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

‍

Highlights & Summary

As we move into the second quarter of the year, the Next-Gen therapeutics space is buzzing with clinical trial updates and exciting new partnerships.

Happy Reading!  

‍

Cell Therapy

Eli Lilly Acquires Kelonia Therapeutics for Up to $7 Billion to Expand In Vivo CAR-T Portfoliov | M&A

Eli Lilly agreed on April 20 to acquire clinical-stage Kelonia Therapeutics for up to $7 billion, with $3.25 billion in upfront cash and the remainder tied to clinical, regulatory, and commercial milestones, marking the largest single acquisition in the in vivo CAR-T space to date. The deal is centered on Kelonia's proprietary in vivo gene placement system (iGPS), which uses engineered lentiviral particles to transduce T cells directly inside the patient's body, bypassing the leukapheresis and centralized ex vivo manufacturing that defines current commercial CAR-T production. Kelonia's lead program, KLN-1010, is a BCMA-targeting in vivo CAR-T in Phase 1 for relapsed/refractory multiple myeloma, with tolerability and proof-of-concept data presented in a plenary session at ASH 2025. The acquisition is Lilly's second in vivo cell therapy deal of 2026, following the $2.4 billion Orna Therapeutics buyout in February, and signals a decisive strategic bet on delivery-platform diversification as the competitive pressure to reduce CAR-T manufacturing complexity intensifies across the industry.

Carvykti Achieves 100% MRD-Negativity in High-Risk Smoldering Myeloma in CAR-PRISM Phase 2 Trial | Clinical Trial

Dana-Farber Cancer Institute researchers reported that a single infusion of ciltacabtagene autoleucel (Carvykti) produced 100% MRD-negativity at a 10-6 sensitivity threshold in all 20 evaluable patients with high-risk smoldering multiple myeloma, with no patient progressing to active disease at a median follow-up of 15.3 months and no high-grade toxicities observed. Results from the single-center CAR-PRISM phase 2 trial were presented at the AACR Annual Meeting in San Diego and published simultaneously in Nature Medicine, representing the first study of BCMA-directed CAR-T therapy in a precursor cancer setting, administered without induction or bridging therapy. The trial's rationale rests on the hypothesis that earlier intervention, before T cell exhaustion from progressive disease and prior therapies, may substantially amplify the depth and durability of CAR-T response. The complete or stringent complete response rate was 90% among all evaluable patients, rising to 100% in those with at least six months of follow-up, setting a response benchmark that has not previously been reported across any stage of the myeloma spectrum.

Verismo Therapeutics Reports First Clinical Data for Mesothelin-Targeting KIR-CAR in Solid Tumors | Clinical Trial

Verismo Therapeutics, a Penn Medicine spinout, presented first-in-human data from the STAR-101 Phase 1 dose-escalation trial of SynKIR-110 in nine patients with advanced mesothelin-expressing solid tumors, including ovarian cancer, mesothelioma, and cholangiocarcinoma, at the AACR Annual Meeting in San Diego. SynKIR-110 is a novel multi-chain CAR-T construct in which antigen recognition and intracellular stimulation are carried on separate receptor chains modeled on natural killer cell inhibitory receptors (KIRs), a design intended to allow T cells to enter a resting state between tumor encounters and thereby resist the chronic activation that drives exhaustion in conventional single-chain CAR-T products. The Phase 1 data showed a manageable safety profile with no dose-limiting toxicities at early dose levels, and an increase in anti-tumor activity at higher dose cohorts consistent with dose-dependent pharmacodynamics. The findings represent the first clinical evidence for a KIR-based CAR architecture and mark the entry of an NK receptor-informed T cell design into human testing for solid tumor indications where exhaustion has historically limited CAR-T durability.

Oricell Therapeutics Closes $110M Pre-IPO Round to Advance GPC3-Targeted CAR-T for Hepatocellular Carcinoma | Financing

Shanghai-based Oricell Therapeutics closed a pre-IPO financing round in excess of $110 million on April 9, co-led by Vivo Capital, Qiming Venture Partners, and the Beijing Medical and Health Care Industry Investment Fund, with participation from an international sovereign wealth fund and six additional institutional investors. The proceeds are earmarked for pivotal clinical development of Ori-C101, an autologous GPC3-targeting CAR-T therapy for advanced hepatocellular carcinoma (HCC), which has completed a registrational Phase 1 study across three dose levels in 10 patients and generated data presented at the ASCO Annual Meeting. GPC3 is a surface glycoprotein highly overexpressed on HCC cells with limited expression on healthy hepatic tissue, and no CAR-T therapy has been approved globally for this indication, giving Ori-C101 a first-in-class regulatory profile in one of the world's most prevalent solid tumors. The round follows a $70 million Series C1 close in January 2026 and precedes an anticipated public offering, reflecting continued investor interest in solid tumor CAR-T programs with mature clinical packages.

Cartherics and Catalent Expand cGMP iPSC Cell Line License for Allogeneic CAR-NK Manufacturing | Manufacturing

Australian biotech Cartherics and contract development and manufacturing organization Catalent announced on April 9 an expanded commercial license agreement granting Cartherics rights to use Catalent's cGMP-compliant, donor-consented iPSC cell line for the manufacture and commercialization of multiple allogeneic CAR-NK cell therapy candidates, with the FDA having already cleared the licensed iPSC starting material for Cartherics' lead program, CTH-401. CTH-401 is an iPSC-derived CAR-NK product targeting TAG-72, a tumor-associated antigen broadly expressed across ovarian cancer, triple-negative breast cancer, and other solid tumors, with additional gene edits to delete immunosuppressive checkpoint pathways and enhance NK cell persistence in the tumor microenvironment. The agreement provides Cartherics with validated GMP workflows spanning iPSC reprogramming, gene editing, directed differentiation, and quality control, while establishing Catalent as the preferred CDMO for commercial-scale production. The deal reflects a structural trend in the allogeneic cell therapy sector, where access to well-characterized, clinical-grade donor iPSC banks is emerging as a manufacturing bottleneck and competitive differentiator for companies developing off-the-shelf immune cell products.

‍

‍

‍

Gene Therapy

FDA approves Regeneron’s Otarmeni for hearing loss | Regulatory
The FDA has granted accelerated approval to Otarmeni, an AAV gene therapy from Regeneron for OTOF-related sensorineural hearing loss, based on data showing significant improvements in hearing sensitivity in a Phase 1/2 trial. In the CHORD study, 80% of patients met the primary endpoint at 24 weeks, with some achieving normal hearing, while confirmatory data will be required to support continued approval.

Ultragenyx’s gene therapy BLA accepted by FDA | Regulatory
The FDA has accepted Ultragenyx’s resubmitted BLA for UX111, an AAV9 gene therapy for Sanfilippo syndrome Type A, and set a PDUFA date of September 19, 2026. The application includes long-term clinical data showing sustained biomarker and neurodevelopmental effects, with the therapy under review for accelerated approval in a disease with no approved treatments.

Ray Therapeutics raises $125M Series B | Financing
Ray Therapeutics closed an oversubscribed $125 million Series B financing led by Janus Henderson Investors, with participation from several new and existing investors. Proceeds will support advancement of its optogenetic vision restoration platform and clinical programs, following recent RMAT designation for its lead candidate.

MeiraGTx reacquires XLRP gene therapy bota-vec from Johnson & Johnson | M&A
MeiraGTx has agreed to reacquire global rights to bota-vec for X-linked retinitis pigmentosa from Johnson & Johnson for $25 million upfront plus milestones and royalties. The company plans to pursue regulatory filings in the U.S., EU, and Japan based on Phase 3 data and existing manufacturing readiness.

Astellas licenses muscle-targeting AAV capsid from Dyno | Partnership
Astellas has exercised an option to license an AI-designed AAV capsid from Dyno Therapeutics for skeletal muscle gene delivery, marking the first muscle-targeting capsid from their collaboration. Dyno will receive a $15 million license fee and is eligible for milestones and royalties, while Astellas assumes responsibility for development and commercialization.

‍

‍

Oligo

Argo Doses First Patient in Phase I Study of siRNA Therapeutic BW-50218 | Clinical Trial

Argo Biopharma announced the first patient has been dosed in its Phase I clinical trial evaluating BW-50218, an investigational siRNA therapeutic. The study is designed to assess the safety, tolerability, and pharmacokinetics of the candidate in healthy volunteers. BW-50218 leverages RNA interference technology to target disease-associated gene expression, representing a novel approach within Argo’s pipeline. The initiation of dosing marks a key milestone as the company advances its early-stage clinical development efforts.

Ionis Partner GSK Receives Priority Review for Bepirovirsen in Hepatitis B | Regulatory

Ionis Pharmaceuticals and its partner GSK announced that bepirovirsen has been accepted for Priority Review by regulatory authorities for the treatment of chronic hepatitis B. Bepirovirsen is an antisense oligonucleotide designed to reduce viral proteins and help restore immune control over the infection. The designation accelerates the review timeline, reflecting the therapy’s potential to address a significant unmet medical need. If approved, bepirovirsen could offer a functional cure approach for patients with chronic hepatitis B.

Silexion Submits Phase 2/3 Trial Application for KRAS-Targeting Therapy  | Clinical Trial

Silexion Therapeutics announced the successful submission of a clinical trial application to Germany’s BfArM for a Phase 2/3 study of SIL204. The investigational therapy targets KRAS-driven locally advanced pancreatic cancer, a disease with limited treatment options and poor prognosis. SIL204 utilizes RNA-based technology to inhibit KRAS mutations, a key oncogenic driver. Approval of the application would enable the company to advance into later-stage clinical testing in Europe.

St. Jude Demonstrates AO Therapy Reverses Neurodevelopmental Disorder | Research

Researchers at St. Jude Children’s Research Hospital reported that an antisense oligonucleotide strategy successfully reversed key features of HNRNPH2-related neurodevelopmental disorder in preclinical models. The approach targets the underlying genetic mutation, restoring normal cellular function and improving neurological outcomes. Findings highlight the potential of precision RNA therapies for rare genetic diseases with limited treatment options. The study provides a foundation for future clinical development and personalized medicine approaches in neurodevelopmental disorders.

‍

‍

‍

‍

ADCs

GSK Presents Positive Phase 1 Data for B7-H4 ADC in Gynecological Cancers | Clinical Trial

GSK announced positive results from its global Phase I BEHOLD-1 trial evaluating mocertatug rezetecan, an investigational B7-H4-targeted antibody-drug conjugate, in gynecological cancers. The therapy achieved confirmed objective response rates of 62% in patients with platinum-resistant ovarian cancer and 67% in those with recurrent or advanced endometrial cancer at the highest dose levels studied. These findings will be presented at the Society of Gynecologic Oncology Annual Meeting, highlighting early clinical activity in patient populations with limited treatment options. Mocertatug rezetecan is designed as a fully human monoclonal antibody linked to a topoisomerase inhibitor payload, with a drug-to-antibody ratio of six, aiming to optimize both efficacy and tolerability. The safety profile observed in the study was described as manageable, supporting continued development. Based on these results, GSK plans to initiate five pivotal Phase III trials starting in 2026 to further evaluate the therapy across multiple gynecologic cancer settings.

Eli Lilly Acquires ADC Player CrossBridge Bio | M&A

CrossBridge Bio announced it has entered into a definitive agreement to be acquired by Eli Lilly and Company in a transaction valued at up to $300 million, including upfront and milestone-based payments. The acquisition centers on CrossBridge’s next-generation dual-payload antibody-drug conjugate platform, designed to deliver two therapeutic agents to tumor cells to enhance efficacy and overcome resistance. The company’s lead program, CBB-120, is a TROP2-targeting ADC combining a topoisomerase I inhibitor and an ATR inhibitor payload, with plans to enter clinical development in 2026. CrossBridge, founded in 2023 and based in Houston, has advanced its platform from academic research developed at the University of Texas Health Science Center at Houston. Eli Lilly aims to leverage this technology to expand and differentiate its oncology pipeline with more advanced ADC approaches. The transaction reflects continued investment in ADC innovation, particularly in multi-payload strategies intended to improve therapeutic outcomes in cancer treatment.

FDA Grants Priority Review for Keytruda Padcev Combo in Cisplatin-Eligible Patients with MIBC | Regulatory

Merck announced that the U.S. FDA has granted Priority Review to two supplemental biologics license applications for pembrolizumab, including both the intravenous formulation and the subcutaneous KEYTRUDA QLEX version, each in combination with the antibody-drug conjugate enfortumab vedotin for patients with muscle-invasive bladder cancer who are eligible for cisplatin-based chemotherapy. The applications seek to expand the use of this combination into the perioperative setting, meaning treatment both before and after surgery. The regulatory submissions are supported by Phase 3 trial data demonstrating significant improvements in clinical outcomes, including event-free survival, reduced risk of death, and higher pathologic complete response rates compared with standard approaches. If approved, the regimen could become the first perioperative treatment option shown to improve survival in this patient population. The FDA has assigned a Prescription Drug User Fee Act target action date of August 17, 2026. The decision builds on prior approvals of the combination in patients who are ineligible for cisplatin, with the new filing aiming to broaden its use regardless of chemotherapy eligibility.

Gilead Acquires ADC Player Tubulis | M&A

Gilead Sciences announced it has entered into a definitive agreement to acquire Tubulis, a clinical-stage biotechnology company focused on next-generation antibody-drug conjugates, in a deal valued at up to $5 billion, including $3.15 billion upfront and up to $1.85 billion in milestone payments. The acquisition adds Tubulis’ ADC platform technologies as well as a pipeline of clinical-stage and early-stage programs designed to enhance selective payload delivery and improve therapeutic index. Key assets include TUB-040, a NaPi2b-targeting ADC currently in Phase 1b/2 development for platinum-resistant ovarian cancer and non-small cell lung cancer, and TUB-030, a 5T4-targeting ADC that has demonstrated early clinical activity across multiple solid tumors. The transaction builds on a prior collaboration between the companies and reflects Gilead’s strategy to expand its oncology portfolio through differentiated ADC approaches. Tubulis’ technologies, including its linker-payload platforms, are intended to address limitations of earlier ADCs such as off-target toxicity and stability. Upon closing, expected in the second quarter of 2026, Tubulis will become part of Gilead’s oncology research infrastructure, supporting continued development of its ADC programs.

Akari Therapeutics Announces Strategic Partnership with WuXi XDC | Partnership

Akari Therapeutics announced a strategic partnership with WuXi XDC to accelerate the development of its novel PH1 payload, a next-generation antibody-drug conjugate payload designed to target RNA splicing in cancer cells. The collaboration will support advancement of Akari’s lead program, AKTX-101, which is initially being developed for metastatic urothelial cancer, an area with significant unmet need. WuXi XDC, a global leader in ADC development and manufacturing, will contribute its expertise to help progress the payload and broader ADC platform into more advanced studies. The PH1 payload represents a differentiated mechanism compared to traditional ADC payloads, aiming to disrupt RNA splicing while also activating immune responses against tumors. The partnership is expected to accelerate Akari’s timeline toward an investigational new drug filing by late 2026 and initiation of a Phase 1 clinical trial thereafter, subject to regulatory clearance. Overall, the agreement is positioned as both a validation of Akari’s platform and a means to advance development of its first-in-class ADC payload technology.