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

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Highlights & Summary

May next-generation therapeutics incorporate pivotal data updates from conferences such as ASCO.

In Cell Therapy, Obsidian Therapeutics presented Phase 2 data on OBX-115, showing a 67% ORR in 15 pretreated ICI-refractory melanoma patients. In Gene Therapy, Torque Bio presented preclinical data for TQB-001, a kidney-targeted AAV gene therapy designed to treat autosomal dominant polycystic kidney disease caused by PKD2 mutations. For Oligos, GSK reported positive Phase 3 data for bepirovirsen, an ASO for chronic hepatitis B  In ADCs, Phase 2 data from an ongoing Phase 2/3 trial (NCT06879145) showed that SHR-A2102  combined with PD-L1 inhibitor adebrelimab in the perioperative setting produced a 71.4% ORR in MIBC patients with measurable lesions.

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Cell Therapy

Obsidian Therapeutics Presents Phase 2 Data for Engineered TIL Therapy in Melanoma at ASCO | Clinical Trial

Obsidian presented Phase 2 data on OBX-115 at ASCO showing a 67% ORR in 15 heavily pretreated ICI-refractory melanoma patients, with 80% of responses ongoing at median 4.3-month follow-up and no high-dose IL-2 required. The company has aligned with FDA on a single-arm accelerated approval pathway and plans to open a registration-enabling cohort in mid-2026.

Cartesian Therapeutics Secures Financing to Fund Phase 3 Trial of BCMA CAR-T in Myasthenia Gravis | Financing

Cartesian closed a $150 million credit facility with K2 HealthVentures on May 26, extending runway into 2028 and through topline Phase 3 data for Descartes-08 in generalized myasthenia gravis, expected Q1 2027. Descartes-08 is a BCMA-directed mRNA CAR-T administered outpatient without preconditioning chemotherapy, and is the only CAR-T in autoimmune disease designed around a non-integrating vector.

Kelonia's In Vivo BCMA CAR-T Receives FDA Fast Track Designation | Regulatory

Kelonia announced FDA Fast Track Designation for KLN-1010, its in vivo BCMA CAR-T therapy for relapsed/refractory multiple myeloma, and presented updated Phase 1 inMMyCAR data at ASCO showing a 100% ORR with MRD-negative bone marrow responses across 18 patients dosed using its iGPS lentiviral platform. The program requires no preconditioning chemotherapy or ex vivo manufacturing, with the first patient treated remaining in deep remission beyond 10 months.  

CREATE Medicines Raises Series B to Advance In Vivo CAR Pipeline In Autoimmune Disease and Oncology | Financing

CREATE Medicines closed a $122 million Series B on May 14, co-led by ARCH Venture Partners, Newpath Partners, and Hatteras Venture Partners, to advance its mRNA-LNP in vivo CAR platform, which programs immune cells directly inside the body without viral vectors or ex vivo manufacturing. Lead programs include CRT-402, a repeat-doseable CD19-targeted in vivo CAR-T for autoimmune disease, and a dual CD19 x BCMA program, with the company reporting the largest in vivo CAR clinical dataset to date at over 50 dosed patients.

Fate Therapeutics Presents Positive Phase 1 Data For Its iPSC-Derived CAR-T in SLE | Clinical Trial

Fate Therapeutics presented Phase 1 data at ASGCT on May 11 showing that FT819, its off-the-shelf CD19-targeting iPSC-derived CAR-T, achieved SRI-4 responses in all 3 evaluable SLE patients treated without conditioning chemotherapy, with 2 of 3 reaching lupus low disease activity state. FT819 is manufactured from a clonal iPSC master bank, enabling consistent off-the-shelf supply without the apheresis and patient-specific manufacturing that constrain autologous programs.

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Gene Therapy

Complement Therapeutics Doses First Patient in Phase I/II GA Trial | Clinical Trial

Complement Therapeutics announced dosing of the first patient in the Phase I/II Opti-GAIN study evaluating CTx001, an AAV2 gene therapy for geographic atrophy (GA) secondary to age-related macular degeneration. CTx001 delivers a mini-CR1 transgene designed to modulate both the classical and alternative complement pathways, and the study will assess safety, tolerability, and preliminary efficacy following a single subretinal administration.  

iECURE Reports Reduced Hyperammonemic with ECUR-506 | Clinical Trial

iECURE presented interim data from the OTC-HOPE trial showing that treatment with ECUR-506, an in vivo gene insertion therapy for neonatal-onset OTC deficiency, was associated with a 52% reduction in annualized hyperammonemic crisis rates. Across seven treated infants, 71% experienced no hyperammonemic crises after treatment, while the therapy demonstrated a generally manageable safety profile and provided early proof-of-concept for clinical activity.  

REGENXBIO Reports Positive Pivotal Data in DMD | Clinical Trial

REGENXBIO announced positive topline results from the pivotal portion of the AFFINITY DUCHENNE trial of RGX-202, with 93% of patients achieving the primary microdystrophin expression endpoint at Week 12. The company also reported statistically significant correlations between microdystrophin expression and one-year functional improvements, alongside a generally favorable safety profile, and plans further discussions with the FDA regarding a potential accelerated approval pathway.  

Sangrail Launches with late-stage MPS IIIB Gene Therapy | Company Creation

Newly launched Sangrail Biologics highlighted updated clinical data for SNG-101, an AAV9 gene therapy for Sanfilippo syndrome type B (MPS IIIB). Across 14 treated patients, the program demonstrated sustained biomarker improvements, normalization of NAGLU enzyme activity, reductions in heparan sulfate levels, and stabilization or improvement of neurocognitive outcomes in younger patients, with the company planning regulatory interactions in the second half of 2026 to discuss a potential approval pathway.  

Torque Bio Presents Preclinical Kidney-Targeted AAV for ADPKD | Regulatory

At ASGCT, Torque Bio presented preclinical data for TQB-001, a kidney-targeted AAV gene therapy designed to treat autosomal dominant polycystic kidney disease caused by PKD2 mutations. The program combines a proprietary kidney-tropic AAVk.20 capsid with direct retrograde ureteral administration, demonstrating reduced cyst burden, preservation of kidney function in PKD2 mouse models, and robust kidney-specific transduction with limited systemic exposure in pig studies.

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Oligo

GSK bepirovirsen delivers 19% functional cure rate in hepatitis B | Clinical Trial

GSK reported positive Phase 3 data for bepirovirsen, an ASO for chronic hepatitis B, at EASL on May 28 with simultaneous New England Journal of Medicine publication. Pooled B-Well 1 and B-Well 2 data (n=1,834) showed a 19% functional cure rate versus zero in placebo (p less than 0.001), with bepirovirsen under FDA Priority Review and a PDUFA date of October 26, 2026.

GSK pays $55M upfront to SiranBio for ALK7 siRNA | Partnership

GSK licensed SA030, a Phase 1-stage ALK7-targeting siRNA from China's SiranBio, on May 6, paying $55 million upfront with total potential value of up to $1.005 billion in milestones plus tiered royalties for rights outside China, Hong Kong, Macau, and Taiwan. SA030 is designed to reduce visceral abdominal fat while preserving lean mass, targeting cardiometabolic risk in patients with chronic kidney and liver disease; SiranBio will complete Phase 1 before GSK assumes full development and commercialization.

Madrigal licenses Arrowhead's PNPLA3 siRNA for up to $1B | Partnership                   Madrigal Pharmaceuticals licensed ARO-PNPLA3 from Arrowhead Pharmaceuticals on May 5, paying $25 million upfront against up to $975 million in milestones plus tiered royalties, with Madrigal assuming full development and commercialization responsibility. Phase 1 data showed a 46% liver fat reduction after a single dose in homozygous PNPLA3 I148M patients, a mutation present in roughly 30% of MASH patients with moderate to advanced fibrosis.

Silexion starts GMP manufacturing of KRAS siRNA SIL204 with Catalent | Manufacturing

Silexion Therapeutics announced on May 13 that GMP batch manufacturing of SIL204, its KRAS-targeting siRNA for locally advanced pancreatic cancer, has begun at Catalent's European facility to supply its planned Phase 2/3 safety run-in. Regulatory approvals are in hand from Israel and Tel Aviv Sourasky Medical Center, with a CTA submitted to Germany's BfArM.

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ADCs

FDA Approves Daiichi Sankyo and AstraZeneca’s Datroway for TNBC | Regulatory

The FDA approved datopotamab deruxtecan (Datroway), a TROP2-targeting ADC developed by Daiichi Sankyo/AstraZeneca, for adults with unresectable or metastatic TNBC who are ineligible for PD-1/PD-L1 inhibitor therapy. Approval was based on the Phase III TROPION-Breast02 trial, in which Datroway demonstrated meaningful improvements over investigator's choice chemotherapy: median PFS of 10.8 vs. 5.6 months (HR=0.57, p<0.0001), median OS of 23.7 vs. 18.7 months (HR=0.79, p=0.029), and an overall response rate of 64% vs. 30%.

FDA Approves Abbvie’s DECNUPAZ for BPDCN | Regulatory

The FDA approved DECNUPAZ (pivekimab sunirine-pvzy) for the treatment of adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN); this is AbbVie's first ADC approved for a blood cancer, and the first ADC for BPDCN that can be initiated in an outpatient setting. The approval was backed by Phase 1/2 CADENZA trial data, where newly diagnosed patients achieved a composite complete response rate of ~70% and a median response duration of 9.7 months.  

Pfizer and Innovent Biologics Licensing Agreement Worth More than $10B | Partnership

Pfizer and Innovent Biologics have signed a global licensing and collaboration agreement to jointly research and develop 12 early-stage antibody and ADC candidates targeting various cancers, in a deal worth up to $10.5 billion for Innovent. The partnership covers both ADCs and multi-specific antibodies with novel immune-engaging designs, spanning licensing, co-development, and co-commercialization rights.

Gilead Completes Acquisition of Tubulis GmbH | M&A

Gilead Sciences completed its acquisition of Tubulis GmbH, a German clinical-stage biotech specializing in next-generation ADCs. The deal brings Gilead a platform designed to maximize selective payload delivery to tumors, and Tubulis will establish a dedicated ADC Innovation Center in Munich. The transaction was valued at up to $5 billion and includes TUB-040, a NaPi2b-targeting ADC in Phase 1b/2 development for platinum-resistant ovarian cancer and non-small cell lung cancer.

Tempus AI and Daiichi Sankyo to Implement AI-Driven Biomarker Discovery in ADC Program | Partnership

Daiichi Sankyo and Tempus have formed an oncology-focused AI collaboration that pairs Daiichi Sankyo's clinical and preclinical research assets with Tempus' real-world data platform and multimodal foundation models. The partnership will focus on building proof-of-concept AI tools designed to sharpen patient selection and improve the odds of success for a novel antibody-drug conjugate, drawing on Tempus' PRISM2 model to integrate pathology imaging with clinical data.

SHR-A2102 and Adebrelimab Demonstrates Promising Results for MIBC Patients | Clinical Trial

At ASCO 2026, Phase 2 data from an ongoing Phase 2/3 trial (NCT06879145) showed that SHR-A2102 — a nectin-4-targeting ADC with a topoisomerase 1 inhibitor payload — combined with PD-L1 inhibitor adebrelimab in the perioperative setting produced a 71.4% objective response rate (95% CI: 29.0%–96.3%) in MIBC patients with measurable lesions. While the sample size remains limited and results preliminary, the combination demonstrated a favorable early safety profile across both neoadjuvant and adjuvant settings.