Insights provided by DeciBio, a strategy consultancy focused on the life science and biopharma industry.

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Highlights & Summary

This month, the next-generation therapeutics space saw platform-scale licensing deals from large pharma, a first-ever approval in a rare metabolic disease, and a run of pivotal readouts that cut both ways, with two RNA-based programs hitting their endpoints and a third missing, alongside a high-profile gene therapy clinical hold.

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Cell Therapy

J&J partners with Sail Biomedicines on in vivo CAR-T with $2.58B buyout option | Partnership / M&A

J&J entered a collaboration with Sail Biomedicines to advance its in vivo CAR-T platform, which reprograms a patient's immune cells inside the body for immune reset in immune-mediated diseases. J&J will make $785 million in initial payments, including a $465 million equity investment, plus $140 million in development milestones, and holds an exclusive option to acquire Sail for $2.58 billion.

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Kyverna's CD19 CAR-T miv-cel wins RMAT designation in progressive MS | Regulatory

The FDA granted RMAT designation to Kyverna's mivocabtagene autoleucel (miv-cel), an autologous CD19-targeting CAR-T, for non-active secondary progressive multiple sclerosis. The designation rests on a Stanford Phase 1 trial in six patients showing CAR-T expansion in blood and cerebrospinal fluid plus naive B-cell reconstitution, extending miv-cel's neuroimmunology franchise beyond stiff person syndrome and myasthenia gravis.

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CARsgen's allogeneic BCMA and CD19/CD20 CAR-T programs win NMPA IND clearances | Regulatory

CARsgen received NMPA IND clearances for two allogeneic CAR-T candidates on its THANK-u Plus platform: CT0596 (BCMA-directed) for relapsed/refractory multiple myeloma, and CT1190B (CD19/CD20 dual-targeting) for relapsed/refractory large B-cell lymphoma. The clearances mark CARsgen's push into off-the-shelf CAR-T following June's approval of its autologous solid-tumor product satri-cel.

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Zelluna's ZI-MA4-1, the first MAGE-A4 TCR-NK therapy, clears initial safety review | Clinical Trial

Zelluna's Independent Data Monitoring Committee cleared the first patient dosed in the Phase 1 ZIMA-101 study, the first clinical test of its TCR-NK platform. ZI-MA4-1 was well tolerated with no dose-limiting toxicities, and the committee recommended enrolling the remaining two patients at Dose Level 1.

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Hopstem closes first round of Series C for iPSC-derived neural cell therapy | Financing

Hopstem Biotechnology closed the first tranche of a Series C, drawing new investors Tasly Capital, Haibang Investment, and Guangzhou Health Industry Investment alongside existing backer Longmen Capital. Proceeds will advance hNPC01, an iPSC-derived forebrain neural progenitor cell injection that recently hit a set of FDA milestones.

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Gene Therapy

Ultragenyx wins FDA approval for first GSDIa gene therapy | Regulatory
The FDA granted accelerated approval to Ultragenyx’s GENGLYCOS (pariglasgene brecaparvovec-opnr) for patients aged eight years and older with glycogen storage disease type Ia, making it the first approved therapy for the condition. Approval was based on reduced daily cornstarch requirements, with continued approval contingent on confirmatory evidence of clinical benefit; Ultragenyx also received a Priority Review Voucher.

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Epicrispr raises $90M Series C to advance FSHD epigenetic therapy | Financing
Epicrispr Biotechnologies closed an oversubscribed $90 million Series C financing co-led by Octagon Capital and Janus Henderson Investors. The proceeds will support advancement of EPI-321 toward pivotal studies in facioscapulohumeral muscular dystrophy, as well as expansion of the company’s broader epigenetic medicine pipeline; additional Phase 1/2 data are expected later in 2026.

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FDA places RGX-121 on clinical hold after spine MRI findings | Clinical Trial
REGENXBIO said the FDA placed RGX-121, its gene therapy for Hunter syndrome, on clinical hold after asymptomatic spinal MRI abnormalities were identified in five participants treated roughly three to six years earlier. The company no longer expects to resubmit the BLA in the near term and is reviewing additional imaging and longer-term follow-up data with partner NS Pharma before determining next steps.

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Opus Genetics completes enrollment in registrational OPGx-LCA5 trial | Clinical Trial
Opus Genetics announced completion of enrollment in its registrational trial of OPGx-LCA5, an AAV8 gene therapy for LCA5-associated inherited retinal disease. The program was accepted into FDA's Rare Disease Evidence Principles program earlier this year, and dosing of the registrational cohort is planned for Q4 2026.

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Taysha expands Catalent partnership for commercial manufacturing | Manufacturing
Taysha Gene Therapies and Catalent expanded their manufacturing agreement, naming Catalent as the primary commercial manufacturer for TSHA-102 if the Rett syndrome AAV gene therapy is approved. Taysha is now conducting BLA-enabling activities following completion of dosing in its REVEAL pivotal and ASPIRE trials.

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Oligos

Merck and Moderna's personalized mRNA melanoma vaccine hits Phase 3 endpoints | Clinical Trial

The Phase 3 INTerpath-001 trial of intismeran autogene (an individualized mRNA neoantigen therapy) plus Keytruda met both recurrence-free and distant metastasis-free survival endpoints versus Keytruda alone in resected Stage IIB-IV melanoma, it is the first regimen to show this benefit in the adjuvant setting.  

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Silence Therapeutics' divesiran hits primary endpoint in Phase 2 trial | Clinical Trial

Silence Therapeutics announced positive Phase 2 SANRECO topline results for divesiran, a first-in-class siRNA silencing TMPRSS6, in 48 phlebotomy-dependent polycythemia vera patients. The trial hit its primary endpoint with an 88% clinical response rate on divesiran versus 19% on placebo (p<0.0001), with both dosing schedules showing strong efficacy and no new safety findings; a Phase 3 trial is anticipated to begin in the first half of 2027.

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Arrowhead pays for a Priority Review Voucher to speed plozasiran filing | Regulatory

Arrowhead Pharmaceuticals signed an agreement to acquire an FDA Rare Pediatric Disease Priority Review Voucher for $215 million, intending to use it on an upcoming supplemental NDA for plozasiran (REDEMPLO) in severe hypertriglyceridemia. The move follows positive Phase 3 SHASTA-3/4 topline data and would cut the FDA's target review window from 10 months to six.

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Ionis and AstraZeneca's eplontersen misses primary endpoint in ATTR-CM trial | Clinical Trial

Full CARDIO-TTRansform data, presented at ESC Congress and published simultaneously in NEJM, confirmed the antisense therapy eplontersen (Wainua) did not significantly reduce the composite of cardiovascular mortality and recurrent events versus placebo in 1,432 ATTR-CM patients, despite a 77% placebo-corrected reduction in serum TTR. A prespecified subgroup not on background TTR stabilizers showed a nominally significant benefit, raising questions about whether RNA silencers should be positioned as monotherapy rather than add-on treatment.

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ADCs

DualityBio licenses novel-payload ADC platform to Genentech in deal worth over $1 billion | Partnership

Shanghai-based DualityBio granted Genentech a global license to build ADCs on its DUPAC platform, which uses payload mechanisms distinct from the topoisomerase I inhibitors that power Enhertu and Trodelvy. DualityBio will receive $45 million upfront and is eligible for more than $1 billion in milestones plus tiered royalties, with Genentech assuming clinical development and commercialization once Phase 1a finishes.

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Formosa Pharmaceuticals files for first Kadcyla biosimilar trial, targeting HER2-positive breast cancer | Clinical Trial

Formosa Pharmaceuticals and CDMO partner EirGenix filed a Clinical Trial Application with European regulators for TSY-110, a biosimilar referencing Roche's Kadcyla (ado-trastuzumab emtansine), which combines trastuzumab with a mertansine payload and generated roughly $2.5 billion in global sales in 2025. The pivotal trial, designed to satisfy both U.S. and EU guidance, will compare pharmacokinetics, safety, and immunogenicity against the reference product, positioning TSY-110 as a potential first-to-market ADC biosimilar.

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Natera pairs its Signatera liquid biopsy with Angiex's dual-targeting ADC in early trial | Partnership

Natera will apply its Signatera circulating tumor DNA test to Angiex's Phase 1 trial of AGX101, a TM4SF1-directed nuclear-delivered ADC designed to hit both tumor cells and tumor-associated blood vessels in patients with advanced solid tumors. The collaboration will use longitudinal ctDNA readings to help interpret ambiguous imaging results during treatment, an approach both companies hope can sharpen early response assessment for this novel ADC subclass.

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BioNTech taps Sobi's Guido Oelkers as CEO to steer ADC-led oncology pipeline | Personnel

BioNTech named Guido Oelkers, currently CEO of Sobi, as its incoming chief executive, with a mandate to push the company toward multiple approved oncology products by 2030. The most advanced near-term asset is trastuzumab pamirtecan, a HER2-targeted ADC licensed from China's DualityBio, which is progressing toward a potential approval filing alongside BioNTech's bispecific and mRNA immunotherapy programs.

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Whitehawk Therapeutics advances three-asset ADC portfolio on $190 million cash position | Financing / Clinical Trial

Whitehawk continued enrolling patients in Phase 1 trials of its PTK7-targeted ADC HWK-007 and MUC16-targeted ADC HWK-016, both built on its Carbon Bridge Cysteine Re-pairing conjugation platform, while a third candidate, HWK-206, remains on track to start dosing in small cell lung cancer and neuroendocrine tumors in the third quarter. The company holds $190 million in cash following an $87.5 million private placement in May, and separately secured an option to license payload technology from Hangzhou DAC for up to five future bispecific ADC programs.