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Insights provided by DeciBio, a strategy consultancy focused on the life science and biopharma industry.
Highlights & Summary
Intro
As we move into the second quarter of the year, the Next-Gen therapeutics space is buzzing with clinical trial updates and exciting new partnerships.
Happy Reading!
Cell Therapy
- Orca Bio’s T-Cell Therapy Shows Potential to Reduce Transplant Complications in Blood Cancer | Clinical Trial
Gene Therapy
- MeiraGTx Announces Joint Venture with halogen Neuro AI | Partnership
- REGENXBIO Reports Positive Interim Data for RGX-202 in DMD | Clinical Trial
- AskBio Advances Phase 1/2 Trial of AB-1003 Gene Therapy for Limb-Girdle Muscular Dystrophy | Clinical Trial
- AAVantgarde Announces Presentations at ARVO 2025 Annual Meeting | Clinical Trial
- Tenaya Therapeutics Announces Updated Preclinical Data for TN-201 | Clinical Trial
Oligo Therapy
- Alnylam Pharmaceuticals Secures FDA Approval for Amvuttra (Vutrisiran) for treating ATTR-CM | Regulatory
- Arrowhead Pharmaceuticals Highlights Two Clinical-Stage RNAi Therapeutics for Obesity and Metabolic Diseases | Clinical Trial
- Avidity Biosciences Reports Positive Topline Data for Del-zota in Duchenne Muscular Dystrophy | Clinical Trial
- Ono Pharmaceutical Enters License Agreement with Ionis Pharmaceuticals for Sapablursen | Partnership
Antibody-Drug Conjugates
- Japanese Ministry of Health Approves Genmab’s TIVDAK | Regulatory
- GV20 Therapeutics Announces Collaboration with Mitsubishi Tanabe Pharma for ADC Development | Partnership
- FibroGen Announces Publication of Phase 1 Monotherapy Study for Prostate Cancer Patients | Clinical Trial
Cell Therapy
AstraZeneca to Acquire EsoBiotec for $425M to Bolster In Vivo Cell Therapy Pipeline | M&A
AstraZeneca announced it will acquire cell therapy company EsoBiotec for $425 million upfront, with additional milestone payments potentially increasing the deal’s value. EsoBiotec specializes in in vivo CAR-T therapies, which use lentiviral vectors to reprogram T cells directly inside the patient’s body, offering a potentially more scalable and cost-effective alternative to traditional ex vivo CAR-T approaches. The acquisition aims to strengthen AstraZeneca’s cell therapy pipeline, particularly in hematologic malignancies. The deal is expected to close in the first half of 2025, subject to regulatory approvals.
Bristol Myers Squibb Acquires 2seventy bio for $95M, Gaining Full Rights to Abecma | M&A
Bristol Myers Squibb (BMS) announced a definitive agreement to acquire 2seventy bio for $95 million, gaining full ownership of the BCMA-directed CAR-T therapy Abecma (ide-cel). BMS and 2seventy bio previously co-developed Abecma, which was the first CAR-T therapy approved for relapsed or refractory multiple myeloma. The acquisition is expected to streamline Abecma’s commercialization and allow BMS to accelerate the development of next-generation cell therapies. The transaction is anticipated to close in the second quarter of 2025.
FDA Grants Fast Track Designation to Novel CAR-T Therapy for Relapsed/Refractory DLBCL | Regulatory
Imugene announced that the FDA has granted Fast Track designation to its allogeneic CAR-T cell therapy, azer-cel, for patients with relapsed or refractory CD19-positive blood cancers. Azer-cel is designed for patients who have relapsed following autologous CAR-T treatment and uses donor-derived T cells engineered to target CD19. The designation is intended to accelerate the therapy's development and review process. Imugene stated that the Fast Track status will support its ongoing clinical program and facilitate more frequent interactions with the FDA, rolling submission review, and potential for eligibility for Accelerated Approval and Priority Review.
Orca Bio’s T-Cell Therapy Shows Potential to Reduce Transplant Complications in Blood Cancer | Clinical Trial
Orca Bio announced positive Phase 3 data for its investigational T-cell therapy, Orca-T, in patients with blood cancers undergoing allogeneic stem cell transplants. Orca-T uses a precision cell separation process to deliver a highly purified mix of stem cells and T cells, aiming to reduce graft-versus-host disease (GvHD) while preserving anti-cancer effects. The study met its primary endpoint, with 78% of patients treated with Orca-T being free of GvHD, compared to 38% of those who received conventional allogeneic stem cell transplants. Orca Bio plans submit the data in a BLA to the FDA this year, and results will be presented at the 51st Annual Meeting of the EBMT.
ElevateBio Reduces Workforce by 17% Amid Streamlining Efforts | Personnel
ElevateBio announced it is reducing its workforce by 17% as part of a broader effort to streamline operations and extend its financial runway. The layoffs come as the company focuses on advancing its core cell and gene therapy platforms, including its manufacturing subsidiary, Life Edit Therapeutics. ElevateBio stated that the restructuring will allow it to prioritize key programs and partnerships while maintaining its long-term growth strategy. The company did not disclose the exact number of employees affected.
Gene Therapy
MeiraGTx Announces Joint Venture with halogen Neuro AI | Partnership
MeiraGTx and Hologen Limited have formed a joint venture, Hologen Neuro AI Ltd., to integrate AI-driven insights into the development of gene therapies for neurological disorders. As part of the deal, MeiraGTx transferred exclusive rights to its AAV-GAD and AAV-BDNF investigational gene therapies, along with other CNS-targeted genetic medicines and a proprietary delivery device, in exchange for a $200 million upfront payment and a 30% stake in the venture. Hologen has committed up to $230 million in additional funding to advance these programs through commercialization.
REGENXBIO Reports Positive Interim Data for RGX-202 in DMD | Clinical Trial
REGENXBIO has released new interim data from the Phase I/II AFFINITY DUCHENNE® trial of RGX-202 in DMD. The data showed robust microdystrophin expression in patients as young as one year old, with levels reaching up to 122.3% of control in younger patients. No serious adverse events were reported, and the therapy demonstrated a favorable safety profile. REGENXBIO aims to complete trial enrollment in 2025 and submit a BLA in mid-2026.
AskBio Advances Phase 1/2 Trial of AB-1003 Gene Therapy for Limb-Girdle Muscular Dystrophy | Clinical Trial
AskBio announced the advancement of its Phase 1/2 LION-CS101 clinical trial for AB-1003, an investigational AAV-based gene therapy for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). The trial progressed to its second cohort following a favorable safety review by an independent Data Safety Monitoring Board. AB-1003 is designed to restore FKRP enzyme activity in muscle cells. Sarepta has a competing asset in Ph3 (SRP-9003) and expects to file a BLA this year. Genethon also has a clinical-stage asset for the disease. AskBio has received rare pediatric disease, orphan-drug, and fast track designations from the FDA, and trial enrollment is ongoing.
AAVantgarde Announces Presentations at ARVO 2025 Annual Meeting | Clinical Trial
AAVantgarde Bio will present data from its LUCE-1 clinical trial for Usher syndrome Type 1B and preliminary results from a non-human primate study on Stargardt disease at the ARVO 2025 Annual Meeting. There are several other assets in development for Usher Syndrome from the likes of Atsena and Eli Lilly (Type 3A). These presentations will provide insights into the therapeutic potential of AAVantgarde's gene therapy programs.
Tenaya Therapeutics Announces Updated Preclinical Data for TN-201 | Clinical Trial
Tenaya Therapeutics announced the publication of positive preclinical data for TN-201, its AAV9-based gene therapy for MYBPC3-associated hypertrophic cardiomyopathy, in Nature Communications. Preclinical studies demonstrated that TN-201 increased MyBP-C protein levels in a dose-dependent manner, leading to improvements in cardiac function, reversal of left ventricular hypertrophy, and extended survival in a severe disease model. TN-201 is currently being evaluated in the ongoing MyPEAK-1 Phase 1b/2 clinical trial at doses of 3x10¹³ and 6x10¹³ vg/kg, with additional human trial data expected later in 2025.
Oligo
Alnylam Pharmaceuticals Secures FDA Approval for Amvuttra (Vutrisiran) for treating ATTR-CM | Regulatory
The FDA has approved Alnylam Pharmaceuticals' RNAi therapeutic Amvuttra (vutrisiran) for the treatment of cardiomyopathy associated with wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults. This approval is based on data from the Phase 3 HELIOS-B trial, which demonstrated a reduction in cardiovascular mortality, hospitalizations, and urgent heart failure visits. Amvuttra is administered subcutaneously every three months and works by silencing the transthyretin (TTR) gene, reducing amyloid deposits that cause heart damage. Amvuttra is currently the first FDA-approved treatment for both ATTR-CM and polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN).
Arrowhead Pharmaceuticals Highlights Two Clinical-Stage RNAi Therapeutics for Obesity and Metabolic Diseases | Clinical Trial
Arrowhead Pharmaceuticals showcased its investigational RNAi-based therapeutics, ARO-INHBE and ARO-ALK7, designed to treat obesity and metabolic diseases by targeting pathways that regulate fat storage and lipolysis. Both candidates demonstrated potential to reduce body weight and fat mass while preserving lean muscle mass in preclinical studies. ARO-INHBE silences the hepatic INHBE gene, reducing visceral adiposity and improving glycemic control, while ARO-ALK7 targets adipose ALK7 to suppress fat accumulation and enhance lipid mobilization. Phase 1/2 clinical trials are underway, with dosing for ARO-INHBE initiated in December 2024 and dosing for ARO-ALK7 expected to begin in Q2 2025. Initial data for both studies is anticipated by the end of 2025. Arrowhead also plans to evaluate these therapies in combination with tirzepatide, a GLP-1/GIP receptor co-agonist, to explore additive effects on weight and fat reduction.
Rona Therapeutics Receives FDA IND Clearance for Phase 2 Study of RN0361 | Regulatory
Rona Therapeutics has received Investigational New Drug (IND) clearance from the FDA to initiate a Phase 2 clinical trial for RN0361, an APOC3-targeted siRNA therapeutic for severe hypertriglyceridemia (SHTG), mixed dyslipidemia, and familial chylomicronemia syndrome (FCS). The randomized, double-blind, placebo-controlled study will evaluate the efficacy, safety, and duration of RN0361 over a nine-month follow-up period. RN0361 utilizes Rona’s proprietary GAIA siRNA conjugate platform for hepatocyte-specific silencing via subcutaneous administration. Phase 1 data demonstrated a favorable safety profile and sustained triglyceride reduction following a single dose over six months. The therapy achieves durable gene silencing through optimized chemical modifications, significantly reducing APOC3 mRNA and protein expression. Rona plans to collaborate with global investigators to advance RN0361’s development for patients with significant unmet needs in lipid disorders.
Avidity Biosciences Reports Positive Topline Data for Del-zota in Duchenne Muscular Dystrophy | Clinical Trial
Avidity Biosciences released topline results from its Phase 1/2 EXPLORE44 trial evaluating del-zota, an antibody-oligonucleotide conjugate (AOC), in patients with Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping. The study demonstrated statistically significant improvements, including a 25% increase in dystrophin production, a 40% increase in exon skipping, and an 80% reduction in creatine kinase levels compared to baseline. Safety and tolerability were confirmed across dose cohorts, with participants receiving three doses of either 5 mg/kg or 10 mg/kg del-zota or placebo over six to eight weeks. Del-zota has received Rare Pediatric Disease, Orphan Drug, and Fast Track designations from the FDA. Avidity plans to submit a Biologics License Application (BLA) for accelerated approval by the end of 2025.
Ono Pharmaceutical Enters License Agreement with Ionis Pharmaceuticals for Sapablursen | Partnership
Ono Pharmaceutical has entered a license agreement with Ionis Pharmaceuticals for sapablursen, an investigational RNA-targeted therapeutic for polycythemia vera (PV). Sapablursen, currently in the fully enrolled Phase 2 IMPRSSION study, has received FDA Fast Track and orphan drug designations. Under the agreement, Ono will gain exclusive worldwide rights to develop and commercialize sapablursen, while Ionis will complete the Phase 2 study. Ono will pay $280 million upfront, with up to $660 million in milestone payments and royalties in the mid-teens on annual net sales.
ADCs
Japanese Ministry of Health Approves Genmab’s TIVDAK | Regulatory Approval
The Japanese Ministry of Health has approved Genmab’s ADC, TIVDAK, for advanced cervical cancer—the first ADC approved in Japan for this indication. This decision follows the Phase 3 301 trial, which included over 500 participants, 20% of whom were Japanese. The study showed a 30% reduction in mortality compared to chemotherapy and a two-month improvement in overall survival. TIVDAK introduces a novel mechanism of action for treating advanced or recurrent cervical cancer, which often has poor prognoses.
Roche Invests in ADC Collaboration with Oxford Biotherapeutics | Partnership
Oxford Biotherapeutics (OBT) and Roche have partnered to identify novel oncology targets, combining OBT’s proprietary OGAP-Verify discovery platform with Roche’s drug development expertise. Under the collaboration, targets identified with OBT’s platform will be validated by both companies, while Roche will lead further development and commercialization. OBT will receive $36 million upfront, with potential milestone payments exceeding $1 billion.
GV20 Therapeutics Announces Collaboration with Mitsubishi Tanabe Pharma Corporation for ADC Development | Partnership
GV20 Therapeutics (GV20) has partnered with Mitsubishi Tanabe Pharma Corporation (MTPC) to develop antibody-drug conjugates (ADCs). Under this collaboration, MTPC will leverage GV20’s proprietary antibodies, identified through its STEAD AI platform. STEAD integrates omics data, advanced AI models, and B-cell portfolio insights to better analyze tumor microenvironments and identify therapeutic antibody targets. GV20 is eligible for milestone payments in addition to an upfront payment from MTPC; MTPC will receive exclusive licensing rights to antibodies during the collaboration.
FibroGen Announces Publication of Phase 1 Monotherapy Study for Prostate Cancer Patients | Clinical Trial
FibroGen has accounced publication of results from its Phase I study, FG-324, in patients with metastatic castration-resistant prostate cancer (mCRPC). The study demonstrated anti-cancer activity with an acceptable safety profile, with Phase 2 expected to begin later this year. Side effects were consistent with other MMAE-based ADCs, with an objective response rate of 20% and a median response duration of 7–8 months. The median radiographic progression-free survival was 8.7 months. This is the first clinical trial targeting CD46 in prostate cancer.
Callio Therapeutics Launches with $187 Million Series A Funding | Financing
Callio Therapeutics has launched with the closing of a $187 million Series A financing round, led by Frazier Life Sciences and supported by investors including Jeito Capital, Novo Holdings A/S, Omega Funds, and ClavystBio. The company is focused on developing innovative multi-payload antibody-drug conjugates (ADCs) to enhance cancer treatment. Unlike traditional ADCs that deliver a single cytotoxic payload, multi-payload ADCs incorporate multiple mechanisms of action, potentially improving efficacy. Callio has also secured an exclusive worldwide licensing agreement with Hummingbird Biosciences for its ADC platform and additional intellectual property, in exchange for equity, future milestone payments, and royalties. The company's leadership includes experts from companies such as SeaGen, Genentech, and ProfoundBio.
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