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 is buzzing with billion-dollar M&A, growing momentum in partnerships across different indications, and promising gene therapy trial results.  

Happy Reading!  

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Cell Therapy

Umoja wins FDA clearance for first CD22-directed in vivo CAR-T | Regulatory

The FDA cleared Umoja Biopharma's IND for UB-VV400, described as the industry's first known CD22-directed in vivo CAR-T candidate, for adults with relapsed or refractory B-cell malignancies. Built on the company's VivoVec lentiviral platform, which reprograms a patient's T cells directly inside the body and bypasses the ex vivo manufacturing chain, the Phase 1/2 program targets patients who have progressed after prior CAR-T therapy, with first dosing expected in Q3 2026.

Repligen to acquire BioLife Solutions for $1.5B, deepening cell therapy manufacturing reach | M&A

Repligen agreed to acquire BioLife Solutions for a total enterprise value of approximately $1.5 billion in a cash-and-stock deal valuing BioLife at $31.00 per share. The deal brings BioLife's CryoStor biopreservation media, which supports 18 approved cell therapies, into Repligen's bioprocessing portfolio and is expected to close in the fourth quarter of 2026.

Allogene's allogeneic CD70 CAR-T ALLO-316 posts 31% response in CD70-high renal cell carcinoma | Clinical Trial

Allogene published full Phase 1 TRAVERSE data in the Journal of Clinical Oncology for ALLO-316, an allogeneic CD70-directed CAR-T therapy for heavily pretreated advanced clear cell renal cell carcinoma. Among 20 patients dosed in the Phase 1b cohort, the confirmed response rate was 25% overall and 31% in patients with a CD70 tumor proportion score of at least 50%, supporting the RMAT-designated program as early proof of concept for allogeneic CAR-T in solid tumors.

Fate Therapeutics reports first iPSC-derived CAR-T responses in systemic sclerosis | Clinical Trial

Fate Therapeutics presented preliminary Phase 1 data at ISSCR 2026 for FT819, an off-the-shelf, iPSC-derived CD19-directed CAR-T therapy, in treatment-refractory systemic sclerosis. All four patients treated as of the June 2026 cutoff showed meaningful skin-score improvement at three months with no cytokine release syndrome, ICANS, or graft-versus-host disease, and most were treated under reduced conditioning without fludarabine.

Allotera raises $35M to advance off-the-shelf CD7 CAR-T for T-cell cancers | Financing

Allotera Therapeutics, formerly Wugen, closed a $35 million round of equity and venture debt, bringing its Series C to $150 million. Proceeds support pivotal development of soficabtagene geleucel (Sofi-cel), a CD7-directed allogeneic CAR-T therapy for relapsed or refractory T-ALL and T-LBL, indications where shared target antigens complicate autologous manufacturing

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Gene Therapy

FDA approves Casgevy for young children with sickle cell disease | Regulatory

The FDA issued a supplemental approval extending Casgevy (exagamglogene autotemcel) to patients as young as 2 years old with sickle cell disease or transfusion-dependent beta thalassemia. This makes Casgevy the first gene therapy approved for sickle cell disease in this younger pediatric population, having previously been limited to patients 12 and older.

Sarepta Therapeutics appoints Michael Severino as CEO | Personnel

Sarepta Therapeutics named Michael Severino, M.D., as chief executive officer, effective July 28, 2026, succeeding Doug Ingram, who is retiring. Severino, most recently CEO of Tessera Therapeutics and previously Vice Chairman and President of R&D at AbbVie, also joins Sarepta's board.  

Ocugen signs binding term sheet for OCU400 MENA licensing deal | Partnership

Ocugen entered a binding term sheet for a regional partnership covering its AAV gene therapy OCU400 in the Middle East and North Africa, with Ocugen retaining manufacturing and commercial supply responsibilities. The deal is part of Ocugen's broader strategy to expand access to its retinal gene therapy pipeline, with a definitive agreement expected within 90 days.

Krystal Biotech discloses positive KB801 data for neurotrophic keratitis | Clinical Trial

Krystal Biotech disclosed in an SEC filing that a patient treated with its gene therapy KB801 experienced complete healing of a corneal defect, newly public data from a discontinued study that the company is advancing into a pivotal trial. The study was stopped not for safety or efficacy reasons but to move the program into a larger registrational trial.

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Oligo

AstraZeneca pays $30M upfront for CSPC's siRNA kidney platform | Partnership

AstraZeneca has entered a collaboration with CSPC Pharmaceutical Group worth up to $1.77 billion to discover preclinical siRNA candidates for two undisclosed kidney disease targets. CSPC receives $30 million upfront plus up to $540 million in development and $1.2 billion in sales milestones, while AstraZeneca gains an exclusive option for worldwide rights to one candidate.

Eplontersen misses primary endpoint in Phase 3 ATTR-CM trial | Clinical Trial

Ionis and AstraZeneca's Phase 3 CARDIO-TTRansform trial of eplontersen missed its primary composite endpoint of cardiovascular mortality and recurrent events in 1,432 ATTR-CM patients on standard of care. A prespecified monotherapy subgroup showed a nominally significant hazard ratio of 0.71, while patients on background stabilizers showed no benefit.

Biogen's tau-targeting ASO shows cognitive benefit in Alzheimer's | Clinical Trial

Biogen's Phase 2 CELIA study of diranersen, an antisense oligonucleotide targeting tau mRNA, showed its 60 mg dose slowed cognitive decline by up to 50% versus placebo at 18 months in 416 early Alzheimer's patients, alongside 50–65% reductions in CSF tau. The trial missed its primary dose-response endpoint, but Biogen will advance diranersen into Phase 3.

Arcturus taps Thermo Fisher for mRNA cystic fibrosis manufacturing | Manufacturing

Arcturus Therapeutics has signed a strategic collaboration with Thermo Fisher Scientific covering Phase 3 clinical supply and prospective commercial manufacturing rights for ARCT-032, its investigational mRNA therapy for cystic fibrosis. The deal leverages Thermo Fisher's Accelerator Drug Development platform to support the program through potential commercialization.

Ribo and Madrigal hit first candidate-nomination milestone in siRNA MASH program | Partnership

Suzhou Ribo Life Science and its subsidiary Ribocure Pharmaceuticals AB, together with Madrigal Pharmaceuticals, announced the first candidate drug nomination milestone under their siRNA collaboration targeting metabolic dysfunction-associated steatohepatitis (MASH). The companies will immediately begin IND-enabling studies to support planned clinical trials; financial terms of the underlying partnership were not disclosed.

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ADCs

Novartis to acquire Myricx Bio for up to $1.5B | M&A
Novartis has agreed to acquire ADC specialist Myricx Bio, centered on its novel N-myristoyltransferase inhibitor (NMTi) payload platform, which is designed to combat resistance to current payloads. The proposed acquisition could help Novartis build next-generation ADCs that overcome limitations across different solid tumors.  

FDA approves Padcev plus Keytruda for muscle-invasive bladder cancer regardless of cisplatin eligibility | Regulatory
The FDA approved enfortumab vedotin (Padcev) plus pembrolizumab (Keytruda) as neoadjuvant and adjuvant treatment for muscle-invasive bladder cancer, removing the prior cisplatin-eligibility restriction. This broadens the eligible patient population for the ADC-immunotherapy combination in the curative-intent setting, and is supported by Phase 3 evidece demonstrating a ~50% reduction in risk of tumor recurrence or progression versus standard of care.  

SystImmune's Iza-bren wins second China approval, for esophageal cancer | Regulatory
SystImmune announced a second regulatory approval in China for its ADC iza-bren, this time for recurrent or metastatic esophageal squamous cell carcinoma. The approval is supported by PANKU-Esophagus 01, which met primary endpoints of significance, such as a demonstrated overall survival of 9.8 months in iza-bren versus 7.2 months for chemotherapy.  

FDA grants Fast Track Designation to SOTIO's SOT109 for colorectal cancer | Regulatory

The FDA granted Fast Track Designation to SOT109, SOTIO's CDH17-targeting ADC, for colorectal cancer. The Phase 1/2 trial of SOT109 for advanced CRC patients is expected to begin in Q3 of this year, where the therapeutic targets an antigen expressed in most (~90%) of CRC cases.  

Kelun-Biotech's sac-TMT hits primary endpoint in first-line NSCLC | Clinical Trial
Kelun-Biotech announced that its Phase III study of sacituzumab tirumotecan (sac-TMT) combined with pembrolizumab (KEYTRUDA) met its primary endpoint as a first-line treatment for PD-L1-negative, advanced non-squamous NSCLC in OptiTROP-Lung06. This is the first Phase III trial to show that an ADC–immunotherapy combination is effective as a first-line treatment for PD-L1-negative advanced non-squamous NSCLC.

Lonza and Engitix license agreement to advance targeted ADC development | Partnership
Lonza and Engitix have entered a license agreement aimed at advancing targeted antibody-drug conjugate development, SYNtecan E™ linker-payload and GlycoConnect^® and HydraSpace^®. The deal pairs Lonza's manufacturing expertise with Engitix's ability to conduct R&D and drive commercialization.