2026年第二季度基因、细胞和RNA治疗前景报告

Q2 2026 Quarterly Data Report Contents Introduction The second quarter of 2026 marked a renewed acceleration in regulatory activity across thegene, cell, and RNA therapy landscape, with eight new approvals globally following acomparatively subdued first quarter. Five gene therapies received marketing authorization,including treatments for hearing loss, cancer, and limb ischemia. Moderna's mCombriaxbecame the latest approved RNA therapy in the European Union for seasonal influenza andCOVID-19, while Japan authorized two non-genetically modified cell therapies forepidermolysis bullosa and meniscal injuries. Together, these approvals reflect continueddiversification across therapeutic modalities and indications, bringing global totals to45approved gene therapies (including genetically modified cell therapies),38 RNA therapies,and 77 non-genetically modified cell therapies. Innovation also accelerated during the quarter, with 11 new mRNA-encoded CAR-T celltherapies entering development, matching the total reported across all of 2025. This rapidexpansion signals growing confidence in next-generation engineering approaches designedto improve the scalability and accessibility of cell therapies. Cartesian Therapeutics' PhaseIII candidate, Descartes-08, remains the most advanced program in this emerging modality. Industry activity strengthened alongside scientific progress. Total dealmaking increased15%quarter over quarter to 118 transactions—the highest quarterly total in the past year—while earlystage financing rebounded sharply, with20 companies raising a combined$500.4million in seed or Series A funding. Following the disciplined portfolio optimizationobservedin late 2025 and the steady regulatory progress seen in Q1, Q2suggests the fieldhas regained momentum across regulatory, scientific, and investment fronts, reinforcingcontinued confidence in the long-term growth of advanced molecular therapies. David Barrett,JD CEO, ASGCT Key takeaways from Q2 2026 •5 gene therapies were approved:•Regeneron’s Otarmeni (lunsotogene parvec) in the US for hearing loss •Curocell’s Rimqarto (anbalcabtageneautoleucel) in South Korea for diffuse large B-cell lymphoma and primary mediastinal large B-cell lymphoma•Helixmith’s Huasuoling (donaperminogene seltoplasmid) in China for limb ischemia•Oncolys BioPharma’s Telomelysin (suratadenoturev) in Japan for esophageal cancer•CARsgen Therapeutics’ Satri-cel (satricabtageneautoleucel) in China for gastric/gastroesophageal junction adenocarcinoma Eight new approvalsacross the gene, cell, andRNA categories tookplace in Q2 2026 •1 RNA therapy was approved: Moderna’s mCombriaxin the EU for seasonal influenza (flu) and COVID-19 •2 non-genetically modified cell therapies were approved:•Ishin Pharma’s Allostem (nivadostrocel) in Japan for epidermolysis bullosa•Fujifilm’s Savyscus(synovial mesenchymal stem cell therapy) in Japan for meniscal injuries •Q2 2026 saw 11 new non-genetically CAR-T cell therapies engineered using mRNA-encoded chimeric antigen receptors in T cells -matching the total number reported across the entirety of 2025 •These 11 new therapies are being developed across 12 companies, with only ParcelBio associated with more than one of these newassets•The most advanced mRNA-encoded CAR-T therapy to date is Cartesian Therapeutics’ Descartes-08, which is in Phase III trials for thetreatment of myasthenia gravis Accelerating growth ofnon-genetically modified,mRNA-engineered CAR-Tcells •All deal types increased over the previous quarter to reach 118 transactions in Q2 2026, a 15% jump, and represented a quarterlyhigh within the last year•Start-up financing also had a strong showing with 20 companies raising an aggregate $500.4 million in seed or Series A funding,a54% and 29% increase in volume and value, respectively, over Q1 2026•Serapha Bio, one of several companies to announce a reverse merger in Q2, completed the largest Series A round at $138 million,which will be used to develop itsin vivo base editing therapy SERP-01 (licensed from YolTech) for alpha-1 antitrypsin deficiency Deal activity foradvanced moleculartherapy companiesrebounded in Q2 2026 Key highlights in Q2 2026 Approved gene, cell, and RNA therapies Globally, for clinical use: • 46 gene therapies have been approved (including geneticallymodifiedcell therapies)-Regeneron’s Otarmeni (lunsotogene parvec) has been approved in the US for hearing loss-Curocell’s Rimqarto (anbalcabtageneautoleucel) has been approved in South Korea for diffuse large B-celllymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL)-Helixmith’s Huasuoling (donaperminogene seltoplasmid)has been approved in China for limb ischemia-CARsgen Therapeutics’ Satri-cel (satricabtageneautoleucel) has been approved in China forgastric/gastroesophageal junction adenocarcinoma-Oncolys BioPharma’s Telomelysin (suratadenoturev) has been approved in Japan for esophageal cancer• 37 RNA therapies have been approved-Moderna’s mC