Gene and Cell Therapy
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JCR Pharmaceuticals to Present at the European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Congress
Businesswire· 2025-09-30 17:11
Core Insights - JCR will present non-clinical data from its novel JUST-AAV gene therapy platform technology at the ESGCT 32nd Annual Congress [1] Company Overview - The presentation will focus on the advancements and findings related to the JUST-AAV gene therapy platform [1]
CAMP4 Reports Second Quarter 2025 Financial Results and Corporate Highlights
Globenewswire· 2025-08-14 20:05
Core Insights - CAMP4 Therapeutics presented positive translational data from its SYNGAP1-related disorders program, demonstrating efficacy in a humanized mouse model and increased protein levels in non-human primates at the ASGCT Annual Meeting [1][7] - The company reported a net loss of $12.6 million for Q2 2025, consistent with the loss from the same quarter in 2024 [8][13] - CAMP4 is advancing its CMP-SYNGAP-01 program with plans to initiate GLP toxicology studies in Q3 2025 and a potential global Phase 1/2 clinical trial in SYNGAP patients in H2 2026 [2][6] Corporate Highlights - The company has a strong pipeline focused on regulatory RNA-targeting therapeutics aimed at treating genetic diseases [1][9] - CMP-CPS-001 is being developed as a first-in-class, disease-modifying therapy for common urea cycle disorders (UCDs), with safety and biomarker data expected in Q4 2025 [2][6] Financial Results - As of June 30, 2025, CAMP4 had cash, cash equivalents, and marketable securities totaling $39.1 million, down from $49.3 million as of March 31, 2025 [4][13] - Research and development expenses for Q2 2025 were $10.3 million, an increase from $9.4 million in Q2 2024, primarily due to higher clinical and preclinical study costs [4][13] - General and administrative expenses rose to $4.2 million in Q2 2025 from $3.3 million in Q2 2024, driven by increased personnel-related and overhead costs [5][13]
Bio-Techne to Showcase Next-Generation Solutions for Gene and Cell Therapy at American Society of Gene & Cell Therapy 2025
Prnewswire· 2025-05-12 11:00
Core Insights - Bio-Techne Corporation will showcase its latest spatial biology and cell and gene therapy workflow solutions at the ASGCT 2025 meeting in New Orleans from May 13th to 17th [1] - The company will highlight advancements in cell and gene therapy development, including the RNAscope™ protease-free workflow and ProPak™ GMP cytokines [2][4] - Bio-Techne generated approximately $1.2 billion in net sales in fiscal 2024 and employs around 3,100 people globally [5] Product Highlights - The RNAscope™ protease-free workflow allows visualization of biodistribution of AAV vectors, transgene mRNA, and small RNAs [2] - ProPak™ GMP cytokines are designed for closed-system cell therapy manufacturing, featuring dose-optimized, single-use bags [2] - The Simple Western Leo™ System enables simultaneous processing of up to 100 samples in a single 3-hour run [2] Symposium and Presentations - ACD's symposium will feature innovations with RNAscope technology, including NextGen RNAscope™ Multiomics Solutions [3] - A talk on protein quantitation applications to advance gene therapy development will be presented by guest speakers from Sangamo Therapeutics and Nervosave Therapeutics [3][6] - Additional presentations will cover AI-designed AAV capsids and RNA gene writer activity in therapeutic contexts [6]