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Insights provided by DeciBio, a strategy consultancy focused on the life science and biopharma industry.
Highlights & Summary
Intro
June 2025's Next-Gen Therapeutics ecosystem presented various clinical trial announcements, especially in cell therapies.
Happy Reading!
Cell Therapy
- Cero Therapeutics Begins Dosing in Phase I Trial of CD33-Targeted Cell Therapy for AML | Clinical Trial
Gene Therapy
- REGENXBIO Shares Updated Data on Duchenne Candidate | Clinical Trial
- Sangamo Announces Positive Interim Results on Fabry Candidate | Clinical Trial
- ViGeneron Rebrands and Advances Two Clinical Programs | Clinical Trial
- Beacon Makes Key CMO Hire | Personnel
- Arbor Bio Adds to its Board of Directors | Personnel
Oligo Therapy
- Ethris and Thermo Fisher Scientific Announce Strategic Collaboration for mRNA Technology Platforms | Partnership
- Silence Therapeutics Presents Additional Phase 1 Data for Divesiran in Polycythemia Vera | Clinical Trial
Antibody-Drug Conjugates
- Roche’s Lunsumio and Polivy Combination Prolongs Remission in Refractory Large B-Cell Lymphoma | Clinical Trial
- NextCure Strikes $745M ADC Deal with Simcere | Partnership
- Simtra BioPharma and Millipore Sigma Announce Partnership for ADC Manufacturing Services | Partnership
Cell Therapy
Cero Therapeutics Begins Dosing in Phase I Trial of CD33-Targeted Cell Therapy for AML | Clinical Trial
Cero Therapeutics announced that the first patient has been dosed in a Phase I clinical trial of CERO-101, its lead engineered cell therapy targeting CD33 for relapsed/refractory acute myeloid leukemia (AML). CERO-101 is based on the company’s Chemically Self-Assembled Nanoring (CSAN) platform, which engineers T-cells to recognize CD33-expressing leukemia cells. The open-label trial is a dose-escalation study designed to enroll approximately 36 adult patients across four sites in the U.S. to evaluate safety, tolerability, and preliminary anti-leukemic activity. The company highlighted that CERO-101 represents a potentially differentiated approach compared to traditional CAR-T therapies, given its modular and non-viral engineering method.
Legend Biotech Reports Positive 5-Year Outcomes for CARVYKTI in Multiple Myeloma | Clinical Trial
Legend Biotech presented five‑year durability and survival data from its Phase Ib/II CARTITUDE‑1 trial of CARVYKTI at the 2025 ASCO Annual Meeting. At a median follow‑up of 61.3 months, the median overall survival was 60.7 months, and 33% of heavily pre‑treated patients remained progression‑free for at least five years following a single infusion, without additional myeloma therapy. Safety findings were consistent with the known profile of CARVYKTI, with no new neurocognitive or movement‑related adverse events reported; two new solid tumor second primary malignancies occurred. According to Chief Medical Officer Mythili Koneru, “For one‑third of these heavily pre‑treated patients to remain progression‑free for five years after a single infusion—and without needing further myeloma therapy—represents a potential paradigm shift” in the treatment of multiple myeloma.
Gilead’s DualTarget CART Shows Tumor Shrinkage in Glioblastoma in Phase I Trial | Clinical Trial
Gilead Sciences’ Kite unit and the University of Pennsylvania reported interim Phase I data on a dual‑target CAR‑T therapy administered into the cerebrospinal fluid for recurrent glioblastoma at ASCO. In 18 patients treated after surgery, 13 had measurable tumors, and 62% of those showed tumor shrinkage. While many patients experienced relapse within two to three months, some maintained disease control for longer periods, including one patient with stable disease beyond 16 months. The therapy targets both EGFR and IL‑13Rα2 to address glioblastoma’s heterogeneity. Further trials are planned to evaluate additional targets and use in newly diagnosed patients.
Diakonos Oncology Reports Positive Phase I Results for Dubodencel in Glioblastoma | Clinical Trial
Diakonos Oncology presented Phase I data on Dubodencel (DOC1021), a dendritic cell therapy, at ASCO 2025. The study enrolled 16 newly diagnosed and 2 recurrent glioblastoma patients who received three biweekly doses of DOC1021 alongside weekly pegylated interferon. The therapy demonstrated a favorable safety profile and induced a robust immune response, with an 88% 12-month overall survival rate—significantly higher than the ~60% expected with standard care. Additionally, the trial revealed a favorable safety profile, with mild flu-like symptoms and injection-site reactions as the most common adverse events and no dose-limiting toxicities reported. Observations of pseudo-progression on MRI suggest an early immune-reactive microenvironment that may predict longer survival without immediate surgical intervention. Based on these results, Diakonos plans to advance to a randomized Phase II trial.
Vertex’s Zimislecel Restores Insulin Secretion in 10 Type 1 Diabetes Patients | Clinical Trial
Vertex Pharmaceuticals presented Phase 1/2 data on Zimislecel (VX-880), an allogeneic stem cell–derived islet cell therapy, for type 1 diabetes (T1D) at ASCO 2025. In a cohort of 12 patients with impaired hypoglycemic awareness, all achieved the American Diabetes Association’s target of HbA1c under 7% and over 70% time in range (70–180 mg/dL). Notably, 10 patients (83%) no longer required exogenous insulin use at month 12, with a mean reduction of 92% in daily insulin use. All participants demonstrated glucose-responsive C-peptide production, indicating restored endogenous insulin secretion. The therapy was generally well tolerated, with no reported serious adverse events related to Zimislecel.
Gene Therapy
REGENXBIO Shares Updated Data on Duchenne Candidate | Clinical Trial
REGENXBIO shared positive new interim data from its Phase I/II AFFINITY DUCHENNE trial, highlighting functional improvements and strong biomarker results for its Duchenne muscular dystrophy gene therapy candidate, RGX-202. At the pivotal dose, participants showed clinically meaningful gains in motor function and North Star Ambulatory Assessment scores at 9 and 12 months, alongside high microdystrophin expression and a favorable safety profile, positioning the therapy for a potential accelerated approval filing in mid-2026.
Sangamo Announces Positive Interim Results on Fabry Candidate | Clinical Trial
Sangamo Therapeutics reported positive topline results from its Phase 1/2 STAAR study of ST-920, showing that a single gene therapy dose led to a favorable mean annualized eGFR slope at both 52 and 104 weeks, exceeding the decline seen with existing Fabry treatments. Patients also experienced durable α-Gal A expression, withdrawal from enzyme replacement therapy, quality of life improvements, and no serious safety concerns, positioning the therapy for a potential accelerated approval submission in early 2026.
ViGeneron Rebrands and Advances Two Clinical Programs | Clinical Trial
VeonGen Therapeutics, formerly ViGeneron, announced the advancement of two AAV-based programs—VG801 for Stargardt disease and VG901 for CNGA1-related retinitis pigmentosa—into human trials. Lead candidate VG801, which recently received FDA Rare Pediatric Disease Designation, is being evaluated in a Phase 1/2 trial and leverages VeonGen’s proprietary vgRNA REVeRT and vgAAV platforms to deliver large genes intravitreally, with the company also pursuing applications in cardiovascular and CNS indications.
Beacon Makes Key CMO Hire | Personnel
Beacon Therapeutics has appointed Dr. Daniel Chung as Chief Medical Officer, who brings industry experience from prior roles at Spark Therapeutics and the development of Luxturna. His hiring comes as Beacon nears full enrollment in its pivotal Phase 2/3 VISTA trial of laru-zova for X-linked retinitis pigmentosa, following recent positive interim results from the Phase 2 DAWN study.
Arbor Bio Adds to its Board of Directors | Personnel
Arbor Biotechnologies has appointed Dr. Mikael Dolsten, former Chief Scientific Officer and President of Worldwide R&D at Pfizer, to its Board of Directors. Dolsten brings over 16 years of leadership experience at Pfizer and a track record of advancing breakthrough therapies, including the company’s COVID-19 response, and will advise Arbor as it advances its in vivo gene editing pipeline targeting genetic diseases.
Oligo
Ethris and Thermo Fisher Scientific Announce Strategic Collaboration for mRNA Technology Platforms | Partnership
Ethris has entered a strategic collaboration with Thermo Fisher Scientific to provide biopharmaceutical developers with integrated access to Ethris’ proprietary mRNA technology platforms and Thermo Fisher’s GMP-compliant manufacturing capabilities. The partnership will enable rapid advancement of mRNA medicines from research through clinical proof-of-concept by combining Ethris’ Stabilized Non-Immunogenic mRNA (SNIM® RNA), minimal UTR, and mRNA manufacturing technologies with Thermo Fisher’s end-to-end manufacturing expertise. Ethris’ SNIM® RNA platform has demonstrated positive pharmacodynamic effects, safety, and targeted engagement in Phase 1 topline data for its lead candidate, ETH47, which is administered via nasal spray for asthma exacerbations. The collaboration will also support ongoing optimization of Ethris’ platform technologies and expand global access to scalable, high-quality mRNA solutions for therapeutic and vaccine development.
Silence Therapeutics Presents Additional Phase 1 Data for Divesiran in Polycythemia Vera | Clinical Trial
Silence Therapeutics presented updated Phase 1 data for divesiran, an siRNA targeting TMPRSS6, in patients with polycythemia vera (PV) at the EHA 2025 Annual Meeting. The 34-week, open-label SANRECO Phase 1 study evaluated subcutaneous divesiran at 3, 6, and 9 mg/kg every six weeks for four doses in 21 PV patients with a history of frequent phlebotomies. Divesiran maintained rapid and durable control of hematocrit, with most patients avoiding phlebotomies during the study period, and demonstrated increased hepcidin and ferritin levels, indicating target engagement. Divesiran has received FDA Fast Track and Orphan Drug designations and is the first siRNA in clinical development specifically targeting red blood cell production in PV.
Arrowhead Pharmaceuticals Initiates Phase 1/2a Study of ARO-ALK7 for Obesity | Clinical Trial
Arrowhead Pharmaceuticals has initiated a Phase 1/2a clinical trial of ARO-ALK7, the first investigational RNAi therapeutic targeting the ACVR1C gene in adipose tissue for the treatment of obesity. The AROALK7-1001 study will enroll up to 90 adults with obesity and is designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-ALK7 as both monotherapy and in combination with tirzepatide, a GLP-1/GIP receptor co-agonist. Preclinical studies demonstrated that ARO-ALK7 silences ALK7 expression in adipose tissue, resulting in reduced body weight and fat mass while preserving lean muscle. The trial will assess single and multiple doses of ARO-ALK7, with the goal of improving body composition and metabolic parameters in patients with obesity.
Ionis and Biogen Advance Salanersen for SMA Following Positive Phase 1 Results | Clinical Trial
Ionis Pharmaceuticals and Biogen reported positive interim Phase 1 results for salanersen (ION306/BIIB115), an investigational antisense oligonucleotide designed for spinal muscular atrophy (SMA), showing substantial slowing of neurodegeneration and clinically meaningful motor function improvements in children previously treated with gene therapy. The study included a randomized, placebo-controlled segment in healthy adults and an open-label segment in pediatric SMA patients with suboptimal clinical status after ZOLGENSMA® (onasemnogene abeparvovec). In the pediatric cohort (n=24), annual dosing of 40 mg or 80 mg salanersen was generally well tolerated and led to a mean 70% reduction in neurofilament light chain (NfL) at six months, sustained through one year. Biogen is engaging with global regulators to advance salanersen into registrational Phase 3 studies for SMA.
BioNTech Announces Strategic Transaction to Acquire CureVac in Public Exchange Offer | M&A
BioNTech has entered an agreement to acquire CureVac in an all-stock transaction, exchanging each CureVac share for approximately $5.46 in BioNTech American Depositary Shares, a 55% premium over CureVac’s three-month volume weighted average price as of June 11, 2025. The deal values CureVac at about $1.25 billion and is subject to a collar mechanism that adjusts the exchange ratio based on BioNTech’s share price at closing. Upon completion, CureVac shareholders will own between 4% and 6% of BioNTech, and CureVac’s operating subsidiary will become a wholly owned subsidiary of BioNTech. The transaction is supported by major CureVac shareholders, including dievini Hopp BioTech holding and the German government’s KfW bank, with contractual commitments already representing over 50% of CureVac shares toward the 80% minimum acceptance threshold. The acquisition is expected to close in 2025 following regulatory approvals and a CureVac shareholder vote.
ADCs
Roche’s Lunsumio and Polivy Combination Prolongs Remission in Refractory Large B-Cell Lymphoma | Clinical Trial
Roche’s phase III SUNMO trial demonstrated that combining its bispecific antibody Lunsumio (mosunetuzumab) with the antibody-drug conjugate Polivy significantly reduced the risk of disease progression or death by 59% compared to the standard chemotherapy regimen R-GemOx in second-line, transplant-ineligible large B-cell lymphoma (LBCL) patients. The median progression-free survival with the Roche combo was 11.5 months versus just 3.8 months with R-GemOx. While overall survival data are still maturing, the new regimen showed a promising early trend toward improved survival, including an 87% improvement in progression-free survival among North American patients. Safety profiles were comparable between both treatments, though the Roche combo had fewer treatment-related deaths (1.5% vs. 3.1%). Roche plans to submit these results and aims to offer a convenient, outpatient-friendly, and potentially more effective alternative to traditional chemotherapy for relapsed or refractory LBCL.
NextCure Strikes $745M ADC Deal with Simcere | Partnership
NextCure has doubled down on its focus on antibody-drug conjugates (ADCs) by securing a licensing deal worth up to $745 million for the ex-China global rights to Simcere Zaiming’s CDH6-targeted ADC, SIM0505, currently in Phase 1 trials in China and slated for U.S. study initiation in Q3 2025. The ADC combines a CDH6-binding antibody with a proprietary topoisomerase 1 inhibitor payload designed for broad anti-tumor effects and improved systemic clearance, aiming to enhance efficacy and safety. As part of the arrangement, NextCure also gains access to Simcere’s linker and payload technology for its own preclinical ADC program, while Simcere retains Greater China rights to that candidate. The licensee expects to see initial clinical data in H1 2026, while tying payments to milestones and tiered royalties outside China. This strategic move complements NextCure’s broader ADC push, including its in-clinic B7-H4 ADC (LNCB74), and positions the company well for solid-tumor expansion using both licensed and proprietary ADC assets.
ADC Therapeutics to Shutdown UK Research Site Amid Broader Cuts | Commercial
ADC Therapeutics is implementing a major restructuring, including closing its UK R&D site and cutting 30% of its global workforce by the end of September. The company is discontinuing several early-stage ADC programs—such as those targeting Claudin-6 and ASCT2—after recently halting development of its CD22-targeted asset. This strategic shift focuses resources on its approved lymphoma drug Zynlonta and a preclinical PSMA-targeted ADC for prostate cancer. The move reflects a broader effort to streamline operations and concentrate on its most promising oncology assets.
Simtra BioPharma and Millipore Sigma Announce Partnership for ADC Manufacturing Services | Partnership
MilliporeSigma, Merck KGaA’s North American life science division, has formed a five-year strategic partnership with Simtra BioPharma Solutions to streamline the manufacturing of antibody-drug conjugates (ADCs). Under the agreement, MilliporeSigma will manage bioconjugation and, if needed, payload synthesis, while Simtra will handle drug formulation and fill-finish. Both companies will share responsibilities for analytics and overall project coordination. The partnership aims to reduce manufacturing hand-offs, accelerate development timelines, and offer biotech clients a seamless, end-to-end “turnkey” service. With the ADC manufacturing market expected to grow significantly, the alliance is positioned to help clients bring therapies to market more efficiently.
OBI Pharma Enters Into ADC Collaboration with TegMine Therapeutics | Partnership
TegMine Therapeutics has partnered with OBI Pharma to use OBI’s GlycOBI® technology for advancing its antibody-drug conjugate (ADC) pipeline. This collaboration focuses on developing ADCs that target tumor-specific glycans, using OBI’s site-specific conjugation platform to improve stability, uniformity, and manufacturing scalability. TegMine, founded by former Stemcentrx scientists, aims to enhance ADC precision and therapeutic potential by targeting carbohydrate antigens unique to tumors. If promising ADC candidates emerge, the companies plan to formalize licensing agreements.
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