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Highlights & Summary
Intro
October included exciting developments across the Next-Gen therapeutics landscape, including new partnerships throughout various modalities and regulatory updates for ADCs.
Cell Therapy
- New York State Commits $150M in Next Phase of Long Island’s Cell and Gene Therapy Hub | Manufacturing
- Adva Biotechnology to Incorporate OpenCell’s Transfection Technology Into Decentralized Manufacturing Platform | Partnership
- AvenCell Therapeutics Secures $112M in Series B to Advance Universal Switchable CAR-T Platform | Financing
Gene Therapy
- Roche Expands Partnership with Dyno Therapeutics | Partnership
- Neurogene to Present Updated Data on Rett Syndrome Candidate | Clinical Trial
- REGENXBIO Provides Updated Data on Wet AMD Candidate | Clinical Trial
Oligo Therapy
- Judo Bio Debuts with $100M in Series A and Presents Receptor Mediated Oligonucleotide Delivery to Knockdown Genes in the Kidney | Company Creation
- Lilly and insitro Partner to Develop AI-Powered siRNA Therapies for Metabolic Diseases | Partnership
- City Therapeutics Launches with $135M in Series A Funding to Develop RNAi-Based Therapies | Company Creation
Antibody-Drug Conjugates
- Ono Enters into License Agreement for LCB97, and to Generate Novel ADC Candidates with LigaChem Biosciences | Partnership
- Adcentrx Announces FDA Clearence of its IND Application for a Potential First-in-Class ADC Targeting STEAP1 | Regulatory
- Enhertu Granted Priority Review in the U.S. for Patients with HER-low or HER2-ultralow Metastatic Breast Cancer | Regulatory
Cell Therapy
New York State Commits $150M in Next Phase of Long Island’s Cell and Gene Therapy Hub | Manufacturing
BioGenesis Park, a $430M cell and gene therapy hub in Long Island, is progressing with New York’s $150M commitment—the largest state investment in a CGT hub nationwide. Phase One will include a 331,000-square-foot facility with CGT and CDMO towers. Upon completion, the park will span 700,000 square feet, featuring spaces for public engagement, research, manufacturing, and collaboration. The hub is expected to create around 700 jobs in CGT research, development, and operations.
Adva Biotechnology to Incorporate OpenCell’s Transfection Technology Into Decentralized Manufacturing Platform | Partnership
OpenCell has partnered with Adva Biotechnology to integrate its Softporation™ technology with Adva’s ADVA X3 platform. This non-exclusive commercial agreement aims to streamline cell therapy production by combining a scalable, gentle transfection process with an automated, end-to-end manufacturing platform. The companies plan to jointly develop and commercialize this offering.
Immatics Advances TCR-T Therapy For Melanoma Patients With Promising Phase 1b Data | Clinical Trial
Immatics released Phase 1b data on its ACTengine® IMA203 TCR-T therapy targeting PRAME in certain pretreated melanoma patients. The trial demonstrated a 54% objective response rate and a median progression-free survival of 6 months, with deep responders showing a median PFS of over 1 year. With these results, Immatics plans to proceed directly to a registration-enabling Phase 3 trial, “SUPRAME,” which plans to commence in December 2024 and conclude in 2026.
March Biosciences Secures $24.8M to Advance CAR-T Therapy for T-Cell Lymphoma | Financing
March Biosciences has raised $28.4 million in an oversubscribed Series A round, bringing their total funding to over $51 million. The round, led by Mission BioCapital and 4BIO Capital, will fund Phase 2 development of their CD5 CAR-T therapy, “MB-105,” aimed at treating relapsed / refractory T-cell lymphoma. With positive safety and efficacy data from Phase 1, the next phase 2 is set to begin in early 2025. The funding will also support the expansion of their pipeline, alongside a new partnership with Volnay Therapeutics to scale manufacturing processes.
AvenCell Therapeutics Secures $112M in Series B to Advance Universal Switchable CAR-T Platform | Financing
AvenCell Therapeutics, a clinical-stage company, has raised $112M in a Series B round led by Novo Holdings, with new investors F-Prime capital, Eight Roads Ventures Japan, Piper Heartland Healthcare Capital, and NYBC Ventures. The funding will help support clinical validation of AvenCell’s universal switchable CAR-T platform, which gives doctors more control by allowing CAR-T cells to be switched “On” and “Off” after infusion. This technology aims to tackle tough-to-treat blood cancers like relapsed / refractory AML. AvenCell’s lead therapies, “AVC-101” and “AVC-201,” target CD123, offering hope for patients with limited options.
Gene Therapy
Astellas Pharma Announces License and Option with AviadoBio | Partnership
AviadoBio Ltd. and Astellas Pharma Inc. announced a licensing agreement for AVB-101, an investigational gene therapy targeting frontotemporal dementia with progranulin mutations (FTD-GRN), currently in Phase 1/2 trials. Under the deal, Astellas gains an option to acquire global rights for AVB-101, making a $20 million equity investment and up to $30 million upfront, with potential milestones and royalties up to $2.18 billion. Other groups with AAV therapies in development for FTD include Eli Lilly (legacy Prevail) and Passage Bio, who recently provided a program update at ESGCT.
Roche Expands Partnership with Dyno Therapeutics | Partnership
Dyno Therapeutics has expanded its partnership with Roche to develop advanced AAV vectors for neurological gene therapies, leveraging Dyno’s AI-based technology to improve vector targeting, immune evasion, and manufacturability. Under the agreement, Dyno will create optimized capsids while Roche will handle further development, validation, and commercialization, with Dyno receiving $50 million upfront and potential future payments exceeding $1 billion. This collaboration builds on their previous 2020 partnership aimed at advancing gene therapy delivery solutions.
Ocuphire Pharma to Acquire Opus Genetics | M&A
Ocuphire Pharma has announced its acquisition of Opus Genetics, creating a combined company focused on advancing gene therapies for inherited retinal diseases (IRDs). The combined company will continue under the name Opus Genetics and trade on Nasdaq as “IRD” beginning October 24, 2024. The merger brings together Ocuphire’s experience in late-stage ophthalmic drug development with Opus’s gene therapy expertise, bolstering a pipeline that includes treatments for presbyopia, diabetic retinopathy, and IRDs such as LCA5.
Neurogene to Present Updated Data on Rett Syndrome Candidate | Clinical Trial
Neurogene will present interim efficacy data for its NGN-401 gene therapy for pediatric Rett syndrome during a webcast on November 11, 2024. The webcast will include safety data from both low- and high-dose cohorts of the ongoing Phase 1/2 trial. Additionally, these findings will also be presented at the Child Neurology Society Annual Meeting. Neurogene’s NGN-401 utilizes the company’s proprietary EXACT transgene regulation platform and leverages an AAV9 serotype.
REGENXBIO Provides Updated Data on Wet AMD Candidate | Clinical Trial
REGENXBIO has released positive data from a Phase II sub-study examining the gene therapy ABBV-RGX-314 for wet age-related macular degeneration (wet AMD) in patients’ second eye. The nine-month data, presented at the American Academy of Ophthalmology meeting, indicated a 97% reduction in the need for anti-VEGF injections, with 78% of patients remaining injection-free after treatment. ABBV-RGX-314, developed in partnership with AbbVie, demonstrated sustained visual acuity and retinal thickness improvements with no serious drug-related adverse events. This follows releases from 4DMT on their program for the same indication.
GenSight Faces Near-Term Cash Crunch | Financing
GenSight Biologics faces financial challenges, with cash reserves expected to run out by mid-November 2024. The Paris-based gene therapy company has previously relied on revenues from a compassionate use program for its eye disease treatment, Lumevoq, but resuming these sales has been delayed until late 2024. To bridge the funding gap, GenSight is seeking additional financing, with hopes of securing funds to continue operations into early 2026 and support a planned Phase 3 trial for Lumevoq. The asset did not meet original endpoints in clinical testing due to unilaterally treated subjects experiencing a contralateral effect in their placebo-treated eye, diminishing the difference between eyes. The company worked extensively to understand and highlight this surprising effect.
Oligo
Judo Bio Debuts with $100M in Series A and Presents Receptor Mediated Oligonucleotide Delivery to Knockdown Genes in the Kidney | Company Creation
Judo Bio, upon launching October 7th with $100M in Series A funding to develop RNA drugs, presented preclinical data demonstrating that its megalin-STRIKER (Selectively Targeting RNA into Kidney) platform enables receptor-mediated uptake of ligand-siRNA in kidney cells. The megalin receptor is targeted by a ligand-bound siRNA in proximal tubule cells to uptake the siRNA, inducing silencing of mRNA expression. The approach focuses on inhibiting solute carrier (SLC) proteins, potentially reducing harmful metabolite levels associated with various diseases. The findings support Judo Bio’s strategy of using RNA therapeutics for precise kidney-targeted gene modulation, presenting a new method for treating systemic diseases through gene silencing.
Lilly and insitro Partner to Develop AI-Powered siRNA Therapies for Metabolic Diseases | Partnership
Insitro and Lilly have formed three strategic agreements to advance therapies for metabolic diseases, including metabolic dysfunction-associated steatotic liver disease (MASLD). As per the first two agreements, Lilly’s GalNAc delivery technology, already used in FDA-approved medicines, will be used to enhance targeted siRNA delivery. Insitro plans to use its machine-learning platform to identify and develop siRNA molecules targeting the liver, and to develop a novel antibody targeting metabolic disease targets (as per the third agreement). Insitro retains rights to the projects, while Lilly may receive milestone payments and royalties.
Alnylam has submitted a regulatory application to the European Medicines Agency for Vutrisiran, an RNAi therapeutic targeting transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The application is based on Phase 3 HELIOS-B trial results, which showed significant reductions in mortality and cardiovascular events compared to placebo. The study also demonstrated positive effects on functional capacity, quality of life, and heart failure symptoms. Vutrisiran's safety profile was consistent with prior studies, showing comparable adverse event rates to placebo. Additional regulatory submissions are planned globally.
City Therapeutics Launches with $135M in Series A Funding to Develop RNAi-Based Therapies | Company Creation
City Therapeutics has secured $135 million in Series A funding, primarily led by ARCH Venture Partners, with participation from Fidelity Management & Research Company, Invus, Slate Path Capital. City Therapeutics was lauched by John Maraganore, former CEO of Alnylam Therapeutics and current venture partner at ARCH. The funding will enable development of RNAi therapies targeting metabolic diseases, specifically using cleavage-inducing tiny RNAs (cityRNAs).
ADCs
Ono Enters into License Agreement for LCB97, and to Generate Novel ADC Candidates with LigaChem Biosciences | Partnership
Ono Pharmaceutical has signed a license agreement with LigaChem Biosciences for LCB97, a pre-clinical antibody-drug conjugate (ADC) aimed at treating solid tumors by targeting the L1 cell adhesion molecule (L1CAM). The agreement grants Ono exclusive rights to develop, manufacture, and commercialize LCB97, with potential payments totaling up to $700 million, including upfront fees and royalties based on sales. Additionally, Ono and LigaChem will collaborate to create new ADC candidates using LigaChem’s proprietary ConjuAll™ ADC platform, which enhances the effectiveness and safety of ADC therapies.
Adcentrx Announces FDA Clearence of its IND Application for a Potential First-in-Class ADC Targeting STEAP1 | Regulatory
Adcentrx Therapeutics has received FDA clearance for its IND application for ADRX-0405, a next-generation antibody-drug conjugate targeting STEAP1, which is upregulated in prostate cancer, yet has limited expression in healthy tissues. Utilizing proprietary i-Conjugation® technology, ADRX-0405 has shown promising preclinical safety and efficacy. The first patient is expected to be dosed in a Phase 1a/b trial by Q4 2024, with initial data anticipated in Q4 2025.
Enhertu Granted Priority Review in the U.S. for Patients with HER-low or HER2-ultralow Metastatic Breast Cancer | Regulatory
AstraZeneca and Daiichi Sankyo's supplemental Biologics License Application (sBLA) for Enhertu has been accepted and granted priority review in the U.S. for patients with HER2-low or HER2-ultralow metastatic breast cancer who received at least one endocrine therapy. This priority review underscores the potential for Enhertu to move into the earlier disease setting and to treat a broader patient population that includes HER2-ultralow.
MilliporeSigma Invests $76M to Expand ADC Manufacturing for Novel Cancer Therapies | Manufacturing
MilliporeSigma has announced a $76 million investment to triple its manufacturing capacity at its St. Louis, Missouri site to meet increased global demand for antibody-drug conjugates (ADCs) and enhance the company's contract development and manufacturing (CDMO) offering. The investment will support the development of novel cancer therapies, enabling faster delivery to market. MilliporeSigma's commitment underscores its goal to provide industry-leading support to early-stage and commercial bioconjugates.
Kivu Bioscience has successfully raised $92 million in a Series A funding round led by Novo Holdings, aimed at advancing multiple oncology next-generation antibody-drug conjugate (ADC) programs into the clinic. The investment will support the development of Kivu's innovative therapeutic platform designed to improve the precision and effectiveness of cancer treatments. With this funding, Kivu plans to accelerate its research and development efforts, moving closer to clinical trials. The funding round reflects strong investor confidence in Kivu's potential to revolutionize cancer therapies through enhanced ADC technology.
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