Insights provided by DeciBio, a strategy consultancy focused on the life science and biopharma industry.

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Highlights & Summary

The start to 2026 includes an abundance of financing and regulatory updates across the next‑generation therapeutics landscape. In Cell Therapy, Cellares secured a $257M Series D to scale and globalize automated cell therapy manufacturing. Relevant to both cell and gene therapies, the FDA outlined a more flexible, risk‑based approach to overseeing CMC requirements for cell and gene therapies given the unique complexities of their manufacturing. Gene Therapy news included FDA IND clearance for AskBio’s AAV gene therapy AB‑1009 for late‑onset Pompe disease, IND clearance for Siren Biotechnology’s inaugural AAV‑based immuno‑gene therapy for recurrent high‑grade glioma, and an FDA Fast Track designation for VectorY Therapeutics’ ALS candidate VTx‑002. The Oligo sector featured a new partnership between Bayer and Soufflé Therapeutics to develop a heart‑targeted siRNA therapy for a rare form of dilated cardiomyopathy, as well as positive phase 3 data from GSK’s B‑Well 1 and B‑Well 2 trials of the antisense oligonucleotide bepirovirsen in chronic hepatitis B. Within ADCs, Fortitude Biomedicines launched with a $13M seed financing round to advance its lead immune cell‑targeting biologic through IND‑enabling studies and further develop its GLUE‑DAC ADC platform, and MediLink signed a licensing agreement with Roche for its B7‑H3‑targeting ADC YL201, which includes up to $570M in upfront and near‑term milestone payments.

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Cell Therapy

Cellares Secures $257M Series D to Scale Automated Global Cell Therapy Manufacturing | Financing

Cellares announced a $257 million Series D financing round co-led by BlackRock and Eclipse, bringing the company’s total capital raised to $612 million and expanding its investor base to include T. Rowe Price, Baillie Gifford, Duquesne Family Office, Intuitive Ventures, EDBI, and Gates Frontier. The funding is designated to the global build-out of automated IDMO Smart Factories in South San Francisco, Bridgewater, Leiden, and Kashiwa City to support commercial-scale manufacture of cell therapies for hundreds of thousands of patients annually. Cellares’ Cell Shuttle and Cell Q quality control system aim to replace manual contract manufacturing with high-throughput, GMP-compliant automation, offering up to ~10x higher throughput and lower per-patient cost versus conventional CDMOs. The company expects to begin supporting clinical manufacturing in the first half of 2026, with commercial-scale production slated for 2027, and highlights existing agreements such as a $380 million global manufacturing pact with Bristol Myers Squibb.

FDA Increases Flexibility on CMC Requirements to For Cell and Gene Therapies | Regulatory

The FDA announced a more flexible approach to overseeing CMC requirements for cell and gene therapies, aiming to better align regulatory expectations with the unique scientific and manufacturing complexities of these products and to help expedite BLA planning. The FDA highlighted that its Center for Biologics Evaluation and Research is formalizing flexibilities that were previously applied on a case-by-case basis to increase clarity for sponsors. Key aspects include tailoring traditional CMC oversight to accommodate small-batch, individualized therapies while maintaining similar standards for safety, purity, and potency, and proactively communicating these flexibilities broadly across the field. FDA leaders emphasized that these “common-sense reforms” are meant to reduce barriers and perceived misconceptions that could slow innovation, with ongoing engagement through initiatives such as a Cell and Gene Therapy Roundtable.

Imviva Biotech’s CTD402 Receives FDA Orphan Drug Designation for Relapsed / Refractory T-ALL / LBL | Regulatory, Clinical Trial

Imviva Biotech announced that the FDA has granted orphan drug designation to its investigational allogeneic anti-CD7 CAR-T therapy, CTD402, for the treatment of relapsed or refractory T-cell acute lymphoblastic leukemia / lymphoblastic lymphoma (T-ALL / LBL). CTD402 is currently being evaluated in the global Phase 1b / 2 TENACITY-01 trial, which is enrolling adolescents and adults to assess safety, efficacy, and cellular pharmacokinetics, with about 54 patients planned across the U.S., EU, and APAC. The therapy’s platform is designed to eliminate typical autologous manufacturing delays, and early data show a 64.1% complete remission rate and 91.7% MRD-negative status in heavily pretreated patients. The first U.S. patient has already been dosed, with interim Phase 1b data expected by mid-2026 and study completion targeted for late 2028, and CTD402 also holds Rare Pediatric Disease and RMAT designations.

Multiply Labs and AstraZeneca Partner to Automate Cell Therapy Manufacturing | Manufacturing

Multiply Labs announced a new collaboration with AstraZeneca to evaluate the use of its GMP-ready robotic biomanufacturing systems for commercial-scale cell therapy production, aiming to enhance scalability, throughput, and quality consistency in complex manufacturing. The agreement will focus on applying end-to-end autonomous robotics to operate industry-standard cell therapy instruments in parallel, minimizing manual steps while maintaining regulatory and GMP requirements. Multiply Labs’ platform uses multi-arm robotic clusters designed to integrate with existing equipment and enable high-throughput processing without extensive process modifications. AstraZeneca’s involvement brings scientific and clinical expertise to assess how autonomous automation can address major manufacturing bottlenecks in cell therapy production.

ImmunityBio Reports 15-Month Durable Complete Response with Chemotherapy-Free CD19 CAR-NK in Waldenström Lymphoma | Clinical Trial

ImmunityBio reported updated efficacy from the ongoing Phase 1 QUILT-106 trial of its off-the-shelf allogeneic CD19 CAR-NK cell therapy administered in combination with rituximab in patients with Waldenström non-Hodgkin lymphoma. In four treated patients who received eight doses of the CAR-NK therapy and rituximab without lymphodepletion or chemotherapy, a 100% disease control rate was observed, with two patients maintaining complete remission at 7 and 15 months after the final dose. The therapy was delivered entirely in an outpatient setting with no serious adverse events reported, highlighting a favorable safety profile and operational convenience compared to traditional CAR-T regimens. Notably, a patient with ~95% bone marrow tumor infiltration achieved complete bone morphological remission that has persisted for 15 months with no additional treatment.

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Gene Therapy

AskBio Clears IND for Pompe Gene Therapy AB-1009 | Regulatory

AskBio said the FDA has accepted its IND application for AB-1009, an AAV gene therapy for late-onset Pompe disease, enabling initiation of a Phase 1/2 clinical trial in the U.S. The company expects to dose its first patient in early 2026, and the program has also received Fast Track and Orphan Drug designations.

VectorY Wins FDA Fast Track for ALS Antibody Therapy | Regulatory

VectorY Therapeutics announced that the FDA granted Fast Track designation to VTx-002, a vectorized antibody therapy targeting TDP-43 pathology in ALS. The designation follows recent IND clearance, with a Phase 1/2 PIONEER-ALS study expected to begin dosing in early 2026.

Beacon Therapeutics Raises $75M+ in Oversubscribed Series C | Financing

Beacon Therapeutics closed an oversubscribed Series C financing of more than $75 million, led by Life Sciences at Goldman Sachs Alternatives, with participation from new and existing investors. The funds will support completion of pivotal studies and commercial preparation for laru-zova in X-linked retinitis pigmentosa, as well as pipeline expansion.

Siren Biotechnology Enters Clinic With First Oncology IND | Regulatory

Siren Biotechnology said the FDA has cleared its first IND, allowing the company to begin a first-in-human trial of an AAV-based immuno-gene therapy for recurrent high-grade glioma. The clearance marks Siren’s transition to a clinical-stage company and supports evaluation of its localized gene therapy approach in oncology.

Sarepta Reports Durable Three-Year Benefit for ELEVIDYS in Duchenne | Clinical Trial

Sarepta Therapeutics reported positive three-year functional data from the Phase 3 EMBARK study of ELEVIDYS in ambulatory boys with Duchenne muscular dystrophy, showing sustained improvements vs. an external control across multiple motor measures. No new safety signals were identified, and the results extend earlier findings supporting long-term disease modification.

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Oligo

Bayer & Soufflé Therapeutics Partner on siRNA for Dilated Cardiomyopathy | Partnership

Bayer and Soufflé Therapeutics announced a strategic collaboration and global licensing agreement to advance a cell-specific, heart-targeted small interfering RNA (siRNA) therapy aimed at a rare subset of dilated cardiomyopathy. The program focuses on delivering siRNA specifically to cardiomyocytes using Soufflé’s cell-selective ligand and delivery technologies, with financial terms not disclosed.

GSK’s Bepirovirsen Hits Phase 3 Endpoints, Setting Up Regulatory Push | Clinical Trial

GSK reported that two pivotal Phase 3 trials (B-Well 1 and B-Well 2) of bepirovirsen, an antisense oligonucleotide partnered with Ionis, met their primary endpoints in chronic hepatitis B across 1,800+ patients in 29 countries. The studies showed statistically significant improvements in “functional cure” rates (defined as HBsAg loss and undetectable HBV DNA for at least 24 weeks after treatment) for bepirovirsen plus standard of care versus standard of care alone in patients with baseline HBsAg <3,000 IU/ml, with stronger effects noted below 1,000 IU/ml. GSK said it plans to submit to regulators in Q1 2026 and described safety/tolerability as consistent with prior studies.

Sanofi Deprioritizes Its mRNA Seasonal Flu Vaccine Program | Clinical Trial

Sanofi disclosed it is scrapping/deprioritizing its Phase 1-stage mRNA seasonal influenza vaccine program and does not anticipate launching an mRNA seasonal flu product in the near term. The company said it remains committed to mRNA as part of a broader vaccine strategy and continues an mRNA H5 pandemic flu vaccine study (Phase 1/2) that it characterized as having encouraging preliminary data. Sanofi framed the move as a broader strategic shift rather than an isolated decision, after earlier efforts where first-generation hemagglutinin mRNA flu candidates performed better against influenza A than B.

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PLaN Therapeutics Licenses Allele-Specific siRNA Therapy | Partnership

UMass Chan Medical School licensed technology from the lab of RNA therapeutics researcher Anastasia Khvorova to PLaN Therapeutics to develop an allele-specific siRNA-based therapy for PLN‑R14del genetic cardiomyopathy, with PLaN retaining exclusive rights. Under the collaboration, PLaN will work with the inventors to optimize chemical modifications so the siRNA selectively silences the mutant PLN allele without affecting the healthy allele, aiming to improve durability, tissue distribution, and target engagement. The teams described the goal as enabling delivery to the heart with a potentially patient-friendly subcutaneous regimen around once every 2–3 months.

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Corsera Health Announces $80M Series A for RNAi Medicines  | Financing

Corsera Health raised an oversubscribed $80 million Series A co-led by Forbion and Population Health Partners, with participation including Corsera co-founder/co-CEO John Maraganore. The company said proceeds will fund its prevention-focused RNAi program for cardiovascular disease and support development of its AI-enabled risk prediction platform, and it also announced initiation of dosing in a Phase 1 trial of COR-1004, a subcutaneously administered siRNA targeting PCSK9.

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ADCs

Fortitude Biomedicines Launches with $13M Financing for Autoimmune Diseases and Cancer | Financing

Fortitude Biomedicines has launched with a 13 million dollar seed financing round co-led by K2 Bio Partners, Shanghai Healthcare Angel Capital, and Elikon Venture, with additional investors Everjoy Fortune and Taihill Venture. The company is developing immune cell–targeting biologics and a next-generation GLUE-DAC antibody–drug conjugate (ADC) platform that uses proprietary molecular glue payloads to couple ADC precision with targeted protein degradation. Proceeds will advance its lead immune cell–targeting biologic through IND-enabling studies and support a broader pipeline in oncology and autoimmune diseases. GLUE-DAC is designed to expand ADCs’ therapeutic window, overcome resistance mechanisms, and unlock new therapeutic targets by providing payloads with distinct mechanisms of action. 

MediLink Inks $570M Licensing Deal with Roche for B7H3 ADC | Commercial

MediLink Therapeutics has entered a new collaboration and exclusive licensing agreement with Roche for YL201, a B7H3-targeting antibody-drug conjugate being developed for multiple solid tumors. Roche receives exclusive rights to develop, manufacture, and commercialize YL201 worldwide except in mainland China, Hong Kong, and Macau, while MediLink will receive 570 million dollars in upfront and near-term milestones plus additional milestones and tiered royalties on ex-China sales. The deal builds on a prior 2024 collaboration around the YL211 c-Met ADC and aims to use both companies’ strengths to accelerate YL201 toward global regulatory approvals and broad patient access. YL201, built on MediLink’s TMALIN platform, is in multinational trials and has entered two Phase III registrational studies in China for small cell lung cancer and nasopharyngeal carcinoma, where early data show promising response rates and survival in second-line SCLC. The FDA granted YL201 Breakthrough Therapy Designation for SCLC in June 2025, adding to earlier Orphan Drug Designations in SCLC, NPC, and esophageal squamous cell carcinoma. 

Genmab Pauses Enrollment in Early ADC Trial from ProfoundBio | Clinical Trial

Genmab has halted enrollment in an early-stage trial of GEN1286, an experimental dual-target antibody-drug conjugate it acquired through the €1.52 billion purchase of ProfoundBio, while keeping the study open for existing participants. The trial, originally designed for about 214 patients with advanced solid tumors, stopped recruitment after only 23 patients enrolled, and Genmab has not publicly disclosed the reasons for this decision. GEN1286 is part of a new wave of ADCs that use dual targeting to enhance precision against tumor growth and resistance mechanisms, but this added complexity may contribute to development challenges. The pause occurs as Genmab broadens its ADC portfolio, including the planned 8-billion-dollar acquisition of Merus and prior ProfoundBio-derived ADCs GEN1160 and GEN1107, which have already been discontinued after early development hurdles.

Genmab Announces Topline Results from Phase 3 EPCORE DLBCL-1 Trial | Clinical Trial

Genmab reported topline results from the Phase 3 EPCORE DLBCL-1 trial evaluating epcoritamab (DuoBody CD3xCD20) monotherapy versus investigator’s choice chemotherapy in relapsed/refractory diffuse large B-cell lymphoma (DLBCL) patients ineligible for high-dose chemotherapy and autologous stem cell transplant. The trial met its primary endpoint, showing a statistically significant improvement in progression-free survival with epcoritamab, along with favorable trends in complete response rate, duration of response, and time to next therapy compared with R-GemOx or BR chemotherapy regimens. Overall survival showed a hazard ratio below 1 but did not reach statistical significance at this analysis. The global, open-label study enrolled 483 patients and is the first Phase 3 trial to demonstrate a PFS benefit for a CD3xCD20 T-cell–engaging bispecific antibody monotherapy in this setting. Genmab and AbbVie plan to present full data at a future medical meeting and will engage global regulators to discuss next steps while continuing broader development of epcoritamab across multiple B-cell malignancies and lines of therapy.

Gilead Announces Positive Phase 3 Data in 1L PD-L1+ Metastatic TNBC | Clinical Trial

The Phase 3 ASCENT-04/KEYNOTE-D19 trial showed that Trodelvy (sacituzumab govitecan-hziy) plus Keytruda (pembrolizumab) significantly improved progression-free survival versus standard-of-care Keytruda plus chemotherapy in first-line PD-L1+ (CPS ≥10) metastatic triple-negative breast cancer, reducing the risk of disease progression or death by 35% (HR 0.65; p<0.001). Median PFS was 11.2 months with Trodelvy plus Keytruda compared with 7.8 months for Keytruda plus chemotherapy. The safety profile of the combination was consistent with that of each individual drug, with no new safety signals and a lower rate of treatment discontinuation due to adverse events versus the chemotherapy-containing control arm (12% vs 31%). The most frequent grade ≥3 adverse events with the combination were neutropenia (43%) and diarrhea (10%), while in the control arm they were neutropenia (45%), anemia (16%), and thrombocytopenia (14%). Trodelvy is a first-in-class Trop-2-directed ADC already approved in many countries for later-line metastatic TNBC and HR+/HER2- metastatic breast cancer, and ASCENT-04 supports its potential as a new first-line standard of care in PD-L1+ metastatic TNBC when combined with Keytruda.

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