Recent Articles in Cell & Gene Therapy

Beijing CANbridge Pharma reported positive data from its second-gen gene therapy for spinal muscular atrophy in preclinical tests. In a mouse model of SMA, a novel second-gen hSMN1-AAV gene therapy vector improved life span and motor function better than the benchmark vector, The therapy was delivered via intracerebroventricular (ICV) injection, consisting of an endogenous SMN1 promoter and codon-optimized human SMN1 transgene. CANbridge, which developed the novel therapy together with UMass Chan Medical School, develops rare disease and rare oncology therapies. More details....

Stock Symbol: (HK: 1228)

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Chengdu’s Keymed Biosciences partnered with Shanghai Rona Therapeutics to discover first-in-class siRNA therapeutics for severe kidney diseases, also known as Glomerulonephritis. Rona will use its proprietary RAZOR™ platform to discover and identify siRNA lead compounds aimed at a novel glomerulonephritis target, and Keymed will contribute biologic research and translational expertise. The collaboration will build on Rona's proprietary platform for RNAi targeting of liver and extrahepatic tissues. The two companies have signed a profit/loss agreement to develop and commercialize candidates from the collaboration, though details were not disclosed. More details....

Stock Symbol: (HK: 02162)

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Gracell Biotechnologies, a Shanghai-San Diego CAR-T company, reported its dual-targeting BCMA/CD19 autologous FasTCAR will begin a China investigator-initiated trial for refractory systemic lupus erythematosus (SLE). The FasTCAR platform prepares a clinical dose overnight. GC012F, a next-gen CAR-T CD19/BCMA candidate, has already started trials for two multiple myeloma indications and B-cell non-Hodgkin's lymphoma. The new SLE trial is the first time GC012F has been tested in patients with an autoimmune disease. More details....

Stock Symbol: (NSDQ: GRCL)

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Guangzhou Reforgene Biotechnology closed a $15 million Pre-B financing for its portfolio of gene editing candidates, including a clinical stage therapy for thalassemia. In a Phase I trial, the company’s lead β-thalassemia gene-editing drug, RM-001, produced a 100% cure rate. In addition, Reforgene is developing in vivo gene editing therapies for major genetic diseases in the fields of ophthalmology, nervous system and other fields. The financing was led by Yuexiu Industrial Fund with participation by Yuanyin Venture Capital, Shanfeng Venture Capital, Xinxing Fund, Hong Kong and Guangdong Capital, and Guoju Venture Capital. More details....

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Suzhou GenAssist Therapeutic reported the US FDA accepted an IND for its novel in vivo gene editing candidate as a treatment for Duchenne muscular dystrophy (DMD). GEN6050 is a base editing drug that is designed to restore the expression of dystrophin protein that is caused by exon 50 skipping in the DMD gene. GenAssist’s TAM BE (Targeted AID-based Mutagenesis) base editing technology was discovered by one of its Founders, Xing Chang, PhD, who now serves as the company’s CSO. DMD is a rare disease that occurs once per 4000 newborn boys. Exon 50 skipping can be used to treat 4% of DMD patients with large deletions. More details....

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