The Promise Of Cell And Gene Therapies For Rare Diseases

By Brittany Enzmann, Ph.D.

Recognizing the need in 1983, Congress passed the Orphan Drug Act to create incentives for companies to develop drugs that treat rare conditions. Economic assistance is now available for companies to create orphan drugs, helping provide solutions for people who have no other options. There will likely be more solutions for rare conditions on the horizon. With the rapid rise of cell and gene therapies over the past decade, biopharma, biotechs, and nonprofits are taking on diseases that are traditionally hard to treat — including rare ones.

While the potential of cell and gene therapies offers new optimism for patients afflicted with rare diseases, the path forward is a cautionary one. Cell and gene therapies are inherently complex, making it difficult to ensure their quality and consistency.

We explore the challenges of developing cell and gene therapies and the innovations in biotechnology that may help offer more solutions will soon be available to patients who have little else to turn to.