RNA therapeutics: targeting previously 'undruggable' diseases
Unleashing the potential of RNA molecules (900 words, 4.5 minutes)
RNA therapeutics are becoming a viable method for addressing diseases previously considered untreatable. Such diseases, marked by elusive targets or intricate biological processes, have historically defied conventional drug development methods. Yet, RNA-based treatments are transforming the landscape. Studies published in the EMBO Journal suggest that these therapies could transform the treatment and prevention of human diseases entirely.
One of the most promising applications of RNA therapeutics is in the field of rare genetic metabolic diseases. The journal Gut notes that messenger RNA-based therapy holds promise as an alternative strategy to protein replacement therapy and gene therapy for treating rare diseases. RNA therapeutics are showing promising results in studies involving conditions traditionally considered challenging to treat due to the complexity of their underlying genetic mechanisms, such as acute intermittent porphyria or methylmalonic acidaemia.
RNA-based therapy may also be a promising and innovative strategy for cancer treatment. Pharmaceutics notes that mRNA-based cancer therapeutics include mRNA cancer vaccines, mRNA-encoding cytokines, chimeric antigen receptors, tumour suppressors, and other combination therapies. The National Cancer Institute emphasizes that mRNA-based cancer treatment vaccines have promising results. They note that several clinical trials have tested and are currently testing mRNA treatment vaccines in people with multiple types of cancer, some even in combination with drugs that enhance the body’s immune response to tumours. However, mRNA cancer vaccines have not been approved by the U.S. FDA.
RNA therapeutics offer a versatile platform for addressing diseases beyond rare genetic disorders and cancer. The Journal of the Missouri State Medical Association notes that other conditions, such as neurodegenerative diseases, are also being explored as potential targets for RNA-based interventions. Conditions such as Alzheimer’s and Parkinson’s disease, some forms of Huntington’s disease, frontotemporal dementia, or amyotrophic lateral sclerosis have long posed challenges due to their genetic complexity. RNA-based treatments identify the genetic bases of diseases and the specific molecular targets to treat them.
Despite the potential of RNA therapeutics, several challenges remain. EMBO Molecular Medicine notes that delivery remains a crucial challenge because RNA molecules are susceptible to degradation and cannot penetrate cell membranes.
Additional reading:
https://www.sciencedirect.com/science/article/abs/pii/S1877117323001965
https://molecular-cancer.biomedcentral.com/articles/10.1186/s12943-023-01807-w
THIS WEEK 04/16/24
A U.S. appeals court affirmed a decision for Bausch Health that barred rival drugmaker Alvogen from marketing a proposed generic version of Bausch’s diarrhea treatment Xifaxan until 2029.
Viatris Inc. launched PrGlatiramer Acetate Injection 20 mg/mL for injection, a generic bioequivalent version of Teva’s Copaxone 20 mg/mL, in Canada. This medication is indicated for Tx of patients with Relapsing-Remitting Multiple Sclerosis.
Fresenius announced the immediate U.S. availability of tocilizumab-aazg (Tyenne), a biosimilar of Actemra, for Txof several autoimmune diseases, including rheumatoid arthritis, giant cell arteritis, polyarticular juvenile idiopathic arthritis, and systemic juvenile idiopathic arthritis.
Health Canada is warning consumers about multiple unauthorized health products seized from Vanette Keast Health Consulting in Red Deer, Alta. The seized products are labelled to contain prescription, controlled, or other drug ingredients and may pose serious health risks. They are promoted for various uses, including as Txs for diabetes, parasites, or hypothyroidism.
LISTEN UP
In season 12 of the NPC Podcast, Aldona Armstrong, Commercial Solutions Lead, Immunology at UCB, gives insights into patient advocacy initiatives, working with key opinion leaders, and ways to implement specialty marketing approaches. Hear her in conversation with podcast hosts Mitch Shannon, Jim Shea and Mark McElwain.
HEALTHBIZ REWIND
This feature of NPC Healthbiz Weekly looks back at some of the most insightful moments from previous NPC Podcast episodes. In Healthbiz Rewind, you’ll read bold life sci predictions made during the renowned “Prognostication Korner” segment of the NPC Podcast.
Dave Cann
Senior Director of Business Development
Inizio Engage
Toronto
Season 11, episode 05
Listen to this episode here
What bold predictions will you make about the life sciences industry during the coming 12 to 24 months?
I don’t know if I’d go on a limb and say it’s bold. I think it’s more just looking at what we’ve seen play out in the last five or 10 years, and I see more of the same. I think there will be more organizations streamlining their businesses, divestitures, and M&A activity. I think it’s all to appease shareholders and align with laser-focused businesses with very niche offerings. Therefore, it will strain strategic agility and create a need for stronger strategic partnerships, and hopefully, outsourced providers will be part of that picture. That’s my bold prediction: more M&A, streamlining, lean, and mean organizations.
Life Sciences Ontario: 2024 Annual Awards
Join LSO for a night of celebration of the #lifesciences sector and individuals and companies that have made outstanding contributions — Bill Mantel of Centre for the Commercialization of Regenerative Medicine (CCRM); Dr. Benjamin Rovinski of Lumira Ventures; Dr. Christine Allen of Leslie Dan Faculty of Pharmacy, University of Toronto; Dr. Durhane Wong-Rieger of Canadian Organization for Rare Disorders; and Satellos Biosciences Inc.
Date: Wednesday, May 15th, 2024
Venue: Liberty Grand, Exhibition Place - 25 British Columbia RoadMay 15o
May 15ception - 5:30 p.m. / Dinner: 6:30 p.m.
Grab your tickets now here.
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