蔡磊带动中国渐冻症药物研发提速，但钱呢？蔡磊团队：今年融资依然很难

Core Insights - The ALS (Amyotrophic Lateral Sclerosis) drug development landscape in China is evolving, with significant advancements in research and clinical trials, but challenges remain in funding and commercialization [1][2][9] Group 1: Drug Development Progress - The first "cause-targeted" ALS drug, Tofersen, has been approved in China, filling a gap for SOD1-ALS patients and aligning domestic treatment options with international standards [2][3] - Multiple ASO (Antisense Oligonucleotide) therapies are in early clinical stages, including Ionis Pharmaceuticals' Ulefnersen, and domestic companies like Zhongmei Ruikang are developing siRNA therapies [3][4] - Since 2025, there have been numerous drug development advancements in the ALS field in China, with at least 15 new drug candidates reported [6][7] Group 2: Funding Challenges - The ALS drug development sector is primarily driven by small and medium-sized enterprises, facing significant funding challenges due to the high costs and risks associated with early-stage research [9][10] - Initial funding for ALS projects is often reliant on external investments, as many startups lack mature products to generate revenue [10][11] - Despite the overall interest in innovative drugs, the ALS sector has seen limited large-scale financing, with only a few companies achieving significant funding rounds [10][12] Group 3: Research and Regulatory Environment - The complexity of ALS, including unclear etiology and heterogeneous patient populations, complicates drug development and increases the risk of early-stage failures [11][12] - Companies are advocating for improved regulatory frameworks to expedite drug approvals and support for rare disease research, including tax incentives and the establishment of specialized funds [12][13] - Collaborative efforts are suggested to create national registries and databases to enhance research and data sharing among stakeholders [12][13]