StemCell Therapy

A new report has predicted that gene therapy development will pick up pace but a high price point continues to pose a challenge.

According to a new report, ongoing collaborations between different industry players and a buildup of real-world evidence establishing safety and efficacy are expected to drive the growth of gene therapies for neurology indications.

The report from GlobalData, continues that, of the 38 pipelines products that are currently in development, 45 percent are adeno-associated virus (AAV) based delivery platforms. Other types include Lentiviral, which accounts for 13 percent.

A majority of the current pipeline products are in Phase II development and the most common neurology indications for which gene therapies are currently being evaluated include Parkinsons disease, pain and amyotrophic lateral sclerosis, said Vinie Varkey, Senior Analyst at GlobalData. The dominance of viral vectors is expected to continue as such platforms account for the bulk of these pipeline products, with adeno-associated virus being the most common among the viral vectors.

A high price point poses a challenge for the development of gene therapies, Varkey says, with key opinion leaders (KOLs) interviewed by GlobalData highlighting the need to create sustainable funding solutions so that such therapies become accessible to patients everywhere irrespective of where patients are located.

While the development of gene therapies are expected to pick up pace, the next wave of such therapies are expected to be ones that target diseases that are more frequent.

While monogenic rare diseases are the obvious first-to-go choice for which gene therapies can be developed, targeting more frequent diseases will need a holistic approach in order to address a wider mechanism of action, Varkey concludes. If gene therapies for frequent diseases do become available, then that will result in a more pronounced effect on healthcare not only in terms of providing better treatment options for patients but also test the ability of healthcare organisations to adapt with high price points of these therapies.

Excerpt from:
New report predicts growth of gene therapies for neurology indications - European Pharmaceutical Review

This entry was posted on October 29, 2019 at 2:45 pm and is filed under Gene therapy. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |