. The two companies established their first collaboration in late 2022. More details....

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Suzhou Ribo Life Science formed a collaboration with Boehringer Ingelheim that will apply its siRNA expertise to develop novel treatments for nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH) in a deal that could be worth up to $2 billion. Ribo will receive an upfront payment plus milestones and royalties for developing multi-target candidates. The agreement will include Ribocure AB, a Swedish subsidiary of Ribo Life Science that develops siRNA molecules. Ribocure uses its vertically integrated oligonucleotide research platform to deliver new candidates efficiently. In 2022, it advanced three projects to clinical stage in 20-24 months from concept to implementation. More details....

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