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Highlights & Summary
Intro
March was an exciting month for the next-generation therapeutics space, full of new partnership announcements and clinical trial updates.
Happy reading!
Cell Therapy
- Kytopen Announces Technology Access Program | Manufacturing
- Novel CAR-T Therapy Shows Dramatic Responses in Glioblastoma | Clinical Trial
- Kyverna Doses Multiple Sclerosis Patients with KYV-101 | Clinical Trial
Antibody-Drug Conjugates
- Tubulis Closes Upsized €128 Series B | Financing
Gene Therapy
- Nanoscope Reports Positive Data for MCO-010 in Vision Loss | Clinical Trial
- Sangamo Reports Enhanced Neurotropic Capsid | Pipeline
- Charles River Announces Development Partnership with Navega | Manufacturing
Oligo Therapy
- Altamira Therapeutics announces collaboration with Univercells Group on Nanoparticle-Delivered mRNA vaccines | Partnership
- Silence Therapeutics Announces Positive Topline 36-Week Data from Ongoing Phase 2 Study of Zerlasiran in Patients with High Lipoprotein(a) | Clinical Trial
- Moderna Achieves Positive Interim Results from Phase 3 Trial of Next-Generation COVID-19 Vaccine | Clinical Trial
- Capstan Achieves Series B Financing | Financing
Cell Therapy
Fiocruz and Caring Cross Collaborate to Manufacture Cheaper CAR-T Therapies | Partnership
The two companies seek to establish local manufacturing capabilities for CAR-T cell therapies, for the treatment of leukemia, lymphoma, and HIV, thereby reducing the treatment cost to around $35,000 per dose, a fraction of the cost in the US and Europe. This partnership not only enables access to life-saving therapies but also positions Fiocruz as a central hub for advanced medicine development and production in the region.
NKGens Presents Additional Positive Trial Data in Alzheimer’s Disease | Clinical Trial
NKGen presented additional Phase 1 clinical trial data on its autologous NK cell therapy, SNK01, showing up to an 80% decrease in cerebrospinal fluid and plasma Tau proteins in Alzheimer’s patients, suggesting potential clinical activity in advanced Alzheimer’s disease. Despite 70% of patients receiving relatively low doses of SNK01, significant improvements were observed. Cognitive function in patients also showed improvement.
Kytopen Announces Technology Access Program | Manufacturing
The company has launched the Flowfect Technology Access Program (TAP), offering early access to partners for their manufacturing system, supported by an expanded applications team. The program provides hands-on support to take partner therapeutics from proof-of-concept testing through process development optimization and into clinical and commercial manufacturing. Kytopen's platform integrates continuous fluid flow with electric fields for gentle delivery of payloads to engineered cells and extracellular vesicles, maximizing cell health, safety, and scalability.
Novel CAR-T Therapy Shows Dramatic Responses in Glioblastoma | Clinical Trial
A groundbreaking CAR-T cell clinical trial led by Marcela Maus’ team at Mass General Cancer Center has reported dramatic tumor reductions in glioblastoma patients treated with CARv3-TEAM-E T cells. Early results showed near-complete tumor regression in one patient just 5 days after treatment, highlighting the potential of this innovative therapy. The technology engineers patients' own T-cells to express two proteins, anti-EGFRviii CAR on the T-cell surface for high concentration at the tumor site, and an anti-wtEGFR T-cell engaging bispecific antibody to recruit surrounding T-cells, promising a brighter future for cell therapy innovation.
Kyverna Doses Multiple Sclerosis Patients with KYV-101 | Clinical Trial
Kyverna Therapeutics' groundbreaking use of KYV-101 in patients with progressive MS, marks a significant advancement in autoimmune disease treatment. Administered to two patients, KYV-101 demonstrated an acceptable safety profile, showing promise for larger clinical studies. The therapy's success could pave the way for broader application in autoimmune diseases, potentially transforming the standard of care.
ADCs
BrickBio Achieves Unprecedented Preclinical Breakthrough | Pipeline
BrickBio has achieved a remarkable fivefold increase in the therapeutic window for Pyrrolobenzodiazepine (PBD), a significant advancement in the ADC space, leading to complete tumor eradication in animal models. Through site-specific conjugation, BrickBio securely integrates the highly efficacious payload into the antibody backbone, preventing toxicity during delivery to cancer cells. The company is planning for advanced studies targeting CD276 (B7-H3) and IND-enabling studies.
Biocytogen Enters Collaboration with ABL Bio to Develop Bispecific ADCs | Partnership
The collaboration focuses on developing new bispecific ADCs. Biocytogen's RenLite® mice platform, known for producing fully human antibodies, will be utilized to develop these novel therapies. This partnership combines Biocytogen's bsADC platform technology with ABL's expertise in cancer immunotherapy and treatments for CNS diseases, aiming to accelerate the development of innovative bsADC therapies.
Biotheus Expanded Partnership with Hansoh Pharma for EGFR/cMET Bispecific ADCs | Partnership
The two companies have expanded their strategic collaboration to develop EGFR/cMET bispecific ADCs. Hansoh Pharma will gain exclusive worldwide rights to use Biotheus' proprietary antibody for ADC product development, with potential upfront and milestone payments totaling up to $800M, along with royalties on global net sales. Adcoris Biopharma, Novatim Immune, AstraZeneca, and Biocytgen are all actively developing EGFR/cMET bispecific ADCs.
Bio-Thera Solutions Receives IND Clearance for Phase II Study for its FRa ADC | Regulatory
BAT8006, has shown promising results in preclinical studies, demonstrating stability, safety, and strong anti-tumor activity. The phase II study aims to evaluate BAT8006's efficacy in treating platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer. According to DeciBio’s TheraTrack, there are currently over 14 anti-FRa ADCs in development.
Tubulis Closes Upsized €128 Series B | Financing
The round was co-led by EQT Life Sciences and Nextech Invest, with participation from new US-based funds, Frazier Life Sciences and Deep Track Capital, as well as all of our existing investors. The company will be revealing preclinical proof-of-concept data at AACR for two lead candidates targeting 5T4 and Napi2b in solid tumors.
Gene Therapy
Nanoscope Reports Positive Data for MCO-010 in Vision Loss | Clinical Trial
Nanoscope Therapeutics is advancing its gene therapy for retinal diseases, aiming for FDA approval later this year. Results from a two-year phase 2b study showed vision improvement among patients with advanced retinitis pigmentosa. The therapy, MCO-010, demonstrated significant improvement in best-corrected visual acuity compared to placebo, with no serious adverse events reported. Nanoscope plans to submit for FDA approval in 2024, targeting the higher dose of MCO-010 for commercial use. The therapy, which has received fast-track and orphan designations, is competing with similar programs from the likes of 4DMT, Gensight, and Janssen, among others.
Sangamo Reports Enhanced Neurotropic Capsid | Pipeline
Sangamo Therapeutics unveiled preclinical data on its proprietary AAV capsid variant, STAC-BBB, showing robust penetration of the blood-brain barrier and strong transgene expression across the central nervous system in non-human primates. The results suggest STAC-BBB's potential in neurological diseases by enabling widespread delivery of therapeutic genes. The company plans to leverage STAC-BBB in its prion disease and tauopathy programs and explore its application in other neurological conditions. Sangamo is leveraging the capsid in both tau and prion disease programs.
Charles River Announces Development Partnership with Navega | Manufacturing
Contract manufacturer Charles River has inked a new gene therapy agreement with Navega Therapeutics, focusing on an AAV9 candidate intended as a non-opioid solution for rare disease patients grappling with chronic pain. Under the deal, Navega gains access to Charles River’s contract development and manufacturing capabilities to produce the AAV gene therapy candidate NT-Z001 for phase 1 clinical trials. This partnership underscores Charles River's commitment to expanding its cell and gene therapy portfolio, which includes recent facility expansions and acquisitions, which could see share gain given the geopolitical trouble rival WuXi is facing in the U.S.
Tenaya Reveals Positive Results for TN-401 in Rare Heart Condition | Pipeline
Tenaya Therapeutics unveiled promising results for its gene therapy targeting arrhythmogenic right ventricular cardiomyopathy (ARVC) in mice. The study in Nature Communications Medicine showed a single dose of TN-401 prevented the onset or slowed the progression of ARVC in mice, significantly extending their lifespan compared to untreated counterparts. ARVC, caused by a mutation in the PKP2 gene, leads to abnormal heart rhythms and reduced heart function, often resulting in sudden death. TN-401 works by restoring production of the PKP2 protein. In preclinical trials, TN-401 not only prevented disease development in mice but also reversed cardiac remodeling in those already affected. The company is poised to begin human testing in the second half of 2024. The asset is competing with Lexeo, which also has a development candidate aimed at delivering the PKP2 gene.
Research Study Suggests Species-Specific Transcriptional Activation for AAV | Research
Researchers have found that avian AAV behaves differently from primate AAV when introduced into human cells, as it is transcriptionally silenced. The study, which was published in Cell Reports, demonstrated that altering a specific component of the AAV capsid lead to enhanced viral transcription and enabled robust transduction in both human cells and mouse tissue. This modification also led to changes in genome accessibility and histone methylation. The study suggests that the AAV capsid may have evolved mechanisms to recruit host factors, facilitating species-specific transcriptional activation.
Oligo
Altamira Therapeutics announces collaboration with Univercells Group on Nanoparticle-Delivered mRNA vaccines | Partnership
Altamira Therapeutics has unveiled a collaboration with Univercells Group to develop nanoparticle-delivered mRNA vaccines. The partnership aims to combine Altamira's SemaPhore platform with Univercells Group’s extrahepatic targeting mRNA platform. The collaboration aims to reduce mRNA loss during cell entrance, allowing for lower dosing and favorable tolerability.
Silence Therapeutics Announces Positive Topline 36-Week Data from Ongoing Phase 2 Study of Zerlasiran in Patients with High Lipoprotein(a) | Clinical Trial
Silence Therapeutics has disclosed positive topline data from the ongoing Phase 2 study of Zerlasiran, an RNAi-based therapeutic aiding patients with high lipoprotein(a) levels. The 36-week data demonstrates a statistically significant reduction in lipoprotein(a) levels, meeting the primary endpoint of the trial. Zerlasiran exhibited a favorable safety profile with no significant adverse events reported during the study period.
Moderna Achieves Positive Interim Results from Phase 3 Trial of Next-Generation COVID-19 Vaccine | Clinical Trial
Moderna has announced interim results from its Phase 3 trial of a next-generation COVID-19 vaccine. mRNA-1283 showed a higher immune response against both the Omicron BA.4/BA.5 and original virus strains of SARS-CoV-2, compared to mRNA-1273.222. Additionally, this response was most acutely seen in participants over the age of 65 years, a population that faces high risk for severe outcomes from COVID-19. Furthermore, the shelf life, storage and pre-filled syringe configuration of mRNA-1283 could alleviate healthcare provider burden and increase access of the vaccine into new settings.
Capstan Achieves Series B Financing | Financing
Capstan Therapeutics has secured $175 million in an oversubscribed Series B financing round. Funds raised from the Series B financing will drive the progression of CPTX2309, Capstan’s primary in vivo chimeric antigen receptor T cell (CAR-T) prospect, towards early clinical proof-of-concept in autoimmune disorders. Additionally, the financing will support the ongoing development of Capstan’s tLNP pipeline. CPTX2309, derived from Capstan’s tLNP platform, administers an mRNA payload encoding an anti-CD19 CAR to CD8-expressing T cells.Capstan Therapeutics aims to accelerate the advancement of its pipeline, focusing on therapies for autoimmune and inflammatory diseases.
Blackstone Collaborates with Moderna on Influenza Vaccine | Financing
Blackstone Life Sciences has announced a strategic collaboration to support Moderna's influenza program. Through this collaboration, Blackstone aims to provide up to $750M to accelerate the development of mRNA-based vaccines for influenza and will be eligible to receive milestones and royalties on relevant flu products.
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