.png)
Highlights & Summary
Intro
The year ended with exciting news across modalities, particularly regarding promising results in clinical trials – including for an oligo with an Orphan Drug Designation.
What do you expect to occur in 2025? Please take our attached CGT Sentiment Survey with your thoughts!
Happy Reading!
Take DeciBio's 2025 CGT Sentiment Survey Today
Cell Therapy
- Mesoblast Secures Landmark Approval for Cell Therapy | Regulatory
Gene Therapy
- Capsida Releases New Preclinical Data on Epilepsy Candidate | Clinical Trial
- HuidaGene Doses First Patient in MECP2 Duplication Syndrome Trial | Clinical Trial
- SpliceBio Receives IND Clearance for Stargardt Study | Clinical Trial
- Atsena Completes Part A Dosing of Retinoschisis Trial | Clinical Trial
- Astellas and Sangamo Link up for Brain Delivery | Partnership
Oligo Therapy
- Divesiran Demonstrates Efficacy in Reducing Phlebotomy Need for Polycythemia Vera Patients | Clinical Trial
- Ractigen Initiates Phase I Trial of siRNA Therapy for SOD1-ALS | Clinical Trial
Antibody-Drug Conjugates
- Merck’s Zilovertamab Vedotin in Combination with R-CHP Demonstrates 100% Complete Response Rate in DLBCL | Clinical Trial
Cell Therapy
Altaris to Acquire WuXi AppTec’s U.S. and UK Cell Therapy Business | M&A
Altaris Capital Partners is set to acquire WuXi AppTec’s U.S. and U.K.-based cell therapy manufacturing business, marking a key move in the private equity firm’s healthcare-focused portfolio. The decision comes as the Biosecure Act gains traction in the U.S., threatening to restrict American biotechs from partnering with Chinese manufacturing contractors, including WuXi AppTec and WuXi Biologics. The deal is expected to close in the first half of 2025, with the business set to be rebranded and headquartered in the U.S.
Mesoblast Secures Landmark Approval for Cell Therapy | Regulatory
Mesoblast has received FDA approval for its cell therapy, remestemcel-L, for treating children under 12 with steroid-refractory acute graft-versus-host disease (aGVHD). This is the first MSC product approved by the FDA for any indication. The approval, a significant milestone for the Australian biotech, follows positive Phase 3 trial results showing that remestemcel-L improved survival and reduced the need for additional immunosuppressive therapy in pediatric patients. This long-awaited decision follows years of delays and setbacks, marking a critical milestone for the company.
BMS Reshapes Pipeline with Two Cell Therapy Deal Cancellations | Partnership
According to separate SEC filings, Bristol Myers Squibb (BMS) is cutting partnerships with Century Therapeutics and Immatics. These terminations, which include a significant deal centered around Century’s induced pluripotent stem cell (iPSC)-derived therapies, are part of a broader effort by BMS to reassess its R&D strategy. While the company remains invested in cell therapy, these cancellations reflect a prioritization of other areas in the pipeline in alignment with their plans to cut $1.5B in costs, announced in April. The reshuffling comes as BMS aims to simplify its portfolio and sharpen its focus on existing areas.
Arcellx Reports Positive iMMagine-1 Data for Multiple Myeloma Treatment | Clinical Trial
Arcellx has announced positive new data from its iMMagine-1 study, evaluating its novel cell therapy, ACR-19, in patients with relapsed or refractory multiple myeloma. The data demonstrated 97% ORR and 62% CR/sCR and no observed neurotoxic adverse events in both Phase 1 and Phase 2 studies. Additionally, the data show a 30.2 month median PFS. These findings support the potential of ACR-19 as a next-generation treatment for multiple myeloma and a potential direct competitor to Legend and J&J’s Carvykti. The results strengthen Arcellx’s ongoing efforts to bring its innovative therapies to market and offer new hope for patients with limited treatment options.
Gilead Reports Long-Term Data for KITE’s Tecartus CAR-T Therapy at ASH 2024 | Clinical Trial
Gilead presented the longest follow-up data for its Tecartus CAR-T cell therapy at the 2024 ASH Annual Meeting, reinforcing the treatment’s durable efficacy and survival benefits in patients with relapsed or refractory mantle cell lymphoma (MCL). The data showed that Tecartus continues to deliver significant and lasting therapeutic outcomes, including a 91% ORR and a 73% CRR. These findings further support Tecartus as a robust option in MCL and ALL treatment, particularly for patients with limited alternatives.
Gene Therapy
Capsida Releases New Preclinical Data on Epilepsy Candidate | Clinical Trial
Capsida Biotherapeutics released preclinical data on CAP-002, its intravenous gene therapy candidate for STXBP1-related developmental and epileptic encephalopathy (DEE). CAP-002 demonstrated brain-wide distribution, dose-dependent expression, and limited off-target effects in non-human primates, as well as correction of neuronal network activity in diseased human neurons. Preclinical studies suggest the therapy could address seizures, motor issues, and developmental delays associated with STXBP1-DEE. Capsida plans to initiate clinical trials for CAP-002 in the first half of 2025.
HuidaGene Doses First Patient in MECP2 Duplication Syndrome Trial | Clinical Trial
HuidaGene Therapeutics announced that the first patient has been dosed in the HERO clinical trial testing their RNA-editing therapy for MECP2 duplication syndrome. The trial is testing HG204, a CRISPR RNA-editing therapy that uses a single AAV vector to deliver the company’s proprietary hfCas13Y RNA editor and a guide RNA targeting the MECP2 gene. Preclinical studies showed significant improvements in motor skills, social behavior, and survival in animal models. The therapy has received Orphan Drug and Rare Pediatric Disease designations from the FDA and Orphan Drug designation from the EMA.
SpliceBio Receives IND Clearance for Stargardt Study | Clinical Trial
SpliceBio announced FDA clearance of its investigational new drug application for SB-007, a gene therapy for Stargardt disease that uses Protein Splicing to address mutations in the large ABCA4 gene. SB-007, delivered via an AAV vector, aims to restore full-length ABCA4 protein expression and is the first clinical-stage therapy targeting all ABCA4 mutations. The company plans to begin enrollment for the Phase 1/2 ASTRA study in early 2025 to assess the safety and efficacy of a single subretinal dose. There are several other companies developing AAV therapies for the disease, including Ascidian Therapeutics, who entered the clinic in early 2024.
Atsena Completes Part A Dosing of Retinoschisis Trial | Clinical Trial
Atsena Therapeutics announced the completion of dosing in Part A of its Phase I/II LIGHTHOUSE study evaluating ATSN-201, a gene therapy for X-linked retinoschisis (XLRS). ATSN-201 uses the company’s AAV.SPR spreading capsid to achieve therapeutic gene expression in photoreceptors without requiring foveal detachment. The study has reported structural and functional benefits with no treatment-related serious adverse events thus far.
Astellas and Sangamo Link up for Brain Delivery | Partnership
Astellas Pharma and Sangamo Therapeutics have entered a licensing agreement granting Astellas exclusive worldwide rights to Sangamo’s STAC-BBB capsid, a neurotropic AAV technology that enables potent blood-brain barrier penetration and neuronal transduction. The agreement covers one initial neurological disease target, with the option to license up to four additional targets. Sangamo will receive a $20 million upfront payment and is eligible for up to $1.3 billion in milestone payments and royalties.
Oligo
Factor XI siRNA Shows Promise as Next-Gen Anticoagulant in Phase I Trial | Clinical Trial
Sirius Therapeutics has announced promising preliminary data from its Phase 1 clinical trial of SRSD107, a next-generation siRNA therapeutic for thromboembolic disorders. The trial demonstrated that SRSD107 was safe and well-tolerated, with pharmacokinetic parameters consistent with typical siRNA products. Significant reductions in Factor XI antigen and activity were observed, with maximal reductions exceeding 90% at the highest doses tested. The pharmacodynamic effects were durable, with suppression lasting more than 16 weeks after dosing. The company's approach of targeting Factor XI shows potential for reducing thrombosis without significantly increasing bleeding risk, addressing a major limitation of current anticoagulant drugs.
Silexion Therapeutics and Evonik Initiate siRNA Drug Formulation Collaboration | Partnership
Silexion Therapeutics has revealed an ongoing collaboration with Evonik for the development of an advanced siRNA formulation aimed at enhancing cancer treatment efficacy. The collaboration utilizes Evonik's proprietary biodegradable long-acting PLGA (RESOMER®) microparticle formulation for Silexion's next-generation siRNA candidate, SIL-204. This innovative formulation has shown high efficacy in preclinical models using mice with human pancreatic tumor cell lines carrying various KRAS mutations. The partnership aims to deliver sustained-release RNAi therapy, potentially allowing for more effective targeting of KRAS mutations, which are among the most challenging genetic drivers in oncology. The collaboration highlights the potential of combining Silexion's RNAi expertise with Evonik's formulation capabilities to advance cancer treatments.
Divesiran Demonstrates Efficacy in Reducing Phlebotomy Need for Polycythemia Vera Patients | Clinical Trial
Silence Therapeutics has presented additional results from the Phase 1 open-label portion of the SANRECO study of divesiran in patients with polycythemia vera (PV) at the American Society of Hematology Annual Meeting. The data showed that divesiran substantially reduced phlebotomy frequency and lowered hematocrit levels in PV patients, regardless of baseline hematocrit levels4. The study enrolled 19 PV patients with a combined history of 79 phlebotomies prior to enrollment, and only five phlebotomies occurred during the 18-week treatment period. Divesiran demonstrated a sustained reduction in hematocrit during the treatment period and favorable effects on indices of iron metabolism. The treatment continues to be well-tolerated with no dose-limiting toxicities reported. Based on these results, Silence Therapeutics has dosed the first subject in the Phase 2 portion of the SANRECO study.
AusperBio Secures $73M to Advance Chronic Hepatitis B Therapy Development | Financing
AusperBio, a clinical-stage biotechnology company focused on developing targeted oligonucleotide therapies for chronic hepatitis B (CHB), has successfully completed a USD $73 million Series B financing round. The funding was led by HanKang Capital, with participation from several other investors, including both new and existing backers. This financing follows the company's Series A round completed earlier in the year, demonstrating continued investor confidence in AusperBio's proprietary platform and strategic direction. The proceeds will be used to fund the continued Phase 2 development of AHB-137, AusperBio's lead investigational therapy, supporting clinical studies in China and global trials. Additionally, the funding will facilitate the expansion of the company's therapeutic pipeline and operational capabilities.
Ractigen Initiates Phase I Trial of siRNA Therapy for SOD1-ALS | Clinical Trial
Ractigen Therapeutics has announced the successful dosing of the first patient in the Phase I clinical trial of RAG-17, an innovative siRNA therapy targeting Amyotrophic Lateral Sclerosis (ALS) associated with superoxide dismutase 1 (SOD1) gene mutations. The trial is a randomized, double-blind, placebo-controlled study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of RAG-17 in patients with SOD-ALS. RAG-17 has received Orphan Drug Designation from the FDA and has shown promising results in preclinical studies and an Investigator-Initiated Trial. The therapy utilizes Ractigen's proprietary SCAD™ delivery platform, which conjugates siRNA with an accessory oligonucleotide for enhanced delivery into the central nervous system.
ADCs
Merck’s Zilovertamab Vedotin in Combination with R-CHP Demonstrates 100% Complete Response Rate in DLBCL | Clinical Trial
Merck & Co.'s ROR1-directed antibody-drug conjugate (ADC), zilovertamab vedotin, which was acquired in its $2.75B VelosBio acquisition, showed a 100% complete response rate at the lowest dose in a Phase 2 study for diffuse large B-cell lymphoma (DLBCL). Safety concerns, including serious treatment-related adverse events (TRAEs) in 58% of patients, informed the decision to advance the low dose into phase 3 trials. The trial will compare the ADC plus R-CHP to standard-of-care R-CHOP, aiming for improved progression-free survival and response durability. With a 12-month duration of response (DOR) at 93.5%, Merck hopes to outperform R-CHOP, which cures over 60% of patients but leaves room for improvement.
Gilead and Tubulis Enter into Exclusive License Agreement to Develop ADC Candidate | Partnership
Gilead is partnering with German biotech Tubulis to develop antibody-drug conjugates (ADCs) targeting solid tumors, focusing on a topoisomerase I inhibitor-based ADC candidate. The deal includes $20 million upfront, a $30 million option fee, and up to $415 million in milestone payments, plus royalties. Tubulis will lead early-stage research using its Tubutecan and Alco5 platforms, which have shown tumor-shrinking potential in models of ovarian, lung, and triple-negative breast cancers. Gilead will handle later development and commercialization, aiming to enhance its oncology pipeline with Tubulis’ novel, toxicity-reducing ADC technology. Tubulis previously partnered with Bristol Myers Squibb, highlighting growing interest in its approach to improving ADC safety and efficacy for solid tumors.
Daiichi Sankyo to Invest $152M in New ADC Manufacturing Plant in China | Manufacturing
Daiichi Sankyo is investing $152 million to establish its first antibody-drug conjugate (ADC) manufacturing facility in Shanghai, reflecting its long-term commitment to China’s market. The move aligns with Enhertu’s inclusion in China’s national insurance program, which required significant price concessions, and its expanding approvals for various HER2-related cancers. The Shanghai plant, expected to be operational by 2030, will produce ADCs for the Chinese market, including Daiichi and AstraZeneca’s TROP2-targeted datopotamab deruxtecan, currently under regulatory review. This investment follows Daiichi’s larger $1 billion expansion in Germany, where oncology and ADC manufacturing are also priorities. Meanwhile, AstraZeneca, Daiichi’s partner on Enhertu, faces scrutiny in China over alleged employee misconduct, though the investigation has not implicated the company itself.
Elevation Oncology Licenses ADC Technology from Synaffix to Drive Pipeline Expansion | Partnership
Elevation Oncology has partnered with Lonza’s Synaffix to develop its HER3-directed antibody-drug conjugate (ADC), leveraging Synaffix’s conjugation, polar spacer, and toxSYN linker-payload technologies. The deal, valued at up to $368 million, aims to create a differentiated ADC in a competitive field led by Merck and Daiichi Sankyo, whose HER3 ADC is already in phase 3 trials. Elevation, still in preclinical stages, anticipates a 12- to 18-month timeline for an IND filing, potentially starting human trials by 2026. CEO Joseph Ferra highlighted the preclinical promise of their MMAE payload-based ADC, emphasizing its potential to stand out in a market dominated by topo-based payloads. The collaboration underscores Elevation’s commitment to carving a niche in the HER3 ADC space despite the head start of competitors.
Aadi Bioscience Pivots to Pre-Clinical ADCs After Selling Approved Drug | Commercial
Aadi Bioscience has pivoted from a commercial-stage company to a preclinical biotech by selling its approved product, Fyarro, and licensing three antibody-drug conjugates (ADCs) for $44 million upfront. The shift follows Fyarro's failure in a solid tumor trial, leading to layoffs of 80% of Aadi’s staff and a strategic review. The licensed ADCs target PTK7, MUC16, and SEZ6, addressing cancers such as non-small cell lung cancer, female-origin cancers, and neuroendocrine tumors. While these ADCs trail rival candidates, Aadi’s $200 million from the Fyarro sale and financing provides runway until late 2028, with plans to deliver clinical data before further fundraising. The move positions Aadi to compete in a growing ADC field, with significant milestones and commercial potential ahead.
.png)
