Recent Articles in Cell & Gene Therapy

Jiangsu Ractigen Therapeutics, a novel oligonucleotide biopharma, has dosed the first patient in the first-in-human trial of rag-17, an siRNA candidate, that enrolled ALS patients with sod1 mutation. Rag-17 is a siRNA candidate designed to provide potent and durable knockdown of the sod1 protein in the central nervous system to prevent degradation of motor neurons and delay disease progression. Although delivery to tissue has been historically difficult for siRNA candidates, Rag-17 uses a novel smart chemistry-aided delivery (scad) conjugate that facilitates broad biodistribution and tissue uptake throughout the CNS.

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Suzhou Innovent and Nanjing’s IASO Bio presented positive long-term follow-up results of a Phase Ib/II trial of their partnered BCMA CAR T-cell therapy in relapsed and/or refractory multiple myeloma patients. The data, which was released at the ASCO meeting, showed equecabtagene autoleucel achieved an overall response rate of 96%. 91% of the patients showed at least a very good partial response, and 74% had either a stringent complete response or a complete response. The enrolled patients, who had previously been treated with at least three rounds of treatment, were refractory to their previous therapy round. More details....

Stock Symbol: (HK: 01801)

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Legend Biotech, a Nanjing-New Jersey CAR-T company, announced positive results from a US Phase III trial of its lead drug, cilta-cel, as an earlier treatment for multiple myeloma. The trial showed cilta-cel reduced disease progression by 74% compared to standard treatment in a trial that enrolled patients who had previously received one to three prior therapies. Cilta-cel is a BCMA autologous T-cell drug that shocked the world in 2017 with near 100% efficacy in multiple myeloma. It is already approved in China, the US and EU, though only after as many as four earlier treatment rounds. More details....

Stock Symbol: (NASDAQ: LEGN)

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Shanghai Gracell Biotech reported its dual-targeting BCMA/CD19 FasTCAR-T candidate showed efficacy in heavily pre-treated patients with relapsed/refractory multiple myeloma. The median duration of response was 37 months in patients who had previously received a median of five therapy rounds, and the median progression free survival was 38 months. FasTCAR-T, Gracell’s CAR-T technology, produces an autologous CAR-T treatment overnight instead of nearly a month. The single-arm, investigator-initiated trial enrolled 29 RRMM patients. Gracell released the data for GC012F, its lead candidate, in an abstract at the ASCO meeting. More details....

Stock Symbol: (NSDQ: GRCL)

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Shanghai CARsgen Therapeutics has started enrolling patients in a US Phase II trial of CT041, a CAR-T candidate, in patients with CLDN18.2 positive advanced gastric cancer/gastroesophageal junction cancer (GC/GEJ). CT041 is an autologous CLDN18.2 CAR T-cell candidate that, CARsgen believes, could be the first-in-class globally. CARsgen’s lead drug, a BCMA CAR-T-Cell therapy, is currently under NDA review in China for relapsed/refractory multiple myeloma. The company develops CAR-T therapies for hematological and solid tumor cancers in China and the US. More details....

Stock Symbol: (HK: 2171)

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