The burgeoning field of cell and gene therapies collectively referred to as Advanced Therapeutics (ADVTX), is at last beginning to deliver on a decades-old promise to provide potentially curative therapeutic value to patients in all disease areas, especially those with rare, genetically driven pathologies. Several ADVTX platforms have been clinically validated, including FDA approvals, and deal-making in the space has accelerated in response. Many patients, including those with certain lymphomas, neuromuscular disorders and retinal dystrophies, are already benefiting from these novel ADVTX platforms. The standard of care (SoC) in additional disease areas is poised for disruption in the near-term, as well. However, developmental setbacks have occurred, with some platforms yet to demonstrate satisfactory clinical validation. Despite the overall promise of ADVTX, many challenges remain. Patients must decide whether they are comfortable being treated with novel therapeutic modalities, notably in areas with effective disease-modifying SoC, notwithstanding a burdensome chronic treatment regimen. Drug developers with conventional therapy assets in rare disease are now being tasked with understanding what impact ADVTX platforms will have on their franchises and to either develop defensive strategies against the looming disruption or adopt an M&A plan for integration of ADVTX programs into their pipelines.

Stakeholders across the spectrum of rare disease, including patients, caregivers, healthcare providers, drug developers, payers and advocacy groups will find the content in this whitepaper relevant and informative.

This whitepaper makes the case that advanced therapeutics are finally delivering on their promise to provide potentially curative treatments to patients. Companies who develop rare disease medicines must be keenly aware of the business challenges they are about to face. These challenges come in a variety of flavors, and strategies designed to mitigate the risk ADVTX platforms pose to existing rare disease franchises must be implemented.