RNA Therapies in Rare Diseases

Rare diseases pose a global burden to healthcare despite making up a very small fraction of the global disease landscape. Most treatment options for rare diseases focus on maintaining the disease symptoms, rather than a disease-modifying approach. To address this unmet need, RNA therapies are emerging as disease-modifying approaches addressing the genetic basis, which is the predominant cause of most rare diseases. Over the past ten years, RNA therapies have progressed from experimental to validated treatments, with several approvals for rare diseases demonstrating their potential. Notable approvals that show the efficacy of ASO and siRNA platforms and increase confidence in RNA drug development include Nusinersen for spinal muscular atrophy, Patisiran for hereditary transthyretin-mediated amyloidosis, Givosiran for acute hepatic porphyria, and Tofersen for SOD1-associated amyotrophic lateral sclerosis. RNA therapies are emerging as a rapidly growing therapeutic modality in the rare disease landscape through better delivery methods, precision targeting technologies, and personalization.

The report discusses types of RNA modalities, including anti-sense oligonucleotides (ASOs), small interference RNA (siRNA), and messenger RNA (mRNA) and their application across various rare diseases.

Some key questions that the report will cover are:
What are the key drivers and restraints in the RNA therapies for rare diseases industry?
How fast is clinical development progressing across different RNA modalities?
Which companies are leading innovations for RNA therapeutics in the rare disease segment?
What does the funding, partnership and acquisitions landscape look like?
Where are the growth opportunities in RNA therapies for rare diseases?