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
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Accelerating Approval Pathway For Phase III Ovarian Cancer Trial
A late-stage biotech developing a first-in-class therapy for a rare, chemo-resistant form of ovarian cancer sought help for accelerated FDA approval.
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mRNA Therapeutic Development Through Enhanced IVT Capping Efficiency
See how Codex HiCap RNA Polymerase supports the production of high-performance synthetic mRNA, paving the way for next-generation mRNA therapies.
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Phase 1 Clinical Trial Designs And Strategies
With tailored guidance, sponsors can align their modern model-based and model-assisted design choices to program goals, accelerating progress while safeguarding patients.
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IVT mRNA Encapsulation Efficiency Assessment
In vitro transcribed mRNA revolutionizes biotherapeutics, which necessitates thorough quality assessments. See how an analyzer system enhances encapsulation efficiency and CQAs in IVT mRNA workflows.
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Filling Syringes With Spray-Dried Powders - The How, The Why And The Benefits
Discover how filling spray-dried powders directly into syringes improves stability, minimizes cold chain reliance, and accelerates administration speeds for critical therapies.
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Preparing For Disease X: Effective Vaccine Development In The Face Of A Future Pandemic
CDMOs are playing a critical role in the global effort to prepare for future pandemics. Learn how innovative strategies and flexible platforms are helping to ensure a swift and effective response plan.
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Building A Scalable Digital Foundation To Support CGT Manufacturing
Explore how digital systems strengthen CGT manufacturing by improving traceability and managing batch‑of‑one, as well as essential steps toward scalable, integrated operations.
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High-Performance Extraction And Quantitation Of Host-Cell Residual DNA
This study presents a high-efficiency method for extracting and quantifying residual DNA from host cells using a semi-automated, high-throughput workflow.
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Comparing Sterility Testing Techniques
Is your sterility testing process hindering your product release? Discover how the right approach can streamline your process, reduce false positives, and ultimately save you time and money.
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Expansion Of Cord Blood-Derived CD34+ Cells In A Hollow-Fiber Bioreactor With 10% Standard Cytokine Concentration
CD34+ cells isolated from cord blood were expanded in a hollow-fiber bioreactor using a novel cytokine cocktail at one-tenth the standard concentration using a MSC-free coating strategy.
NEWSLETTER ARCHIVE
- 03.10.26 -- The Real Reason We Haven't Fully Automated Cell Therapy Yet
- 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
- 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.
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