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Insights provided by DeciBio, a strategy consultancy focused on the life science and biopharma industry.
Highlights & Summary
Intro
As Q3 closes, partnerships are reshaping the Next-Generation Therapeutics landscape: streamlining production, ensuring consistency, and expanding access to breakthrough assets across diverse diseases.
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Cell Therapy
- Ori Biotech’s IRO Platform Receives FDA Advanced Manufacturing Technology (AMT) Designation | Manufacturing
- Mytos Partners with StemSight, Rinri & Novadip to Scale Automated Cell Therapy Manufacturing | Partnership / Manufacturing
- Kytopen & BlueWhale Bio Collaborate on Non-Viral Workflow to Speed Cell Therapy Manufacturing | Partnership / Manufacturing
- Cellares Selects Five Technology Providers to Expand Cell Q QC Capabilities | Partnership / Manufacturing
Gene Therapy
- uniQure’s AMT-130 Prevails in Huntington’s Disease | Clinical Trial
- REGENXBIO Shares Updated Pivotal Data for MPS II Asset | Clinical Trial
- AskBio Completes Enrollment for AB-1005 | Clinical Trial
- VectorY Grabs Option to Shape’s Brain-Penetrating Capsid | Partnership
- Kinea Receives New Funding and Licenses Solid Capsid | Partnership
Oligo Therapy
- Arrowhead Pharmaceuticals & Novartis Collaborate on siRNA-based Parkinson’s Disease Therapeutic | Partnership
Antibody-Drug Conjugates
- Merck and Daiichi Sanko Deliver Promising Data for I-Dxd | Clinical Trial
- BioNTech and DualityBio Announce Phase 3 Data of ADC Candidate in HER2-Positive Breast Cancer | Clinical Trial
Cell Therapy
Ori Biotech’s IRO Platform Receives FDA Advanced Manufacturing Technology (AMT) Designation | Manufacturing
Ori Biotech announced that its IRO platform has received FDA Advanced Manufacturing Technology (AMT) designation, one of the first technologies to achieve this recognition. The designation is intended to encourage adoption of innovative manufacturing approaches by providing earlier and more frequent regulatory engagement. IRO is a modular, end-to-end platform that enables automated, closed, and scalable production of cell therapies. Ori stated that this milestone highlights IRO’s potential to improve efficiency and quality in advanced therapy manufacturing while supporting faster patient access to new treatments.
Mytos Partners with StemSight, Rinri & Novadip to Scale Automated Cell Therapy Manufacturing | Partnership / Manufacturing
Mytos has entered into new collaborations with three biotech firms (StemSight, Rinri Therapeutics, and Novadip) to deploy its iDEM automated cell manufacturing platform across a diverse set of cell therapy applications. StemSight will apply the platform for its iPSC-derived corneal stem cell therapy targeting limbal stem cell deficiency (a form of blindness), Rinri will use it for its otic neural progenitor therapy Rincell-1 for sensorineural hearing loss entering clinical trials, and Novadip is evaluating it for autologous stem cell-based bone regeneration in large bone defects. The partnerships are positioned to improve scalability, consistency, and cost efficiency by automating processes previously done manually, such as differentiation and expansion under GMP-compatible conditions.
ArsenalBio Halves Staff as It Shifts Focus to Clinical-Stage Assets | Personnel
ArsenalBio has laid off ~50% of its workforce, reducing to 127 employees, signaling a transformation from early‐stage research toward becoming a clinical-stage company. The restructuring follows its $325M Series C raise and aims to extend cash runway and refocus resources on advancing its clinical programs. Its lead asset, AB-2100, is in an open-label Phase 1/2 trial for recurrent clear-cell renal cell carcinoma; AB-2100 was promoted over previous lead AB-1015 (which targeted ovarian cancer) in September 2024. The company also has two other preclinical assets: one for prostate cancer and another in an undisclosed indication, with first-in-human studies for the prostate cancer asset expected in the first half of 2026. ArsenalBio confirmed its collaboration with Bristol Myers Squibb (focused on solid tumor CAR-T therapies) remains unaffected by the workforce changes.
Kytopen & BlueWhale Bio Collaborate on Non-Viral Workflow to Speed Cell Therapy Manufacturing | Partnership / Manufacturing
Kytopen and BlueWhale Bio announced a collaboration to build a non-viral manufacturing workflow combining Kytopen’s Flowfect continuous flow cellular engineering platform with BlueWhale’s Synecta CDNPs (Cell-Derived Nanoparticles) technology. The joint effort aims to address current manufacturing bottlenecks (especially long vein-to-vein times (3-5 weeks), high costs, and safety concerns) by enabling high-efficiency processing of billions of engineered cells in minutes without viral vectors. Synecta CDNPs mimic antigen-presenting cells, delivering TCR stimulation, co-stimulatory signals, and membrane-bound cytokines in an ex vivo setting, while Flowfect offers tunable parameters (mechanical, electrical, chemical) to maximize transfection, cell fitness, and yield. Both platforms are already in use: Synecta in a Phase I investigator-initiated trial, and Flowfect has multiple therapies advancing toward IND submission. The partnership is positioned to reduce manufacturing times, improve scalability, and lower costs for personalized cell therapies.
Cellares Selects Five Technology Providers to Expand Cell Q QC Capabilities | Partnership / Manufacturing
Cellares has partnered with Tecan, Advanced Instruments, Cytek Biosciences, Slingshot Biosciences, and AltemisLab to enhance its Cell Q platform, an automated quality control (QC) system intended to support commercial-scale cell therapy manufacturing. The upgrades span high-throughput sample preparation, liquid-handling verification, full-spectrum flow cytometry, synthetic controls, and preservation of chain of identity/custody (COI/COC). Cell Q is designed to match the throughput of Cellares’ Cell Shuttle™ system, with capacity to process up to ~6,000 cell therapy batches per year. First performance data from Cell Q will be presented at the 10th CAR-TCR Summit on September 25, 2025. According to Cellares, Cell Q aims to reduce operational burden, enhance data integrity, and meet stringent GMP and regulatory QC demands globally.
Gene Therapy
uniQure’s AMT-130 Prevails in Huntington’s Disease | Clinical Trial
uniQure reported positive topline results from its pivotal Phase I/II trial of AMT-130, a one-time gene therapy for Huntington’s disease, showing a statistically significant slowing of disease progression at 36 months. High-dose AMT-130 reduced decline by 75% on the composite Unified Huntington’s Disease Rating Scale and by 60% on Total Functional Capacity compared to a matched external control, with supportive trends across motor and cognitive measures and reductions in the neurodegeneration biomarker CSF NfL. The therapy was generally well tolerated, with no new serious drug-related adverse events. uniQure plans to meet with the FDA later this year and submit a Biologics License Application in early 2026.
REGENXBIO Shares Updated Pivotal Data for MPS II Asset | Clinical Trial
REGENXBIO reported new positive Phase I/II/III CAMPSIITE trial data for RGX-121 for Mucopolysaccharidosis Type II (Hunter syndrome), showing sustained 82% median reductions in cerebrospinal fluid heparan sulfate D2S6 at one year and strong correlations between biomarker reductions and neurodevelopmental improvement. The therapy was well tolerated across all 26 treated patients, with no new safety concerns, and the FDA is reviewing a Biologics License Application with a decision expected by February 8, 2026.
AskBio Completes Enrollment for AB-1005 | Clinical Trial
AskBio announced completion of enrollment in REGENERATE MSA-101, its Phase 1 trial of AB-1005, an investigational AAV2-based gene therapy delivering the GDNF gene for the treatment of multiple system atrophy-parkinsonian type (MSA-P). The milestone advances the program for the rare, rapidly progressive neurodegenerative disease, which affects about 400,000 people worldwide and currently has no disease-modifying treatments. The randomized, double-blind study enrolled 11 participants to evaluate the safety of AB-1005 delivered to the putamen, with initial results expected after one year of blinded assessments. AB-1005 is also in a Phase 2 trial for Parkinson’s disease and has FDA RMAT designation.
VectorY Grabs Option to Shape’s Brain-Penetrating Capsid | Partnership
VectorY Therapeutics and Shape Therapeutics announced an option and license agreement granting VectorY exclusive rights to evaluate Shape’s deep-brain penetrating AAV capsid, SHP-DB1, for vectorized antibody therapies targeting three neurodegenerative disease programs. Shape will receive an upfront payment and could earn up to $1.2 billion in total fees and milestones, including up to $338 million for rare disease programs and $503.5 million for non-rare disease programs, plus tiered royalties on future sales if VectorY exercises the option and advances therapies using the SHP-DB1 platform.
Kinea Receives New Funding and Licenses Solid Capsid | Partnership
Kinea Bio announced two key advances for its dysferlinopathy gene therapy program, including up to $1.1 million in milestone-based funding from the Jain Foundation to support IND-enabling preclinical studies and a strategic license from Solid Biosciences for its next-generation myotropic AAV capsid, AAV-SLB101. The funding will back critical safety and biodistribution studies for Kinea’s dual-AAV therapy KNA-155, while the licensed capsid is designed to enhance muscle-targeted delivery and reduce liver uptake, strengthening the program’s path toward first-in-human trials.
Oligo
Kalexo Bio Launches With $1B+ siRNA Deal for Cardiometabolic Pipeline | Partnership
Mabwell Bioscience and Aditum Bio have announced the formation of Kalexo Bio, a new company created through an exclusive global license agreement to develop 2MW7141, a novel dual-target siRNA candidate for lipid management in patients with dyslipidemia and prevention of high-risk atherosclerotic cardiovascular disease events. Under the deal terms, Mabwell will grant Kalexo Bio exclusive worldwide rights to develop, manufacture, and commercialize the therapy, with Mabwell eligible to receive up to $1 billion in upfront and milestone payments plus tiered royalties on global sales, including $12 million in non-refundable upfront and near-term cash payments. Aditum Bio will provide funding for Kalexo Bio and collaborate to advance 2MW7141 into clinical development through their incubator model designed to accelerate drug development.
Arrowhead Pharmaceuticals & Novartis Collaborate on siRNA-based Parkinson’s Disease Therapeutic| Partnership
Arrowhead Pharmaceuticals and Novartis have formed a global licensing and collaboration agreement for ARO-SNCA, a preclinical siRNA therapy targeting alpha-synuclein for the treatment of synucleinopathies including Parkinson's disease. Under the deal terms, Arrowhead will receive $200 million upfront and is eligible for up to $2 billion in development, regulatory and sales milestone payments plus tiered royalties on commercial sales. Novartis will gain exclusive worldwide rights to research, develop, manufacture, and commercialize ARO-SNCA, which uses Arrowhead's proprietary Targeted RNAi Molecule (TRiM) platform to deliver siRNA subcutaneously to the central nervous system. The transaction is expected to close in the second half of 2025, pending regulatory approvals and customary closing conditions.
Argo Biopharma Announces Collaboration with Novartis for Cardiovascular Assets | Partnership
Argo Biopharma has created an additional strategic collaboration with Novartis for multiple cardiovascular assets in its pipeline, marking their third transaction together. Under the agreement, Novartis receives an option to license ex-China rights to two discovery-stage next-generation molecules for severe hypertriglyceridemia and mixed dyslipidemia, plus a right of first negotiation to BW-00112 (ANGPTL3), currently in Phase II. The collaboration also includes a reciprocal profit and loss sharing arrangement for an additional hepatic-delivered siRNA candidate expected to begin Phase I trials in 2026. Argo will receive $160 million upfront and is eligible for potential milestone and option payments valued up to $5.2 billion, plus tiered royalties on commercial sales. Novartis has also expressed non-binding intention to participate in Argo's next equity financing round.
Aro Biotherapeutics Completes Phase 1b Trial Enrollment for LOPD Drug | Clinical Trial
Aro Biotherapeutics announced completed enrollment of its Phase 1b clinical trial of ABX1100, a novel investigational therapy for treating late-onset Pompe disease (LOPD) in patients currently receiving enzyme replacement therapy (ERT). ABX1100 has received Orphan Drug Designation and Rare Pediatric Disease status from the FDA and is a first-in-class Centyrin-siRNA conjugate that inhibits expression of glycogen synthase 1 (GYS1), the enzyme responsible for glycogen production. All enrolled patients received one dose of ABX1100 on Day 1 and a booster dose on Day 29 and will be monitored through 20 weeks to assess safety and preliminary therapeutic activity.
Argo Biopharma Receives Phase II Approval from NMPA for PNH Drug | Regulatory
Argo Biopharma announced that China's National Medical Products Administration (NMPA) granted Investigational New Drug (IND) approval for a Phase II clinical trial of BW-40202, an investigational siRNA therapy targeting complement factor B (CFB) for treating paroxysmal nocturnal hemoglobinuria (PNH) and other complement alternative pathway-mediated diseases. BW-40202 utilizes RNA interference to suppress CFB expression in the liver. In preclinical studies, BW-40202 demonstrated significant and durable suppression of serum CFB protein, effective inhibition of complement alternative pathway activity, and a favorable safety profile.
ADCs
Glenmark Inks $1.1B Deal for Rights to Enhertu Rival ADC | Commercial
Glenmark has struck a deal with Jiangsu Hengrui to license rights in emerging markets (excluding the U.S., Europe, Japan, China, etc.) to Hengrui’s HER2‑targeted ADC trastuzumab rezetecan as part of a potential $1.1 billion deal. The agreement includes an $18 million upfront payment plus up to $1.09 billion in regulatory/commercial milestones, along with sales‑based royalties. Hengrui positions rezetecan as a direct competitor to AstraZeneca/Daiichi’s Enhertu, noting that both ADCs use trastuzumab as the targeting antibody but differ in the payload (rezetecan vs. deruxtecan). Trastuzumab rezetecan was approved in China in May 2025 for HER2‑mutant non‑small cell lung cancer, and a breast cancer filing is under review in China; Hengrui also recently received orphan drug designation from the FDA in a different indication. The deal aligns with Glenmark’s strategy to build out its oncology pipeline, while Hengrui aims to expand access to its novel therapies in markets beyond the major developed regions.
Genmab Discontinues Previously Acquired ADC Candidate from ProfoundBio | Commercial
Genmab has discontinued development of GEN1107, an ADC targeting PTK7, citing an unsatisfactory benefit-risk profile. The drug had been acquired through Genmab’s $1.8 billion acquisition of ProfoundBio and was in a Phase 1/2 trial for advanced solid tumors. That trial, which began in January 2024, was halted on August 18, 2025. Despite dropping GEN1107, Genmab continues to develop other assets from the deal, including Rina-S (rinatabart sesutecan), GEN1160, and additional preclinical programs. The decision comes as Genmab faces legal challenges from AbbVie, which alleges that ProfoundBio used stolen trade secrets from a former AbbVie scientist to develop its ADCs.
Merck and Daiichi Sanko Deliver Promising Data for I-Dxd | Clinical Trial
Merck and Daiichi Sankyo presented data from their Phase II IDeate‑Lung01 study showing that their ADC ifinatamab deruxtecan (I‑DXd) achieved a confirmed objective response rate of 48.2% in patients with pretreated extensive‐stage small cell lung cancer, including three complete responses and 63 partial responses, with an additional 54 patients showing stable disease. The observed ORR is lower than their earlier topline figure of 54.8%, which analysts suggest was due to inclusion of patients in a third‑line setting diluting efficacy results. In a subset of patients treated in the second‑line setting, the ORR rose to 56.3%, which analysts described as “impressive.” Because I‑DXd already has Breakthrough Therapy Designation from the FDA, these data increase the possibility of accelerated approval in this indication. The readout represents a strong showing for the Merck–Daiichi ADC collaboration and may help revitalize momentum for their ADC portfolio.
BioNTech and DualityBio Announce Phase 3 Data of ADC Candidate in HER2-Positive Breast Cancer | Clinical Trial
BioNTech and DualityBio reported that their ADC candidate trastuzumab pamirtecan (BNT323/DB‑1303) met its primary endpoint of progression‑free survival in an interim analysis of a Chinese Phase 3 trial comparing it against T‑DM1 in patients with HER2‑positive unresectable or metastatic breast cancer who have previously received trastuzumab and a taxane-based chemotherapy. The trial enrolled 228 patients across 48 sites and randomized them 1:1 to receive either trastuzumab pamirtecan or T‑DM1; secondary endpoints include overall survival, response rates, duration of response, and safety. Under their collaboration (launched in April 2023), BioNTech retains commercial rights globally (excluding mainland China, Hong Kong, Macau), while DualityBio holds rights in those Chinese territories. This marks the first late‑stage oncology program in their partnership to achieve a pivotal trial success. The partners plan to explore applications of this ADC in other tumor types and regions, including potential filings in the U.S. and EU.
Radiance Biopharma Licenses Rights to First in Class Nano Antibody ADC | Commercial
Radiance Biopharma has inked an exclusive license agreement with Novatim Immune Therapeutics to develop and commercialize RB‑601 (KY‑0301), a bispecific nanobody ADC targeting both c‑MET and EGFR. Under the deal, Radiance will pay a $15 million upfront fee and may pay up to $150 million in development/regulatory milestones plus more than $1 billion in commercial milestones, along with tiered royalties. The agreement grants Radiance global rights (excluding mainland China, Hong Kong, Macau, and Taiwan), with Radiance responsible for clinical development in its licensed territories. RB‑601 uses site‑specific conjugation to have a homogeneous drug‑to‑antibody ratio of 4 and employs “tuned down” EGFR affinity to improve safety; it releases an MMAE payload upon internalization. The ADC has already cleared IND applications with China’s NMPA and the U.S. FDA and is being tested in a Phase 1 trial in China.
