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Scrip’s Rough Guide On The State Of RNAi

How Will RNA Modalities Stack Up Against Gene Therapy?

 
• By Daniel Chancellor

The introduction of gene therapies has been the highest-profile new modality to reach the market, but new RNA-based therapeutics hold significant potential and could be more accessible for patients. Scrip reviews the leading drug developers in this space.

RNA_Research

Much of industry's current attention is focused on gene therapies – and rightfully so, considering the therapeutic promise of such one-time cures, and the spate of M&A activity for gene therapy biotechs and manufacturers, despite justifiable concerns over its scalability and affordability. But some of those challenges serve to highlight the opportunities possible with a different breakthrough field – drugs that target RNA, either through double-stranded RNA-mediated interference (RNAi) or antisense oligonucleotides (ASOs).

Arguably overshadowed by gene therapies and their curative hype, RNA therapeutics are gaining prominence, with a number of approvals in...

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BIO Notebook: Woodcock Calls For Doing The Right Thing, Dealmaking Remains Constrained

 
• By Mandy Jackson, Joseph Haas, and Sarah Karlin-Smith

Highlights from Day Four of the BIO International Convention include Woodcock offering practical advice on rare disease trials, the sorry state of dealmaking mid-year, Novartis discussing its approach to partnering, and Generate looking for funding to move into Phase III.

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• By Mary Jo Laffler

Antibody-drug conjugates are a major part of the oncology pipeline, and data presented at the American Society of Clinical Oncology meeting highlight the emergence of new technologies.

Skyhawk Looks To Soar With Huntington’s Hope

 
• By Kevin Grogan

Oral RNA splicing modulator has gone into a Phase II/III trial

BIO Notebook: MFN Pricing, Next-Gen Obesity R&D, FDA’s Rare Disease Hub & Reaction To Review Program

 
• By Sarah Karlin-Smith, Mandy Jackson, and Alaric DeArment

Highlights from Day 3 of the BIO International Convention include the realities of MFN pricing, AstraZeneca's R&D plans for obesity, the need for resources for FDA's rare disease hub and reactions to the Commissioner's National Priority Review Voucher program.

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Skyhawk Looks To Soar With Huntington’s Hope

 
• By Kevin Grogan

Oral RNA splicing modulator has gone into a Phase II/III trial

BIO Notebook: MFN Pricing, Next-Gen Obesity R&D, FDA’s Rare Disease Hub & Reaction To Review Program

 
• By Sarah Karlin-Smith, Mandy Jackson, and Alaric DeArment

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EY Recommends Focusing On Fundamentals, Waiting For Prime Deal Conditions

 
• By Joseph Haas

Government policy ramifications and tight financial markets mean it’s time to prepare strategically for a better business development environment, EY suggests.