The cost of rare diseases to patients and society at large is 10 times higher than for many of the biggest mass market diseases combined. With 95% of rare diseases lacking a treatment or cure, there is an economic imperative for policymakers to keep encouraging pharmaceutical companies to develop more orphan drug treatments. In Vivo looks at the US and EU regulatory impetus to keep rare diseases open for innovation. 

Rare disease treatments have been increasingly attractive targets for investment, but clinical trials of these agents are laden with unique challenges, not least of which is that there are by definition small and possibly disparately located patient populations. However, by cultivating relationships with patient organizations and carefully designing studies, companies in the rare disease space are proving they can overcome logistical hurdles. 

Syngene’s COO, Mahesh Bhalgat, tells Scrip the firm is better placed than some peers to serve clients for biologics-based advanced therapies and address the cost element for new modalities like mRNA. He also outlines the research, development and manufacturing services company's capabilities in oligonucleotide-based therapeutics, an area that's seeing growing interest.

Rona hopes technology acquired from Sanofi will enhance its RNA capabilities in liver and non-liver indications. NeuroFront gets Greater China and Singapore rights to Novaremed’s neuropathic pain drug.