Rare Diseases | Special Topics | Gene Therapies | US/EU5 | 2017

The revolution in genomic sciences has spurred significant drug development in rare diseases, approximately 80% of which have a genetic etiology. The field of gene-based therapies, while still in the early stages, with therapies currently in various phases of preclinical and clinical development, has recently seen its first approvals in Europe. Gene-based therapies have the potential to transform the treatment of rare diseases and even provide a cure for many of them. Coupled with high pricing potential, recent technological advances will likely unlock a tremendous market potential for novel rare disease treatments. Broadly, gene therapies can be classified into three mechanistic categories: viral-based gene delivery systems, oligonucleotide-based therapies, and DNA-editing systems. Each category poses unique therapeutic advantages and development challenges. This special report will provide an analysis of the unique challenges and advantages of these approaches as well as examine the pipeline for gene-based therapies and the regulatory and market access issues that will impact the approval and uptake of these potentially transformational treatments.


What are the unique clinical development challenges for gene therapies? What regulatory pathways exist for gene therapies in the United States and Europe?

How are payers approaching pricing and reimbursement of gene therapies; which may be curative? How will high annualized costs affect reimbursement?

What lessons can be learned from successful and unsuccessful commercial launches in Europe?

 What characteristics make an indication a good opportunity for a gene therapy?  Within each subcategory of gene-based therapies, what are the key technological differentiators and how might they provide a competitive advantage? What are the unique manufacturing challenges that gene therapies present?

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