CLINICAL TRIALS ARTICLES
-
![World health organization-GettyImages-1209486927]( "AI And Pharmaceutical Development: WHO Calls For Ethical Framework, Good Governance")
AI And Pharmaceutical Development: WHO Calls For Ethical Framework, Good GovernanceThe World Health Organization (WHO) recently released a document titled Benefits and risks of using artificial intelligence for pharmaceutical development and delivery describing the ethical issues and governance considerations for using AI in drug discovery, preclinical development, and clinical trials.
-
![DNA-molecule-GettyImages-1316503044]( "FDA’s Updated Guidance On Human Genome Editing: New Implications & Remaining Questions")
FDA’s Updated Guidance On Human Genome Editing: New Implications & Remaining QuestionsOn Jan. 29, 2024, the FDA released the final version of the guidance document on human gene therapy products incorporating genome editing. This article shares what changed between the draft and now final version of the guidance, and what open questions remain for the industry and scientific community to help resolve.
-
![HIV possitive blood sample-GettyImages-903375720]( "1 In 300 People In The U.S. Is Living With HIV. That’s Not Okay With Jeff Galvin.")
1 In 300 People In The U.S. Is Living With HIV. That’s Not Okay With Jeff Galvin.By modifying CD4 T cells using miRNA, Jeff Galvin’s company thinks it can deliver a functional cure for HIV. Here’s the ongoing story of Addimmune and its unique clinical strategy.
-
![2024 growth forcasting-GettyImages-1768245455]( "2024’s Market Outlook For Cell & Gene Therapies")
2024’s Market Outlook For Cell & Gene TherapiesRSM is back for the third consecutive year to provide an outlook analysis of the cell and gene therapy market. This article discusses the 2024 state of the macroeconomy, its effects on cell and gene therapy companies, growing approval pipelines and approvals, funding factors, and the unique prospects for the market.
-
![FDA-GettyImages-1139719728]( "FDA Draft Guidance On “Rare Diseases: Considerations For The Development Of Drugs And Biological Products”")
FDA Draft Guidance On “Rare Diseases: Considerations For The Development Of Drugs And Biological Products”Accenture Applied Life Science Solutions' Raj Bandaru, Ph.D. breaks down, summarizes, and shares detailed insight on the recent FDA Draft Guidance that addresses “Rare Diseases: Considerations for the Development of Drugs and Biological Products.”
-
![Developing mRNA Vaccines GettyImages-1307593959]( "RNA Vaccines: Just Scratching The Surface")
RNA Vaccines: Just Scratching The SurfaceRNA vaccines have shown promise in preventing infectious diseases and treating cancer, but clinical development varies markedly between the two. HDT Bio’s Dr. Berglund discusses this and how they can be improved.
CLINICAL TRIALS VIDEOS
Among the more prevalent genetic conditions, Duchenne muscular dystrophy affects an estimated one in 3,500 male births worldwide. It's caused by mutations of the DMD gene, which regulates the production of a protein called dystrophin. Approved DMD treatments haven't demonstrated strong clinical outcomes, but James McArthur, Ph.D. and his team at PepGen are seeking to change that with a pipeline of disease-modifying peptide-conjugated oligonucleotide candidates derived from the company's Enhanced Delivery Oligonucleotide platform. The Business of Biotech caught up with Dr. McArthur at PepGen's Cambridge headquarters to learn more.
PepGen, a Cambridge, MA-based biotech, is advancing oligonucleotide therapeutics with the goal of transforming the treatment of severe neuromuscular and neurologic diseases. SVP Head of Clinical Development Dr. Michelle Mellion explains PepGen's trial of PGN-EDO51, the company’s lead product candidate for the treatment of DMD patients whose mutations are amenable to an exon 51 skipping approach. She also explains how the delivery of oligonucleotide therapies differ from other techniques in genetic medicine, and much more.
In this video, Russell Johnson, VP Formulation Research for RVAC Medicines, and Aalok Shah, Director of Formulation & Drug Delivery, Strand Therapeutics, discuss what industry challenges should be top of mind for experts as it relates to the regulatory paradigm, toxicity/safety, and in vivo modeling.
Need help unravelling the complexity of CGTx development? Four experts explain Precision ADVANCE’s unique approach. Spend 50 seconds with Anshul Mangal, Deb Phippard, Megan Liles, and Philip Cyr.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
-
![Documentation Traps]( "Shifting The Approach: 4 Strategies For ICH E6(R3) Implementation")
In addition to codifying some best practices, R3 suggests sponsors take a proportionate approach to identifying and managing areas of risk.
-
![CRISPR Gene Editing]( "Advantages Of Cas9 mRNA For Gene Editing")
Discover why Cas9 mRNA is the preferred choice for precise gene editing, offering efficient delivery, transient expression, and flexible targeting.
-
![Patient Interview]( "A New Era For Patient Recruitment And Retention In Rare Disease")
Pharma and biotech companies prioritize innovation in areas of high and rare unmet need. Though the R&D landscape for these rare diseases can be difficult to navigate, changes are on the horizon.
-
![GettyImages-1366661187 cell tech]( "CRISPR Clinical Trials To Follow In 2024 And Beyond")
CRISPR therapies are being developed for genetic disorders, cancer, and infectious diseases. Explore which CRISPR clinical trials are worth keeping an eye on this year.
-
![GettyImages-667825028 cancer]( "Precision Oncology Clinical Trials And Statistics 2024")
Delve into the latest advancements in clinical trials, targeted therapies, and the promising horizon of cancer care.
-
![Duchenne muscular dystrophy genetic test GettyImages-1042202100]( "Global Prevalence And Clinical Trials Landscape Of DMD")
Stay informed with the latest advancements in the fight against Duchenne Muscular Dystrophy, a disease that impacts thousands of lives worldwide.
-
![Lipid nanoparticle mRNA vaccine-GettyImages-1418152448]( "Discussing mRNA: Challenges And Opportunities For Scale-Out And Optimization")
In a recent Cell & Gene Live event, experts explored challenges surrounding raw materials – both quality and supply – as well as the importance of achieving more efficient scale-out and turnaround times for mRNA production.
