Gene Therapies for Rare Disorders Recap
Lessons learned at the Gene Therapy for Rare Disorders Summit, especially insights for Natural History Studies.
3200 Rare Diseases: Now Who Is Going to Cure Them?
To the patients who benefit from treatments that may have otherwise never been developed, your contribution would mean everything.
At-Cost AAV: From Production Cost to Patient Dose
Estimating the cost of manufacturing an AAV Gene Therapy by comparing industry benchmarks and published doses used in clinical trials.
Gene Therapies: Justifiably Expensive or a Mirage of Cost Effectiveness
An exploration of value based pricing, how willingness-to-pay and ´novel concepts are inflating the cost effectiveness of gene therapies.
Crowdfunding for a Cure
Crowdfunding is a growing democratic alternative to venture capital. Can it also provide the fuel to accelerate novel therapies for those left behind by for profit healthcare…
Defying Eroom’s Law
Economic challenges in rare disease gene therapy, intensified by Eroom’s law, demand innovative solutions. Amid accusations of pharmaceutical oligopoly and economic inequality, potential remedies lie in collaborative, decentralized business models.