.png)
Insights provided by DeciBio, a strategy consultancy focused on the life science and biopharma industry.
Highlights & Summary
Intro
As we progress through 2025’s Q2, momentum continued across Next-Gen Therapeutics, with notable developments in regulatory approvals and clinical trial activity. Despite ongoing industry uncertainties, financing and strategic partnerships are still occurring in the space as well.
Happy Reading!
Featured DeciBio Updates
1 | Oligo Therapeutics - Key Trends and Current User Perspectives Ad Board | DeciBio
2 | We're attending ASGCT Annual Meeting in May! Connect with Carl to schedule a meeting. Conference | DeciBio
Cell Therapy
- Abeona Therapeutics Receives FDA Approval for ZEVASKYN, First Cell-Based Gene Therapy for RDEB | Regulatory
- Cartesian Therapeutics Reports Positive Phase 2b Results for Descartes-08 in Myasthenia Gravis | Clinical Trial
- Caribou Biosciences Announces Pipeline Prioritization, Focusing on Four Clinical-Stage Programs | Personnel & Clinical Trial
- Ernexa Therapeutics Unveils Preclinical Data for ERNA-101, an Engineered iMSC Therapy for Ovarian Cancer | Clinical Trial
Gene Therapy
- Atsena Raises Oversubscribed Series C | Financing
- HuidaGene Unveils Clinical Data for CRISPR-Based Therapy | Clinical Trial
- Sangamo Licenses CNS Capsid to Lilly for Five Targets | Partnership
- REGENXBIO to Present Updated Manufacturing Process for RGX-202 | Manufacturing
Oligo Therapy
- Arcturus Therapeutics Receives FDA Fast Track for Pandemic Influenza A (H5N1) mRNA Vaccine | Regulatory
- Ethris and Lonza Partner to Develop Room-Temperature Stable, Spray-Dried mRNA Vaccines for Respiratory Disease Prevention | Partnership
- Silexion Therapeutics and Catalent Partner on Advanced siRNA Formulation for KRAS-Driven Cancers | Partnership
- Lilly’s Lepodisiran Achieves Near-94% Reduction in Genetic Heart Disease Risk Factor | Clinical Trial
- Phio Pharmaceuticals Advances siRNA compound PH-762 Skin Cancer Study to Fourth Dose Cohort | Clinical Trial
Antibody-Drug Conjugates
- DATROWAY Approved in EU for Breast Cancer Patients | Regulatory
- Pfizer Presents Preclinical Data on TOP1i-based ADC PF-0805266, Developed on Nona Biosciences’ Proprietary Platform | Partnership
- ALX2004 Receives FDA IND Clearance | Regulatory
Cell Therapy
Abeona Therapeutics Receives FDA Approval for ZEVASKYN, First Cell-Based Gene Therapy for RDEB | Regulatory
The FDA has approved Abeona Therapeutics’ ZEVASKYN, marking the first and only autologous cell-based gene therapy for treating wounds in both adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). ZEVASKYN utilizes genetically corrected keratinocyte sheets derived from a patient’s own skin cells, engineered to express functional type VII collagen, which is essential for anchoring the skin layers. The therapy demonstrated significant wound healing and pain reduction in the pivotal Phase 3 VIITAL study, supporting its approval. ZEVASKYN is expected to become available in the third quarter of 2025.
Cartesian Therapeutics Reports Positive Phase 2b Results for Descartes-08 in Myasthenia Gravis | Clinical Trial
Cartesian Therapeutics announced that its mRNA-engineered CAR-T therapy, Descartes-08, met the primary endpoint in a Phase 2b trial for generalized myasthenia gravis (MG). The therapy, administered as six weekly outpatient infusions without preconditioning chemotherapy, demonstrated deepening responses over time, with an average MG Activities of Daily Living (MG-ADL) score reduction of 4.8 points at Month 12. Patients without prior biologic therapy experienced the deepest responses, with a 7.1-point reduction and 57% achieving minimal symptoms at 12 months. Descartes-08 was well-tolerated, with no cases of cytokine release syndrome or neurotoxicity, supporting its administration in an outpatient setting without the need for lymphodepleting chemotherapy. The company plans to initiate the Phase 3 AURORA trial in the first half of 2025, aiming to enroll approximately 100 participants with acetylcholine receptor autoantibody-positive MG.
Neurona Therapeutics has raised $102 million in an oversubscribed private financing round to support the advancement of its lead regenerative cell therapy candidate, NRTX-1001, into the Phase 3 EPIC (EPIlepsy Cell therapy) trial for drug-resistant mesial temporal lobe epilepsy (MTLE). The funding round included participation from Fidelity Management & Research Company, The Column Group, Soleus Capital, Viking Global Investors, and Cormorant Asset Management, among others. This financing follows positive clinical signals from the ongoing Phase 1/2 trial, where NRTX-1001 demonstrated a 92% median reduction in disabling seizures in the low-dose cohort during the 7–12-month evaluation period, with some patients maintaining over 97% seizure reduction at 24 months post-treatment. The Phase 3 EPIC trial, designed as a randomized, double-blind, sham-controlled study, is expected to commence in the second half of 2025 and aims to serve as the primary basis for a future Biologics License Application submission.
Caribou Biosciences Announces Pipeline Prioritization, Focusing on Four Clinical-Stage Programs | Personnel & Clinical Trial
Caribou Biosciences has announced a strategic pipeline prioritization, concentrating its resources on four clinical-stage programs targeting hematologic malignancies and autoimmune diseases. The prioritized programs are CB-010 for large B cell lymphoma, CB-011 for multiple myeloma, CB-012 for acute myeloid leukemia, and CB-010 for lupus. Notably, CB-010 has received Fast Track designation from the FDA for treating systemic lupus erythematosus, and the GALLOP Phase 1 trial is expected to initiate by the end of 2024.
Ernexa Therapeutics Unveils Preclinical Data for ERNA-101, an Engineered iMSC Therapy for Ovarian Cancer | Clinical Trial
At the AACR Annual Meeting 2025, Ernexa Therapeutics presented preclinical data on ERNA-101, an engineered cell therapy derived from induced pluripotent stem cells (iPSCs) for the treatment of ovarian cancer. ERNA-101 utilizes induced mesenchymal stem cells (iMSCs) engineered to secrete interleukins IL-7 and IL-15, aiming to enhance anti-tumor immune responses. In murine models, ERNA-101 administration resulted in significant tumor growth inhibition and improved survival rates. The therapy increased infiltration of immune effector cells, including T cells, natural killer (NK) cells, and macrophages, into the tumor microenvironment. Additionally, ERNA-101 promoted T cell proliferation in the presence of drug-resistant cancer cells and demonstrated superior expansion capabilities compared to traditional mesenchymal stem cells. These findings support further development of ERNA-101 as a potential treatment for ovarian cancer.
Gene Therapy
Atsena Raises Oversubscribed Series C | Financing
Atsena Therapeutics has raised $150 million in an oversubscribed Series C round led by Bain Capital Life Sciences and Wellington Management to advance its gene therapy pipeline targeting inherited retinal diseases. The funding will support continued development of ATSN-201 for X-linked retinoschisis, expansion of its novel AAV.SPR capsid platform, and progression of its broader ocular gene therapy programs. AAV has seen an uptick in private funding rounds with the likes of Beacon having raised a similar amount last July.
HuidaGene Unveils Clinical Data for CRISPR-Based Therapy | Clinical Trial
HuidaGene Therapeutics, a Shanghai-based biotech, reported early clinical results showing improved motor and cognitive function in a 9-year-old boy treated with its experimental CRISPR RNA-editing therapy for MECP2 duplication syndrome—the first time such a treatment has been delivered to the human brain. The company also unveiled early data from two boys with Duchenne muscular dystrophy treated with its CRISPR DNA-editing therapy, with one boy walking 55 meters farther on a six-minute walk test post-treatment. The company will be giving a presidential symposium at ASGCT’s upcoming annual meeting in New Orleans.
Sangamo Licenses CNS Capsid to Lilly for Five Targets | Partnership
Sangamo Therapeutics has licensed its proprietary STAC-BBB AAV capsid for delivery across the blood-brain barrier to Eli Lilly for use in neurological disease programs. The deal includes an $18 million upfront payment and up to $1.4 billion in potential milestone payments and royalties, marking Sangamo’s third such agreement since unveiling the technology in March 2024. This new agreement spans up to five disease targets and contemplates tiered royalties on potential net sales.
Tenaya Releases Natural History Study Results for Clinical-Stage Asset | Clinical Trial
Tenaya Therapeutics shared interim data from its RIDGE natural history study showing that adults with PKP2-associated arrhythmogenic right ventricular cardiomyopathy experience a high burden of arrhythmias and progressive cardiac damage despite standard treatments. Among 144 patients analyzed, 83% had frequent premature ventricular contractions and nearly half had a history of ventricular tachycardia, while 60% showed disease progression on MRI. Tenaya is developing TN-401, which is now being evaluated in the ongoing Phase 1b RIDGE-1 trial, with early data expected in the second half of 2025.
REGENXBIO to Present Updated Manufacturing Process for RGX-202 | Manufacturing
REGENXBIO has significantly improved the manufacturing process for its Duchenne muscular dystrophy gene therapy, RGX-202 which uses an AAV8 vector to deliver a next-generation microdystrophin. In an abstract presented at this year’s ASGCT annual meeting, the revamped NAVXpress® platform boosted batch yields 56-fold and increased the percentage of full capsids from ~35% to over 80%, addressing key regulatory and supply challenges for systemic AAV gene therapies. The company aims to submit a BLA for the therapy mid next year.
Oligo
Arcturus Therapeutics Receives FDA Fast Track for Pandemic Influenza A (H5N1) mRNA Vaccine | Regulatory
Arcturus Therapeutics’ ARCT-2304, a self-amplifying mRNA (sa-mRNA) vaccine candidate, has received FDA Fast Track designation for the prevention of pandemic influenza A (H5N1). ARCT-2304 specifically targets the hemagglutinin (HA) and neuraminidase (NA) antigens of H5N1. A Phase 1 clinical trial, initiated in November 2024, is currently enrolling 200 healthy adults aged 18 to 80 across multiple U.S. sites, evaluating three dose levels and two vaccination schedules with immune responses measured by hemagglutination inhibition and virus neutralization assays. Interim data from the trial are expected in the second half of 2025, with results to be compared against a licensed influenza vaccine to determine optimal dosing and schedule.
Ethris and Lonza Partner to Develop Room-Temperature Stable, Spray-Dried mRNA Vaccines for Respiratory Disease Prevention | Partnership
Ethris and Lonza have entered a collaboration to develop spray-dried, room-temperature stable mRNA vaccine formulations intended for mucosal delivery against respiratory diseases, with an initial focus on a nasally administered influenza vaccine. The project leverages Ethris’ stabilized non-immunogenic mRNA (SNIM® RNA) and stabilized lipid nanoparticle (SNaP LNP) platform, with Lonza providing spray-drying and particle engineering at its Bend, Oregon facility, which specializes in bioavailability and pharmacokinetic optimization. The nasal, needle-free administration route is designed to elicit localized mucosal immune responses at the site of viral entry, potentially providing immunity comparable to intramuscular vaccines and reducing virus transmission. The collaboration is supported by funding from the Coalition for Epidemic Preparedness Innovations (CEPI) and represents an innovative application of spray-drying technology to RNA-based products, which adds complexity due to the presence of lipid nanoparticles. Lonza’s expertise in particle engineering and Ethris’ RNA platform are combined to address unmet medical needs in non-invasive vaccine delivery and to advance next-generation respiratory disease prevention.
Silexion Therapeutics and Catalent Partner on Advanced siRNA Formulation for KRAS-Driven Cancers | Partnership
Silexion Therapeutics has established a strategic collaboration with Catalent to advance formulation development and clinical manufacturing of SIL204, a next-generation siRNA candidate targeting multiple KRAS mutations implicated in pancreatic, colorectal, and lung cancers. Preclinical studies have demonstrated that SIL204 significantly reduces tumor growth and metastatic spread in orthotopic pancreatic cancer models, targeting KRAS mutations such as G12D, G12V, G12R, Q61H, and G13D. The collaboration will leverage Catalent’s expertise in complex injectable formulations and sustained-release technologies to enhance SIL204’s stability, bioavailability, and delivery precision. Silexion plans to conduct additional toxicology and pharmacodynamic studies throughout 2025, with regulatory submissions anticipated for Israel in the second half of 2025 and for the European Union in the first half of 2026. Initiation of human clinical trials for SIL204 is targeted for the first half of 2026 as part of Silexion’s comprehensive development strategy.
Lilly’s Lepodisiran Achieves Near-94% Reduction in Genetic Heart Disease Risk Factor | Clinical Trial
Lepodisiran, an investigational small interfering RNA (siRNA) therapy developed by Eli Lilly, targets the reduction of lipoprotein(a) [Lp(a)], a genetically inherited cardiovascular risk factor. In the randomized, double-blind, placebo-controlled Phase 2 ALPACA trial, adults with elevated Lp(a) received two doses of lepodisiran (16 mg, 96 mg, or 400 mg) or placebo, with the highest dose group achieving an average 93.9% reduction in Lp(a) levels over a 60- to 180-day period, meeting the primary endpoint. Lower dose groups experienced 40.8% and 75.2% reductions in Lp(a) for the 16 mg and 96 mg cohorts, respectively, and sustained suppression of Lp(a) was observed for up to 18 months after dosing. No serious adverse events related to lepodisiran were reported, and mild injection-site reactions occurred in a dose-dependent manner, with up to 14% in the highest dose group. A Phase 3 clinical outcomes trial is ongoing to further evaluate the long-term cardiovascular benefits of lepodisiran in patients with high Lp(a).
Phio Pharmaceuticals Advances siRNA compound PH-762 Skin Cancer Study to Fourth Dose Cohort | Clinical Trial
Phio Pharmaceuticals’ lead siRNA compound, PH-762, is being evaluated in a Phase 1b multi-center, dose-escalating clinical trial for the neoadjuvant intratumoral treatment of cutaneous squamous cell carcinoma, melanoma, and Merkel cell carcinoma. The Safety Monitoring Committee recommended advancing to the fourth dose escalation cohort after the third cohort, which included three patients with cutaneous squamous cell carcinoma, demonstrated a favorable safety profile with no serious adverse events or dose-limiting toxicities. The ongoing trial aims to determine the recommended dose for further study and assess tumor response to PH-762, which silences the PD-1 gene within the tumor microenvironment using Phio’s proprietary INTASYL gene silencing technology. PH-762 is designed to enhance immune cell activity against cancer cells and is being explored as a potential non-surgical alternative for skin cancer treatment. Pathology results from the third cohort are forthcoming and will provide additional data on efficacy at higher dose levels.
Novartis to Acquire Regulus Therapeutics for Up to $1.7B, Expanding Renal Disease Portfolio | M&A
Novartis has entered into a definitive agreement to acquire Regulus Therapeutics for $7.00 per share in cash at closing, representing a 274% premium to Regulus’ 60-day volume-weighted average stock price, with an additional $7.00 per share contingent value right (CVR) payable upon regulatory approval of Regulus’ lead candidate, farabursen, for autosomal dominant polycystic kidney disease (ADPKD). The total potential transaction value is approximately $1.7 billion and has been unanimously approved by the boards of both companies. Farabursen is a microRNA-targeting oligonucleotide in development for ADPKD, a condition with limited treatment options and significant unmet medical need. The acquisition is expected to close in the second half of 2025, subject to the tender of a majority of Regulus’ outstanding shares and customary regulatory approvals.
ADCs
GSK’s Multiple Myeloma Therapy Blenrep Returns to Market with UK Authorization | Regulatory
GlaxoSmithKline’s multiple myeloma therapy, Blenrep (belantamab mafodotin), was granted authorization in the UK, marking its return to market after a global withdrawal in 2022 following less than optimal results from a confirmatory trial. The approval is for patients who have received prior treatments and includes Blenrep in combination with BVd (bortezomib plus dexamethasone) or with Bristol Myers Squibb’s POMALYST (pomalidomide) plus dexamethasone. Two Phase III trials – DREAMM-7 and DREAMM-8 – demonstrated promising data leading to this reinstatement. DREAMM-7 showed that Blenrep combinations could nearly triple progression-free survival compared to Johnson & Johnson’s treatment (DARZALEX/daratumumab + BVd). Three-year survival approached ~75%, compared to ~60% for the comparator arm. DREAMM-8 also demonstrated superior median progression-free survival for Blenrep compared to other combination therapies. Blenrep is the only BCMA-targeted ADC therapy and has resumed applications for approval in 14 other countries—including the U.S., where approval is expected in July. The drug is also under review in Switzerland, Japan, and China, with GSK projecting annual sales exceeding $4 billion.
DATROWAY Approved in EU for Breast Cancer Patients | Regulatory
DATROWAY (datopotamab deruxtecan), Daiichi Sankyo and AstraZeneca’s TROP2-directed DXd ADC, has received its first approval in the EU for patients with previously treated metastatic HR+, HER2- breast cancer. The approval is based on the TROPION-Breast01 Phase III trial, where there was nearly a 40% reduction in risk of disease progression or death compared to standard chemotherapy. Median progression-free survival was approximately 7 months for patients treated with DATROWAY, around 2 months longer than those treated with chemotherapy. An estimated 70% of diagnosed breast cancer cases are HR+, HER2-, making this approval highly relevant for a substantial patient population. DATROWAY is the third medicine to be approved in the EU from Daiichi Sankyo’s pipeline and is the second ADC for breast cancer patients developed using Daiichi Sankyo’s DXd technology. The ADC is approved in over 30 countries for patients with unresectable or metastatic HR+, HER2- breast cancer who have received prior endocrine-based therapy or chemotherapy.
Pfizer Presents Preclinical Data on TOP1i-based ADC PF-0805266, Developed on Nona Biosciences’ Proprietary Platform | Partnership
Nona Biosciences’ proprietary Harbour Mice and integrated ADC platforms have contributed to the development of Pfizer’s PF-0805266, a TOP1i-based ADC (topoisomerase I inhibitor) that targets MSLN (mesothelin). Pfizer presented preclinical data at AACR 2025, demonstrating potent antitumor activity in various cancers, including ovarian, lung, and colorectal cancers. In in vitro studies, the candidate showed direct cytotoxicity on MSLN-positive cells, and in vivo, it outperformed a DM4-based anti-MSLN benchmark ADC in xenograft models. Pfizer is now enrolling patients in clinical trial NCT06466187, targeting patients with ovarian cancer, non-small cell lung cancer, pancreatic adenocarcinoma, endometrial cancer, colorectal cancer, and mesothelioma.
ALX2004 Receives FDA IND Clearance | Regulatory
ALX2004, an ADC for EGFR-expressing solid tumors and ALX Oncology’s first ADC, has received IND clearance. ALX Oncology will initiate Phase 1 clinical trials later this year. This candidate was developed on ALX Oncology’s proprietary linker-payload platform, with intentional antibody backbone, linker, and payload designs aimed at minimizing toxicity. There are currently no EGFR-targeted ADCs with FDA approval, due in part to challenges with on-target, off-tumor toxicities.
Boehringer Ingelheim’s Subsidiary NBE Therapeutics Opens ADC R&D Facility | Financing
NBE Therapeutics, a subsidiary of Boehringer Ingelheim since its approximately $1.5 billion acquisition in 2020, has opened a new ADC R&D facility in Basel. The facility will support the expansion of ADC pipelines targeting novel tumor antigens and will employ approximately 50 scientists dedicated to this effort. It represents an investment of over CHF 27 million (about $31 million) in ADC research and development in the near term. In addition, the center meets Swiss sustainability standards.
.png)
