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Highlights & Summary
This month saw strong momentum across next-gen therapeutics, driven by clinical readouts, corporate deals, and regulatory updates. In Cell Therapy, Caribou Biosciences reported promising trial results, Aspen Neuroscience secured $115M Series C funding, and Kelonia Therapeutics partnered with Johnson & Johnson to advance its in‑vivo CAR-T programs. Gene Therapy developments included durable outcomes for 4D-150, corporate expansion by Solid Bio, and regulatory approvals for Novartis and Zhongmou Therapeutics. The Oligonucleotide space was marked by exosome innovation from Capricor Therapeutics, milestone and safety updates from Sarepta, and FDA clearance of Arrowhead’s Redemplo. Meanwhile, the ADC field saw clinical wins and corporate activity, including Kelun-Biotech and Merck trial successes, Padcev’s approval with Keytruda, and $120M raised by Solve Therapeutics.
Happy Reading!
Cell Therapy
- Aspen Neuroscience Secures $115M Series C to Advance Personalized Parkinson’s Cell Therapy | Financing
Gene Therapy
- Solid Bio Licenses Muscle-Targeting Capsid to Andelyn | Partnership
- 4D-150 Shows Durable Wet AMD Control With Major Injection Reduction | Clinical Trial
Oligo Therapy
- Sarepta Announces Positive Safety Review for SRP-1003 and Triggers Milestone Payment | Clinical Trial
Antibody-Drug Conjugates
Cell Therapy
Kelonia Therapeutics & Johnson & Johnson Partner to Accelerate In-Vivo CAR-T Pipeline | Partnership
Kelonia Therapeutics announced a strategic collaboration with Johnson & Johnson to leverage Kelonia’s iGPS in vivo gene-delivery platform for the development of next-generation in-body CAR-T therapies. The partnership aims to apply iGPS to Johnson & Johnson’s oncology targets, with Kelonia leading early research and platform optimization and J&J contributing development, regulatory, and commercialization capabilities. According to Kelonia’s CEO, the collaboration is a “powerful validation” of the iGPS platform and a key step toward democratizing CAR-T therapies. The in vivo approach is designed to eliminate the need for ex vivo manufacturing, apheresis, and lymphodepletion, potentially reducing cost and logistics barriers. The companies expect the platform to support more scalable, accessible CAR-T solutions across a broader range of treatment settings.
Cue Biopharma & ImmunoScape Launch “Seed-and-Boost” Solid Tumor Cell Therapy Program | Partnership
Cue Biopharma and ImmunoScape announced a strategic collaboration and licensing agreement to combine Cue’s Immuno-STAT CUE-100 biologics with ImmunoScape’s tumor-specific TCR-T cells in a novel “Seed-and-Boost” regimen for solid tumors. The approach administers a small “seed” dose of engineered T cells followed by targeted “boost” doses of Cue’s IL-2–based biologics to selectively expand those cells in vivo, aiming to improve persistence, tolerability, and scalability compared to traditional cell therapies. As part of the agreement, Cue will receive $15 million in upfront and time-based payments, along with a 40% equity stake in ImmunoScape and eligibility for high-single-digit royalties on net sales. Preclinical data cited by the companies indicate strong T-cell activation and anti-tumor activity across multiple solid tumor models. The collaboration is expected to progress toward IND-enabling studies, with development activities led by ImmunoScape.
Aspen Neuroscience Secures $115M Series C to Advance Personalized Parkinson’s Cell Therapy | Financing
Aspen Neuroscience announced the closing of a $115 million Series C financing round to accelerate development of its lead autologous cell therapy program, ANPD001, for moderate to advanced Parkinson’s disease. The round was co-led by major life-sciences investors including OrbiMed, ARCH Venture Partners, Frazier Life Sciences and Revelation Partners, with new participation from Kite, a subsidiary of Gilead Sciences, among others. The funds will support ongoing clinical trials (including the recently initiated Cohort 3 of the Phase 1/2a study), scale up manufacturing capacity, and expand Aspen’s pipeline of iPSC-derived therapies for additional neurological disorders. After this round, Aspen has raised over $340 million in total capital. Company leadership described the financing as a pivotal moment that bolsters confidence in the team, platform, and market opportunity for regenerative cell therapies in neurodegenerative disease.
Caribou Biosciences Reports Strong Phase 1 Readout for vispa-cel in ANTLER Trial | Clinical Trial
Caribou Biosciences announced positive results from the ANTLER Phase 1 Trial of vispacel (CB-010), its allogeneic anti-CD19 CAR-T therapy for relapsed or refractory B cell non-Hodgkin lymphoma (B-NHL). In the 22-patient confirmatory cohort (2nd-line LBCL, partial HLA matched), vispa-cel achieved an overall response rate (ORR) of 82%, a complete response (CR) rate of 64%, and a 12-month progression-free survival (PFS) of 51%. In an optimized 35-patient cohort (HLA-matched, younger donors), ORR was 86%, CR 63%, and 12-month PFS 53%, with median follow-up of ~11.8 months. The therapy was generally well-tolerated: across key cohorts, no graft-versus-host disease (GvHD) and no grade ≥3 neurotoxicity (ICANS) were reported, and ≥grade 3 cytokine-release syndrome (CRS) occurred in under 5% of patients. Caribou described the results as evidence that an off-the-shelf allogeneic CAR-T can match the efficacy and durability of autologous products, a milestone that could broaden access and enable outpatient administration.
Gene Therapy
Solid Bio Licenses Muscle-Targeting Capsid to Andelyn | Partnership
Solid Biosciences signed a non-exclusive global licensing and collaboration agreement with Andelyn Biosciences, allowing Andelyn to offer Solid’s next-generation AAV-SLB101 capsid to its gene therapy manufacturing clients. The capsid, designed for enhanced skeletal and cardiac muscle targeting with reduced liver biodistribution, has shown favorable early clinical safety in Solid’s Phase 1/2 INSPIRE DUCHENNE trial. The deal expands access to AAV-SLB101, which is already in use across more than 30 external research and development programs.
4D-150 Shows Durable Wet AMD Control With Major Injection Reduction | Clinical Trial
4D Molecular Therapeutics reported interim 1.5- to 3.5-year data showing that its wet AMD gene therapy 4D-150 sustained visual acuity, controlled retinal anatomy, and sharply reduced the need for supplemental anti-VEGF injections across all Phase 1/2 cohorts. A strong dose response favored the Phase 3 dose, and the therapy remained well tolerated with no new inflammation cases through up to 3.5 years of follow-up. The company is currently enrolling patients in two Phase 3 trials with enrollment expected to be completed in 2026.
Novartis Receives Approval for Itvisma in Broader SMA Population | Regulatory
Novartis received FDA approval for Itvisma, expanding access to its SMA gene replacement therapy to children aged two and older, as well as teens and adults. The decision, supported by Phase III STEER and STRENGTH data, showed significant motor-function improvements and sustained stabilization over 52 weeks, with a consistent safety profile. Itvisma delivers a one-time intrathecal dose that replaces the missing SMN1 gene.
Sarepta Set to Begin Testing New Elevidys Immunosuppression Regimen | Regulatory
Sarepta Therapeutics said the FDA has cleared dosing to begin in Cohort 8 of the ENDEAVOR study, which will evaluate whether adding sirolimus to ELEVIDYS gene therapy can reduce acute liver injury in non-ambulant Duchenne muscular dystrophy patients. The cohort will enroll about 25 participants and assess liver-related safety and micro-dystrophin expression at 12 weeks, with dosing expected to start by year-end and primary data in the second half of 2026.
FDA Clears Zhongmou Therpauetics’ IND for Retinitis Pigmentosa | Regulatory
Zhongmou Therapeutics said the FDA has cleared its IND application for ZM-02, enabling a multinational, randomized, placebo-controlled clinical trial of the company’s mutation-agnostic optogenetic gene therapy for advanced retinitis pigmentosa. The authorization makes ZM-02 the first China-originated optogenetic therapy to receive U.S. IND clearance and follows first-in-human data showing meaningful vision gains, functional improvements, and sustained benefit over 52 weeks. The upcoming PRISM trial will evaluate safety and functional vision outcomes in patients in the U.S. and China.
Oligo
Capricor Therapeutics Details Scalable Exosome Loading Framework | Preclinical
Capricor Therapeutics presented data at the 2025 American Association for Extracellular Vesicles (AAEV) Annual Meeting outlining a scalable framework for loading therapeutic siRNA and PMO into exosomes. The company reported that its optimized scale-up and scale-out electroporation strategies achieved loading efficiencies comparable to standard small-volume methods using the StealthX platform. The study demonstrated that integrating these approaches allows for the manufacturing of larger batches of engineered exosomes.
FDA Limits Elevidys Indication and Adds Warning Following Fatalities | Regulatory
The U.S. Food and Drug Administration (FDA) has restricted the approved use of Sarepta Therapeutics’ gene therapy Elevidys to ambulatory patients aged four years and older, removing the indication for non-ambulatory individuals. This label update includes a new Boxed Warning for acute serious liver injury and acute liver failure following reports of fatal outcomes in non-ambulatory patients treated with the therapy. The revised prescribing information mandates enhanced safety protocols, including weekly liver function monitoring for three months after administration. Additionally, the FDA has required Sarepta to conduct a post-marketing observational study to further evaluate the risk of liver toxicity in the approved patient population.
Ionis Announces $700M in Convertible Notes to Refinance 2026 Debt | Financial
Ionis Pharmaceuticals announced its intention to offer $700 million in Convertible Senior Notes due 2030 through a private placement to qualified institutional buyers, with an option for an additional $70 million. The company plans to use the net proceeds to repurchase or repay its existing 0% Convertible Senior Notes due 2026, including potential concurrent repurchases at the time of pricing. The new notes will be unsecured obligations accruing interest semiannually, with conversion options into cash, common stock, or a combination of both. Final terms, including interest rate and conversion rate, will be determined upon pricing of the offering.
FDA Approves Arrowhead’s Redemplo for Familial Chylomicronemia Syndrome | Regulatory
The FDA has granted approval to Arrowhead Pharmaceuticals for Redemplo (plozasiran), an RNAi therapeutic indicated as an adjunct to diet for reducing triglycerides in adults with familial chylomicronemia syndrome (FCS). This decision marks the first regulatory clearance for an RNAi-based medicine targeting the APOC3 gene to treat this rare genetic disorder characterized by severe hypertriglyceridemia and risk of acute pancreatitis. Approval was supported by data from the Phase 3 PALISADE trial, which demonstrated significant and sustained reductions in triglyceride levels compared to placebo. Redemplo is administered subcutaneously once every three months and is expected to launch in the U.S. market shortly.
Sarepta Announces Positive Safety Review for SRP-1003 and Triggers Milestone Payment | Clinical Trial
Sarepta Therapeutics reported a positive review from the drug safety committee regarding its ongoing Phase 1/2 trial of SRP-1003, an investigational siRNA therapeutic for type 1 myotonic dystrophy (DM1). The company confirmed that full enrollment of the third dose cohort has triggered a $200 million milestone payment to licensor Arrowhead Pharmaceuticals, which is payable within 60 days. Following the safety committee's clearance, Sarepta has commenced dosing in a fourth cohort and plans to initiate the final dose-escalation group early next year. The SRP-1003 program utilizes Arrowhead’s Targeted RNAi Molecule (TRiM) platform to deliver gene-silencing therapy directly to skeletal muscle tissue.
ADCs
Solve Therapeutics Raises $120M to Develop ADCs for Solid Tumors | Financing
Solve Therapeutics announced it raised $120 million in an oversubscribed financing round to accelerate development of its ADC pipeline and proprietary CloakLink™ linker technology, bringing total capital raised to about $321 million. The funds are earmarked to complete Phase 1b trials of its lead ADC candidates, SLV-154 and SLV-324, in solid tumor indications. The CloakLink™ platform is designed to improve on conventional ADCs by reducing payload hydrophobicity — enhancing plasma stability, pharmacokinetics, and lowering toxicity — which could enable a better therapeutic index. Both ADCs use antibodies optimized for solid tumor targeting and are paired with diagnostic tools to select patients more precisely. The new funding also aims to expand Solve’s operational capabilities as it prepares for later-stage development.
Day One Biopharmaceuticals to Acquire Mersana Therapeutics | M&A
Mersana Therapeutics has agreed to be acquired by Day One Biopharmaceuticals in a deal that gives Mersana shareholders cash up front plus contingent-value rights (CVRs) valuing the deal at up to about $285 million. The acquisition will be executed via a tender offer followed by a second-step merger, with closing expected by the end of January 2026, contingent on regulatory approval and sufficient shareholder participation. The main asset Day One is getting is Emi-Le (emiltatug ledadotin; XMT-1660), a B7-H4–targeted Dolasynthen ADC currently in early-stage clinical development, offering a potential new cancer therapy.
NEOK Bio Launches with $75M Series A to Advance Next-Generation Bispecific ADCs | Financing
NEOK Bio has emerged from stealth with a $75 million Series A funding round, backed by ABL Bio, to advance a new generation of bispecific antibody-drug conjugates (ADCs) for oncology. The financing will support the development of two lead ADC candidates — NEOK001, which targets ROR1 and B7-H3, and NEOK002, which targets EGFR and MUC1 — with plans to file INDs by early 2026 and begin Phase 1 clinical trials around mid-2026. NEOK uses a proprietary SYNtecan E™ linker-payload technology, designed to offer strong linker stability and improved biophysical properties compared with conventional ADCs. Through its dual-targeting, bispecific design, NEOK aims to increase selectivity, improve internalization and cell killing, potentially reduce off-tumor toxicity, and expand ADC applicability to a broader range of solid tumors (e.g. thoracic, gastrointestinal, gynecological cancers). The company expects first human data readouts from both programs in 2027.
Padcev Keytruda Combo Approved for Cisplatin Ineligible MIBC | Regulatory
The U.S. Food and Drug Administration has approved PADCEV (enfortumab) in combination with Keytruda (or Keytruda QLEX) as neoadjuvant (before surgery) and adjuvant (after surgery) treatment for adults with muscle-invasive bladder cancer (MIBC) who are ineligible for cisplatin chemotherapy. The approval — based on results from the pivotal Phase 3 EV303 trial — showed that this regimen significantly reduces the risk of disease recurrence, progression, or death compared with surgery alone, offering a survival advantage for a patient population with limited options. This marks the first time an ADC plus PD-1 inhibitor combo has been approved in the perioperative setting for bladder cancer. Importantly, the approval addresses a major unmet need since many MIBC patients cannot tolerate cisplatin, and for some, surgery alone still carried high risk of relapse. With this decision, PADCEV + Keytruda is poised to become a new standard of care in cisplatin-ineligible MIBC patients.
Kelun-Biotech and Merck Announce Trial Success in 1L PD-L1-Positive NSCLC | Clinical Trial
Kelun-Biotech and Merck & Co. announced that their TROP2-directed ADC sacituzumab tirumotecan, (sac-TMT) given in combination with pembrolizumab (Keytruda), succeeded in a late-stage trial as a first-line treatment for PD-L1-positive non-small cell lung cancer (NSCLC). The study — OptiTROPLung05 — met its primary endpoint, showing a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared with pembrolizumab alone. The data also showed a favorable trend toward overall survival, though full OS results were not yet definitive. The outcome could pave the way for a potential new standard-of-care option, especially for PD-L1 positive NSCLC patients.
