SynaptixBio's Dr. Dan Williams discusses the company’s effort to develop an antisense oligonucleotide therapy that silences the mutant TUBB4A gene in the ultra-rare leukodystrophy H-ABC, highlighting the scientific, regulatory, and funding challenges of advancing treatments for extremely small patient populations.
- Injecting Cancer Killers: Intratumoral Therapy For Solid Tumors
- Proteomics Tools: A Resurrection
- Powering AI And Synthetic Biology In Therapy Design
- Six Specialized Modalities Testing CDMO Readiness In 2026
- Green Supply Chains Rarely Start With A Pledge; Target Waste Instead
- The Real Reason We Haven't Fully Automated Cell Therapy Yet
- Process Engineering's Key Role In Sterile Injectable Facility Design
- What To Know About — And How To Apply For — FDA's PreCheck Pilot
CELL & GENE ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
-
![GettyImages-1634050535 data, logistics]( "Expert Insights In Navigating CGT's Complex Supply Chain")
Expert Insights In Navigating CGT's Complex Supply ChainExamine insights shared during Cencora's ThinkLive Cell and Gene Therapy Summit 2024 that shed light on the challenges and opportunities in navigating the complexities of the cell and gene supply chain.
-
![GettyImages-2036497686-lab-team-collaboration-laboratory-tablet-computer]( "Standardize Flow Cytometry Data With Easy Assay Transfer Across Sites")
Standardize Flow Cytometry Data With Easy Assay Transfer Across SitesGain insight into how a global pharmaceutical company successfully transferred their cell therapy assays across international locations.
-
![Thermo Fisher - Peptones]( "The Value Of Peptones For Enhancing Biopharmaceutical Productivity")
The Value Of Peptones For Enhancing Biopharmaceutical ProductivityPeptones, versatile media additives derived from various sources, offer numerous benefits for biopharmaceutical production.
-
![Cell & Gene Therapy Scientists GettyImages-1209892070]( "Optimizing Performance For De-Risked Lentiviral Vector Production")
Optimizing Performance For De-Risked Lentiviral Vector ProductionDiscover a de-risked, reproducible, and scalable platform for efficient GMP manufacturing of lentiviral vectors that maximizes titers and enriches recovery through upstream and downstream optimization.
-
![Molecule mRNA-GettyImages-1428646288]( "mRNA Therapeutic Development Through Enhanced IVT Capping Efficiency")
mRNA Therapeutic Development Through Enhanced IVT Capping EfficiencySee how Codex HiCap RNA Polymerase supports the production of high-performance synthetic mRNA, paving the way for next-generation mRNA therapies.
-
![GettyImages-2190247072-laboratory-vials-analysis-scientist]( "Optimized Urine Liquid Biopsy Workflow")
Optimized Urine Liquid Biopsy WorkflowUrine-derived cell-free DNA is revolutionizing liquid biopsy research to offer a non-invasive, accessible tool for early disease detection, monitoring, and comprehensive diagnostics across oncology.
-
![GettyImages-1306663245-intestinal-organoid-scientist]( "Evaluating Compound Toxicity Effects On Healthy Intestinal Organoids")
Evaluating Compound Toxicity Effects On Healthy Intestinal Organoids3D organoids enhance drug toxicity prediction and are crucial for anti-cancer drug development. Examine a method using 3D mouse intestinal organoids that aims to provide vital data on intestinal toxicity.
-
![GettyImages-2163836351_DNA_Cell Gene]( "AAV-Based Gene Editing For Huntington's Disease")
AAV-Based Gene Editing For Huntington's DiseaseLETI-101 uses AAV5 to deliver a compact CRISPR system for allele-selective editing of mutant HTT, showing promise as a one-time treatment for Huntington’s disease with strong preclinical safety and efficacy.
-
![GettyImages-2158862132-vials-robotic-arm-filling-liquid-handling-lab]( "Aseptic Process Design And Simulation Under Annex 1 Guidelines")
Aseptic Process Design And Simulation Under Annex 1 GuidelinesGain insight into how Annex 1 reshapes aseptic process validation, as well as into risk reduction by design and why isolators are redefining modern sterile manufacturing standards.
-
![GettyImages-1327460931 biomanufacturing]( "Single-Use Technology And Environmental Responsibility In Biopharmaceuticals")
Single-Use Technology And Environmental Responsibility In BiopharmaceuticalsLearn about strategies such as regional manufacturing and advanced single-use systems and their potential to minimize carbon footprint and enhance sustainable biomanufacturing.
NEWSLETTER ARCHIVE
- 03.09.26 -- Putting Pressure On CDMOs With Herman Bozenhardt
- 03.06.26 -- New Podcast Episodes: Therapeutic Power Of Fibroblasts, Curative Cell Therapies, Breakthrough HER2 Immunotherapy
- 03.06.26 -- Simplifying Friction In Cell Therapy Clinical Trials With Dr. Panteli Theocharous
- 03.05.26 -- Regulatory, Payer & Distribution Insights For CGT Success
- 03.05.26 -- Process Engineering's Key Role In Sterile Injectable Facility Design
- Inside SynaptixBio's Mission To Develop A Therapy For H-ABC
- Powering AI And Synthetic Biology In Therapy Design
- Inside Mesoblast's Playbook For Making Allogeneic Cell Therapy Real
- Disciplined CGT Will Win On 2026 Regulatory Flexibility
- ARM's Tim Hunt On Expanding Patient Access, Smarter Regulation, And A More Sustainable Market In 2026
CELL AND GENE CONTENT COLLECTIONS
While there are opportunities to scale, there are also manufacturing gaps, capacity issues, and production timelines that require improvement to do so. And then, of course, there’s cost. Because scale up and scale out are significant to everyone in the sector, we’ve curated insightful editorial that addresses the most important aspects of scalability.
More Content Collections
