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Insights provided by DeciBio, a strategy consultancy focused on the life science and biopharma industry.
Highlights & Summary
Intro
August was marked by a wave of clinical trial and regulatory updates across the Next-Generation Therapeutics landscape. While safety concerns and accessibility challenges remain, the industry continues to push forward with meaningful progress.
Happy Reading!
Cell Therapy
- Genentech and Adaptive Biotechnologies Collaboration and Licensing Agreement to be Cancelled February 2026 | Partnership
- Wugen Secures $115M for WU-CART-007 | Financing
- KROMATID Secures $8 Million in Series C for Establishing Genomic Analysis Solutions in Cell and Gene Therapy | Financing
Gene Therapy
- 4DMT Provides Pipeline and Clinical Trial Updates | Clinical Trial
- REGENXBIO Receives Updated PDUFA Date for RGX-121 | Regulatory
- Rocket Announces Lifting of Hold on RP-A501 | Clinical Trial
- Beacon to Present Updated Data on Laru-Zova | Clinical Trial
Oligo Therapy
- Sanofi Acquires Greater China Rights for Arrowhead’s Plozasiran to Address Severe Lipid Disorders | Partnership
- Replicate Bioscience and Novo Nordisk Partner to Develop Self-Replicating RNA Therapies for Obesity and Diabetes | Partnership
- Vanda Pharmaceuticals Receives FDA Orphan Drug Designation for VGT-1849B in Polycythemia Vera | Regulatory
Antibody-Drug Conjugates
- CytomX Continues Phase 1 ADC Trial Despite Patient Death | Clinical Trial
- Merck and Daiichi Sankyo Initiate Phase III ADC Breast Cancer Trial | Clinical Trial
Cell Therapy
Genentech and Adaptive Biotechnologies Collaboration and Licensing Agreement to be Cancelled February 2026 | Partnership
Genentech is planning to terminate a cell therapy deal with Adaptive Biotechnologies. Originally launched in 2019, this deal was designed to support Genentech in developing personalized oncology treatments using Adaptive’s T-cell receptor (TCR) tech. Genentech notes that the decision was not based on safety concerns. The cancellation of the collaboration and licensing agreement will be final of February 9, 2026. Adaptive originally received $300M upfront from the deal, with more than $2 billion in potential milestones.
European Commission Authorizes ExCellThera’s Zemcelpro for Blood Cancers | Regulatory
Zemcelpro has been conditionally authorized by the European Commission (EC) for adults with hematological cancers requiring stem cell transplantation for whom no other suitable donor is available, including leukemias and myelodysplastic syndromes. This decision authorizes marketing of Zemcelpro across all EU member states. ExCellThera’s Zemcelpro, or UM171 Cell Therapy, is a cryopreserved hematopoietic stem cell translation that contains UM171-expanded CD34+ cells and unexpanded CD43- cells. It is a one-time cell therapy. While a conditional approval, a phase 3 study is expected to begin soon, and additional regulatory filings planned in North America and the UK.
Wugen Secures $115M for WU-CART-007 | Financing
Wugen, a biotech company creating allogenic, off-the-shelf CAR-T cell therapies closed with $115 million equity, led by Fidelity Management & Research Company. The proceeds will support their T-RRex study of WU-CART-007 for T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma. WU-CART-007, is a CD7-targeted, CRISPR-edited allogenic CAR-T cell therapy. It has received various recognitions previously, including Rare Pediatric Disease designations from the FDA and a Priority Medicines Scheme designation from the EU. In a Phase 1/2 study, WU-CART-007 patients achieved an overall response rate of 91%, and a composite complete remission rate of over 70% after receiving a Phase 2 dose. Wugen eventually plans to engage with moth the FDA and EMA and anticipates a Biologics License Application submission in 2027.
KROMATID Secures $8 Million in Series C for Establishing Genomic Analysis Solutions in Cell and Gene Therapy | Financing
KROMATID closed an $8 million Series C funding round, surpassing its growth capital target, led by BroadOak Capital Partners. This funding will accelerate deployment of KROMATID’s proprietary genomic structural analysis platforms for detecting chromosomal structural rearrangements. This ultimately provides high-resolution and accurate analysis of genomic integrity, which has implications for gene and cell therapy developers.
Gene Therapy
4DMT Provides Pipeline and Clinical Trial Updates | Clinical Trial
4D Molecular Therapeutics reported progress on its lead program, 4D-150, in wet age-related macular degeneration, highlighting faster-than-expected enrollment in its 4FRONT-1 Phase 3 trial, with topline data now expected in the first half of 2027. The company also initiated its second Phase 3 study, 4FRONT-2, ahead of schedule and presented positive 60-week results from the SPECTRA trial in diabetic macular edema (DME), showing favorable safety, durability, and reduced treatment burden. Both the FDA and EMA have agreed that a single successful Phase 3 trial could support approval of 4D-150 in DME, while the company continues preparations for a Biologics License Application and potential commercialization.
REGENXBIO Receives Updated PDUFA Date for RGX-121 | Regulatory
REGENXBIO said the FDA has extended the review timeline for its BLA of RGX-121, an investigational one-time gene therapy for Hunter syndrome (MPS II), shifting the PDUFA date from November 9, 2025, to February 8, 2026. The extension follows the company’s submission of 12-month clinical data from all 13 patients in its pivotal trial, which the FDA requested, and which showed consistent biomarker and neurodevelopmental benefit. The agency completed recent inspections with no observations and has raised no safety concerns to date.
Rocket Announces Lifting of Hold on RP-A501 | Clinical Trial
Rocket Pharmaceuticals said the FDA has lifted the clinical hold on its pivotal Phase 2 trial of RP-A501 for Danon disease, allowing the study to resume less than three months after the hold was placed. The trial will restart with a recalibrated lower dose of 3.8 x 10¹³ GC/kg in three sequentially treated patients, alongside an updated immunomodulatory regimen aligned with prior pediatric data. To date, six patients have been treated in the study, and additional updates are expected once data from the next three patients are reviewed.
Beacon to Present Updated Data on Laru-Zova | Clinical Trial
Beacon Therapeutics announced it will present new data on its lead gene therapy, laru-zova, for X-linked retinitis pigmentosa (XLRP) at the upcoming EURETINA 2025 conference in Paris. The company will share preliminary 9+-month results from the Phase 2 DAWN trial as well as 36-month findings from the randomized, controlled Phase 2 SKYLINE trial on September 4. This follows Janssen and MeiraGTx’s competing asset AAV5-RPGR missing the primary endpoint in its Phase 3 trial back in May.
PackGene and CMRI Announce New High-Throughput Screening Kits | Partnership
PackGene Biotech and Children’s Medical Research Institute (CMRI) announced a licensing and collaboration agreement to advance AAV Capsid Screening Kits for gene therapy research and development. The kits enable high-throughput in vitro, ex vivo, and in vivo screening to identify tissue-specific AAV capsids, while also providing access to proprietary variants with improved specificity, safety, and productivity. Development and high-throughput screening of next-gen. capsids remains a key challenge for the field, with this being cited as the top development hurdle in DeciBio’s recent AAV stakeholder survey.
Oligo
Regeneron Presents Phase 3 Win for Cemdisiran in Generalized Myasthenia Gravis | Clinical Trial
Regeneron announced positive Phase 3 results in generalized myasthenia gravis (gMG) for cemdisiran, a siRNA therapy targeting hepatic C5 production, as both monotherapy and in combination with pozelimab, a C5 antibody. The trial randomized 190 adult patients with symptomatic, anti-AChR antibody-positive gMG across three arms: cemdisiran monotherapy every 12 weeks, cemdisiran plus pozelimab combination every 4 weeks, or placebo. After 24 weeks, patients receiving cemdisiran alone showed a 2.3-point placebo-adjusted reduction in MG-ADL score, surpassing both the primary endpoint and the combination arm, which saw a 1.74-point difference. Both active arms also achieved statistically significant improvements across their secondary endpoints. Cemdisiran monotherapy’s efficacy compared favorably with existing C5 inhibitor drugs approved for gMG, which have shown MG-ADL differences of -1.6 to -2.1 points in similar trials. Regeneron plans to file for FDA approval of cemdisiran monotherapy in gMG in the first quarter of next year.
Sarepta Therapeutics Advances siRNA Collab and Sells Arrowhead Equity Stake | Financing
Sarepta Therapeutics announced the sale of shares of Arrowhead Pharmaceuticals common stock in a private block trade, generating at least $174 million in gross proceeds to fund a $100 million milestone payment to Arrowhead. The company also agreed to transfer 2,660,989 additional shares of Arrowhead to partially fulfill the milestone payment triggered by progress in the SRP-1003 program for type 1 myotonic dystrophy (DM1), specifically following a safety review and achievement of a key enrollment benchmark in Phase 1/2 studies. Sarepta retains confidence in its siRNA strategy and expects to release preliminary results from the SRP-1003 Phase 1/2 study and early clinical data from its facioscapulohumeral muscular dystrophy (FSHD) program in the second half of 2025. Arrowhead may receive an additional $200 million milestone payment if a second enrollment goal in the SRP-1003 trial is met by end of 2025. Sarepta’s equity divestment aligns with its broader financial and strategic restructuring, which includes a recent 36% workforce reduction and narrowed R&D focus on key siRNA programs.
Sanofi Acquires Greater China Rights for Arrowhead’s Plozasiran to Address Severe Lipid Disorders | Partnership
Arrowhead Pharmaceuticals announced that its majority-owned subsidiary Visirna Therapeutics will grant Sanofi exclusive rights to develop and commercialize plozasiran, a first-in-class RNA interference (RNAi) candidate targeting apolipoprotein C-III (APOC3), in Greater China. Plozasiran is designed to reduce production of APOC3, a key regulator of triglyceride metabolism, and has demonstrated up to 80% triglyceride reduction in Phase 3 FCS studies. Visirna has submitted a New Drug Application for plozasiran to China’s National Medical Products Administration (NMPA), already receiving Breakthrough Therapy and Priority Review designations. Sanofi will pay Visirna $130 million upfront and up to $265 million in additional milestone payments linked to regulatory approvals for familial chylomicronemia syndrome (FCS) and other indications within mainland China. Arrowhead will receive royalties on net sales in Greater China as part of the license agreement assigned to Sanofi.
Replicate Bioscience & Novo Nordisk Team Up to Make Self-Replicating RNA Therapies for Obesity and Diabetes | Partnership
Replicate Bioscience announced a multi-year research collaboration with Novo Nordisk to develop novel self-replicating RNA (srRNA) therapies for obesity, type 2 diabetes, and other cardiometabolic diseases. The agreement grants Novo Nordisk exclusive, worldwide rights to use Replicate’s srRNA platform for therapeutic development and commercialization on selected targets, including research funding, an up-front payment, and milestone payments totaling up to $550 million. Replicate will receive tiered royalties on future product sales and will leverage its proprietary srRNA vector library for candidate development. Replicate’s srRNA technology enables customizable protein expression profiles, delivering higher bioactivity, increased durability, and tunable expression in vivo compared to conventional mRNA. RBI-4000, Replicate’s most advanced clinical candidate, has demonstrated protective immunity at lower doses than other RNA-based vaccines in Phase 1 trials. The partnership aims to combine Novo Nordisk’s leadership in cardiometabolic disease drug development with Replicate’s robust srRNA toolbox to create scalable therapies and expand RNA treatment modalities.
Vanda Pharmaceuticals Receives FDA Orphan Drug Designation for VGT-1849B in Polycythemia Vera | Regulatory
Vanda Pharmaceuticals announced that the FDA granted Orphan Drug Designation to VGT-1849B, a novel peptide nucleic acid-based antisense oligonucleotide (ASO) selectively targeting JAK2 mRNA, for the treatment of polycythemia vera (PV), a rare blood cancer affecting 44–57 per 100,000 people in the US. VGT-1849B utilizes OliPass Peptide Nucleic Acid (OPNA) backbone technology that enhances cell permeability and RNA affinity, allowing highly selective inhibition of JAK2 protein with minimal off-target kinase effects or immunosuppression risks compared to existing JAK inhibitors. Over 95% of PV patients harbor the JAK2 V617F gain-of-function mutation that drives aberrant hematopoiesis and uncontrolled red blood cell proliferation. This selectivity is designed to reduce adverse effects and infection risk, supporting a more favorable safety profile and enabling a potentially less frequent dosing regimen than daily oral therapies.
ADCs
CytomX Continues Phase 1 ADC Trial Despite Patient Death | Clinical Trial
A patient enrolled in CytomX Therapeutics’ Phase 1 trial of its lead ADC, CX2051, in metastatic colorectal cancer has died due to a treatment-related acute kidney injury classified as grade 5. The fatal kidney injury was believed to be secondary to nausea, vomiting, and diarrhea; complicating factors included the patient’s complex medical history. CytomX became aware of the death on July 11 and informed the FDA on July 18, and a safety review committee met and recommended continuing the trial. To date, 73 patients have been enrolled, with around 20 in the dose-expansion phase. Despite the setback, CytomX stated that enrollment is nearly complete and remains on track to provide a data update in the first quarter of 2026, with patient safety cited as a top priority.
Padcev Plus Keytruda Combination Significantly Improves Survival in Bladder Cancer | Clinical Trial
Top-line results from a Phase 3 EV-303 trial (KEYNOTE-905) showed that administering PADCEV (enfortumab vedotin), a Nectin-4-targeting antibody-drug conjugate, together with KEYTRUDA (pembrolizumab) both before and after surgery significantly improved event-free survival—the trial’s primary endpoint—and overall survival, compared to surgery alone in patients with muscle-invasive bladder cancer who were ineligible for cisplatin-based chemotherapy. Additionally, the combination met its secondary endpoint of pathological complete response rate. The safety profile for the combination mirrored known safety profiles of the individual agents. Astellas and Pfizer plan to discuss the data with global health authorities for regulatory submissions and anticipate presenting further details at an upcoming medical congress.
Ipsen Backs Out of Previous $875M Deal with Sutro Biopharma | Commercial
Ipsen has withdrawn from a previously announced deal worth up to $875 million to license STRO003, a ROR1-targeting antibody-drug conjugate from Sutro Biopharma, after initially providing about $75 million in upfront payments and equity. STRO003 had been Ipsen's first ADC candidate, but the company decided not to continue the program following a strategic review of new data and evolving insights in the ROR1 therapeutic landscape. Recent Phase 2 data from Merck's own ROR1-directed ADC highlighted a narrow therapeutic window—showing modest overall response rates and higher discontinuation at elevated doses, raising safety concerns. Additional caution arose from reports of severe toxicity in early trials of a ROR1-targeted CAR-T therapy by Lyell Immunopharma. Despite Sutro emphasizing that STRO003 remains a well-engineered candidate, the company does not plan to pursue its internal development.
Merck and Daiichi Sankyo Initiate Phase III ADC Breast Cancer Trial | Clinical Trial
MSD and Daiichi Sankyo have initiated the Phase III HERTHENA-Breast04 trial to assess patritumab deruxtecan, a HER3-directed antibody-drug conjugate, in patients with advanced HR-positive, HER2-negative breast cancer. The study will compare the ADC to standard treatments in patients who are no longer eligible for endocrine therapy and have progressed after CDK4/6 inhibitor treatment. Around 1,000 patients will be enrolled globally, with stratification based on HER2 and HER3 expression, treatment intent, and visceral disease. The primary endpoints are overall survival and progression-free survival, with secondary measures including safety, objective response rate, and duration of response. Earlier data from a Phase I study suggests the drug has promising activity in this patient population.
Merck and Daiichi Sankyo Granted Breakthrough Therapy Designation for SCLC ADC | Regulatory
Ifinatamab deruxtecan (I-DXd), a B7-H3-directed antibody-drug conjugate, has received Breakthrough Therapy Designation from the U.S. FDA for treating adults with extensive-stage small cell lung cancer that has progressed after platinum-based chemotherapy. This is the first such designation for I-DXd and the first since Merck and Daiichi Sankyo began their collaboration. The designation was based on results from the Phase II IDeate-Lung01 trial, supported by data from the Phase I/II IDeate-PanTumor01 trial. Full results will be presented in a late-breaking oral session at the 2025 IASLC World Conference on Lung Cancer.
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