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

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Highlights & Summary

This month saw continued momentum across next-gen therapeutics, characterized by diverse clinical milestones, strategic partnerships, and new financing. The Cell Therapy space was rich with innovation, highlighted by Kelonia Therapeutics’ positive first-in-human data for in vivo CAR-T and Kyverna Therapeutics' positive registrational Phase II results in Stiff Person Syndrome , while Link Cell Therapies emerged with a $60M Series A. In Gene Therapy, focus shifted to clinical and regulatory progress, with Encoded Therapeutics and Tenaya Therapeutics sharing promising early data , and Latus Bio securing IND clearance. The Oligonucleotide sector was defined by collaboration and capacity expansion, including GSK’s partnership with CAMP4 Therapeutics and Alnylam’s $250M investment in manufacturing. Meanwhile, the ADC field remained dynamic with major deals, such as Ipsen’s $1B licensing agreement with Simcere Zaiming and the Kelun-Crescent partnership , though the sector also saw a setback with a clinical hold on Merck and Daiichi Sankyo’s Phase III lung cancer trial.

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Cell Therapy

1. Kelonia Therapeutics Shares Positive First-in-Human Phase I KLN-1010 in vivo BCMA CAR-T Data | Clinical Trial
2. Link Cell Therapies Launches With $60M Series A to Advance Logic-Gated CAR-T for Solid and Liquid Tumors | Financing
3. Kyverna Therapeutics Reports Positive Registrational Phase II Topline Data for Miv-cel in Stiff Person Syndrome | Clinical Trial
4. FDA Signals Tighter Approval Standards for CAR-T Therapies, Prioritizing Randomized Superiority Trials | Regulatory
5. MD Anderson Reports Updated Phase II Anito-cel Data With High Response Rates in Relapsed / Refractory Multiple Myeloma | Clinical Trial

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Gene Therapy

1. Latus Bio Secures IND Clearance for CLN2 Gene Therapy | Regulatory
2. Encoded Reports Interim Seizure and Developmental Data in Dravet Gene Therapy Trials | Clinical Trial
3. Tenaya Shares Early Safety and Arrhythmia Data for PKP2 Cardiomyopathy Gene Therapy | Clinical Trial
4. uniQure Receives FDA Feedback on AMT-130 Huntington’s BLA Path | Regulatory‍
5. VectorY Cleared to Begin Phase 1/2 ALS Trial Targeting TDP-43 | Regulatory

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Oligo Therapy

1. Rona Therapeutics Advances Bi-Valent siRNA into Development | Clinical Trial
2. Protalix Biotherapeutics and Secarna Pharmaceuticals Enter Collaboration for Rare Renal Indications | Partnership
3. CAMP4 Therapeutics and GSK Collaborate for RNA-Based Therapeutics | Partnership
4.  Alnylam Invests $250M in Enzymatic Ligation Platform Production | Manufacturing‍
5. Rona Therapeutics Doses First Cohort for INHBE siRNA Obesity Study | Clinical Trial

Antibody-Drug Conjugates

1. Kelun-Biotech and Crescent Biopharma Announce ADC Partnership | Commercial
2.  Padcev-Keytruda Combination Scores Phase III Win In MIBC | Clinical Trial
3. Merck and Daiichi Sankyo’s Phase III I-DXd SCLC Trial Put On Hold Due to Patient Deaths | Clinical Trial
4.  Ipsen Licenses Simcere Zaiming’s LRRC15-targeting ADC In Up To $1B Deal | Commercial
5. FDA Grants Orphan Designation to GSK’s Risvutatug Rezetecan for SCLC | Regulatory

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Cell Therapy

Kelonia Therapeutics Shares Positive First-in-Human Phase I KLN-1010 in vivo BCMA CAR-T Data | Clinical Trial

Kelonia Therapeutics presented late-breaking first-in-human data from the Phase 1 inMMyCAR study of KLN-1010, an in vivo BCMA-directed CAR-T gene therapy for relapsed/refractory multiple myeloma, at the 2025 American Society of Hematology Annual Meeting. The initial cohort of four patients achieved a 100 % minimal residual disease (MRD)-negative response rate, with responses maintained through up to five months of follow-up; robust CAR-T cell expansion and persistent memory CAR-T cells were observed without lymphodepleting chemotherapy. The safety profile was favorable, with no grade 3 or higher cytokine release syndrome or immune effector cell-associated neurotoxicity noted, differentiating KLN-1010 from more intensive traditional CAR-T approaches. These data, highlighted for the first time in a late-breaking oral presentation, suggest the potential for an accessible, off-the-shelf in vivo CAR-T treatment paradigm. Company leadership underscored that the deep responses and manageable safety seen to date offer promising early evidence of clinical activity for this next-generation approach.

Link Cell Therapies Launches With $60M Series A to Advance Logic-Gated CAR-T for Solid and Liquid Tumors | Financing

Link Cell Therapies emerged from stealth with a $60M Series A financing, bringing total funding to approximately $92M, to advance a pipeline of logic-gated CAR-T therapies for solid and liquid tumors. The company’s platform is designed to activate CAR-T cells only when specific combinations of tumor antigens are present, with the goal of improving tumor selectivity and reducing on-target, off-tumor toxicity. Its lead program, LNK001, targets dual antigens co-expressed in renal cell carcinoma and is expected to enter a Phase 1 clinical trial in 2026, with a colorectal cancer program planned for 2027. Link Cell is co-founded by Robbie Majzner, MD, whose prior work helped establish logic-gated CAR-T approaches, and the company plans to expand its pipeline both internally and through partnerships. Leadership highlighted the potential for logic gating to enable more potent CAR-T therapies while sparing healthy tissues.

Kyverna Therapeutics Reports Positive Registrational Phase II Topline Data for Miv-cel in Stiff Person Syndrome | Clinical Trial

Kyverna Therapeutics announced positive topline results from its registrational Phase 2 KYSA-8 trial of mivocabtagene autoleucel (miv-cel), a fully human, autologous CD19-targeting CAR-T therapy with CD28 co-stimulation, in patients with stiff person syndrome (SPS), a rare and debilitating autoimmune disorder with no approved therapies. The single-arm study dosed 26 patients and at the Week 16 primary endpoint showed a median 46 % improvement in timed 25-foot walk versus baseline (p=0.0002), with 81 % of patients achieving a clinically meaningful ≥20 % improvement, and highly significant benefits observed across all secondary endpoints. A majority of patients who previously required walking aids no longer needed assistance, and all remained free of immunotherapies through the last follow-up, with miv-cel demonstrating a manageable safety profile. Company leadership stated these results could support a biologics license application (BLA) submission in the first half of 2026 and potentially position miv-cel as the first approved CAR-T therapy for an autoimmune disease. Management highlighted the transformative potential of these data for SPS patients and the broader autoimmune CAR-T field.

FDA Signals Tighter Approval Standards for CAR-T Therapies, Prioritizing Randomized Superiority Trials | Regulatory

The FDA is moving toward stricter approval requirements for new CAR-T cell therapies, emphasizing the need for randomized controlled trials (RCTs) that demonstrate superiority over existing treatments or standard of care for traditional full approval. Historically, all CAR-T therapies approved to date were supported largely by single-arm studies; under the evolving framework, such designs may still enable accelerated approval, but confirmatory evidence from RCTs with survival or other time-to-event endpoints will generally be expected for full licensure. FDA officials, including the agency’s biologics chief, outlined these expectations in a perspective piece published late in 2025, urging sponsors to carefully consider comparator selection and trial endpoints. This regulatory shift comes alongside other FDA actions in the CAR-T space, such as earlier elimination of REMS requirements for approved CD19- and BCMA-directed therapies to reduce access barriers, underscoring the agency’s dual focus on rigorous evidence and patient access. The updated expectations could meaningfully impact how developers design pivotal CAR-T trials and position their products relative to existing therapies.

MD Anderson Reports Updated Phase II Anito-cel Data With High Response Rates in Relapsed / Refractory Multiple Myeloma | Clinical Trial

MD Anderson Cancer Center presented updated Phase II iMMagine-1 trial results showing that anitocabtagene autoleucel (anito-cel), an investigational BCMA-targeting CAR-T therapy, continued to deliver high efficacy in patients with relapsed/refractory multiple myeloma (median three prior lines of therapy). In a cohort of 117 patients, the overall response rate was 97 % with a 68 % complete response rate; 12-month progression-free and overall survival rates were 79 % and 95 %, respectively, with durable outcomes maintained at 18 months. The safety profile was manageable, with mostly low-grade cytokine release syndrome and no unusual neurologic complications, though hematologic cytopenias were common. Investigators highlighted that anito-cel uses a novel synthetic binder designed to simplify manufacturing and potentially improve tolerability compared to other CAR-T constructs. A Phase III study evaluating anito-cel against standard of care is already underway to further define its clinical benefit in this setting.

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Gene Therapy

Latus Bio Secures IND Clearance for CLN2 Gene Therapy | Regulatory

Latus Bio received FDA clearance of its IND for LTS-101, an AAV gene therapy designed to treat the central nervous system manifestations of CLN2 disease. The FDA also granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations, enabling regulatory incentives and potential expedited review as the program enters clinical development.

Encoded Reports Interim Seizure and Developmental Data in Dravet Gene Therapy Trials | Clinical Trial

Encoded Therapeutics reported positive interim data from the POLARIS program evaluating ETX101 in children with SCN1A-positive Dravet syndrome. Early dose cohorts showed dose-dependent reductions in seizure frequency and improvements in neurodevelopmental measures, with no treatment-related serious adverse events reported to date.

Tenaya Shares Early Safety and Arrhythmia Data for PKP2 Cardiomyopathy Gene Therapy | Clinical Trial

Tenaya Therapeutics reported interim results from the first cohort of its RIDGE-1 trial of TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy. In three patients treated at the lower dose, TN-401 was well tolerated, increased PKP2 protein expression in heart tissue, and was associated with reductions in arrhythmia burden in patients with longer follow-up.

uniQure Receives FDA Feedback on AMT-130 Huntington’s BLA Path | Regulatory

uniQure disclosed that the FDA indicated current Phase 1/2 data for AMT-130 are unlikely to support a Biologics License Application for Huntington’s disease on their own. The company plans to request a follow-up meeting in early 2026 to clarify regulatory expectations and potential next steps.

VectorY Cleared to Begin Phase 1/2 ALS Trial Targeting TDP-43 | Regulatory

VectorY Therapeutics received FDA clearance to initiate the PIONEER-ALS Phase 1/2 trial of VTx-002, a vectorized antibody therapy targeting pathological TDP-43 in ALS. The open-label study will evaluate safety, biomarker effects, and exploratory clinical outcomes following a single intracisterna magna administration in up to 12 patients.

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Oligo 

Rona Therapeutics Advances Bi-Valent siRNA into Development | Clinical Trial

Rona Therapeutics submitted RN5681 to the Australian Human Research Ethics Committee (HREC), advancing the first bi-valent siRNA targeting both PCSK9 and LPA genes into clinical development for cardiovascular disease. RN5681 is a GalNAc-conjugated dual-targeting siRNA designed to simultaneously reduce LDL-C and Lp(a) levels within a single molecule. The Phase 1 trial is expected to begin dosing in Q1 2026.

Protalix Biotherapeutics and Secarna Pharmaceuticals Enter Collaboration for Rare Renal Indications | Partnership

Protalix Biotherapeutics and Secarna Pharmaceuticals entered a collaboration and option agreement to jointly discover antisense oligonucleotide (ASO) therapies targeting multiple genes for rare renal indications. Secarna will apply its AI-powered OligoCreator platform to design and profile ASO candidates against pharmaceutical targets selected by Protalix. Under the agreement, Protalix holds an exclusive option to license any active compounds derived from the research for potential clinical development and commercialization.

CAMP4 Therapeutics and GSK Collaborate for RNA-Based Therapeutics | Partnership

CAMP4 Therapeutics entered a research, collaboration, and license agreement with GSK to discover and develop antisense oligonucleotide (ASO) candidates targeting multiple genes associated with neurodegenerative and kidney diseases. CAMP4 will utilize its RAP Platform to identify regulatory RNAs (regRNAs) that modulate gene expression and generate ASO candidates designed to amplify target gene expression, while GSK will be responsible for subsequent development and commercialization. CAMP4 will receive $17.5 million upfront and is eligible for up to $440 million in development and commercial milestone payments, plus tiered royalties on annual net sales ranging from low- to mid-single digits.

Alnylam Invests $250M in Enzymatic Ligation Platform Production | Manufacturing

Alnylam Pharmaceuticals announced a $250 million investment to add its siRELIS enzymatic ligation platform to its Norton, Massachusetts manufacturing facility to increase production capacity and reduce costs for RNAi therapeutics. The siRELIS platform assembles short RNA fragments into complete siRNA molecules more efficiently than traditional methods, reducing use of starting materials and organic solvents while increasing manufacturing throughput. The FDA has accepted Alnylam's enzymatic ligation manufacturing platform into its Emerging Technology Program to fast-track global regulatory engagement.

Rona Therapeutics Doses First Cohort for INHBE siRNA Obesity Study | Clinical Trial

Rona Therapeutics announced the completion of Cohort 1 dosing in its Phase 1 first-in-human study of RN3161, a GalNAc-conjugated siRNA targeting INHBE for obesity treatment, with a favorable initial safety and tolerability profile. RN3161 is designed for deep and durable silencing of INHBE with once- or twice-yearly dosing, based on human genetic evidence supporting INHBE as a metabolic target. The Phase 1 randomized, double-blind, placebo-controlled study is evaluating safety, tolerability, pharmacokinetics, pharmacodynamics, and effects on body weight in adults with overweight and obesity, with additional cohorts planned for completion in 2026.

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ADCs

Kelun-Biotech and Crescent Biopharma Announce ADC Partnership | Partnership

Crescent Biopharma and Kelun-Biotech have entered a strategic partnership to jointly develop and commercialize two oncology candidates, combining Crescent’s PD-1 × VEGF bispecific antibody CR-001 with Kelun-Biotech’s integrin β6-targeted antibody-drug conjugate SKB105. Both drug candidates are being developed for solid tumors and are expected to enter Phase 1/2 clinical trials as monotherapies in the first quarter of 2026, with potential evaluation in combination. Under the terms of the collaboration, Crescent granted Kelun exclusive rights to CR-001 in Greater China, while Kelun granted Crescent exclusive rights to SKB105 in the United States, Europe, and other markets outside Greater China. Under the collaboration, Kelun-Biotech will receive an upfront payment of $80M USD from Crescent and is eligible for milestones up to $1.25B USD, plus middle single-digit to low double-digit royalties on net sales of SKB105. Crescent will receive an upfront payment of $20M USD from Kelun-Biotech and is also eligible to receive additional milestones of up to $30M, plus low to middle single digit royalties on net sales of CR-001.

Padcev-Keytruda Combination Scores Phase III Win In MIBC | Clinical Trial

Pfizer and Astellas’ antibody-drug conjugate Padcev (enfortumab vedotin) combined with MSD’s Keytruda (pembrolizumab) demonstrated superior outcomes versus standard neoadjuvant platinum-based chemotherapy in muscle-invasive bladder cancer in the Phase III EV-304 trial. The combination met the primary endpoint of improved event-free survival and also showed a statistically significant overall survival benefit. It additionally produced higher pathological complete response rates when used in the perioperative setting. The safety profile was consistent with previous studies of the Padcev-Keytruda regimen. These results position the combination as the first chemotherapy-free regimen to improve both event-free and overall survival in cisplatin-eligible MIBC patients.

Merck and Daiichi Sankyo’s Phase III I-DXd SCLC Trial Put On Hold Due to Patient Deaths | Clinical Trial

Merck and Daiichi Sankyo have had the FDA place a partial clinical hold on their late-stage development program for the antibody-drug conjugate ifinatamab deruxtecan after observing a higher-than-expected number of patient deaths, which were attributed to interstitial lung disease, a known risk with DXd-based ADCs. The hold affects the Phase III IDeate-Lung02 trial in relapsed small-cell lung cancer, which had enrolled over 500 patients, and has led the companies to voluntarily halt recruitment while they work with regulators and an independent data board to understand the fatalities. A spokesperson declined to disclose the exact number of deaths but emphasized that the issue is specific to the affected study and does not impact other trials in the ADC’s broader clinical development program. Interstitial lung disease is recognized as a potentially fatal toxicity associated with this class of ADCs, though other DXd ADCs like Enhertu and Datroway have continued to be approved despite similar risks. Merck and Daiichi Sankyo had teamed up in 2023 to co-develop three DXd ADCs, and the current hold focuses only on ifinatamab deruxtecan’s Phase III program. The partners are now engaging with the FDA to determine next steps and how best to proceed given the safety concerns.

Ipsen Licenses Simcere Zaiming’s LRRC15-targeting ADC In Up To $1B Deal | Commercial

Ipsen has signed an exclusive licensing agreement with Simcere Zaiming to gain global development, manufacturing, and commercialization rights outside Greater China for SIM0613, an innovative antibody-drug conjugate targeting the LRRC15 protein in solid tumors. SIM0613 is engineered for enhanced tumor penetration and differentiated anti-tumor activity, supported by robust preclinical efficacy data. The ADC is expected to enter Phase I clinical development in the second half of 2026. Under the agreement, Simcere Zaiming may receive up to $1.06 billion in upfront, development, regulatory, and commercial milestone payments, plus tiered royalties, contingent on successful progress and approvals. Ipsen will assume responsibility for activities outside Greater China, including manufacturing and regulatory filings. This collaboration expands Ipsen’s early development oncology pipeline and aligns with its ambitions to advance first- and best-in-class therapies globally.

FDA Grants Orphan Designation to GSK’s Risvutatug Rezetecan for SCLC | Regulatory

The FDA has granted orphan drug designation to the B7-H3-targeted antibody-drug conjugate risvutatug rezetecan (also known as GSK’227) as a potential treatment for patients with small cell lung cancer (SCLC). This designation was supported by preliminary data from the Phase 1 ARTEMIS-001 trial, which showed promising antitumor activity in patients with extensive-stage SCLC, including overall response and disease control rates at evaluated dose levels. In these cohorts, risvutatug rezetecan demonstrated median durations of response and progression-free survival measured in several months. The safety profile included common treatment-related adverse effects such as decreased neutrophil, white blood cell, and lymphocyte counts, decreased platelets, and anemia. The orphan designation highlights the drug’s potential in a cancer type with limited treatment options and high unmet need. These findings support further development of risvutatug rezetecan in SCLC.