Cell and gene therapies (CGTs) offer potentially curative treatments for various diseases by addressing underlying genetic or disease issues. Gene therapy replaces or modifies disease-causing genes, while cell therapy uses new cells to treat conditions. Currently, there are 34 FDA-approved therapies in the U.S., and more CGTs are on their way with ongoing clinical trials, with the majority focused on cancer treatment.
Gene therapy involves adding or replacing genes, often using modified viruses as vectors. In vivo gene therapy injects vectors directly into patients, while ex vivo modifies cells outside the body before injection. Casgevy™ is an example of a gene-editing therapy using CRISPR technology to treat sickle-cell disease.
Cell therapy directly fights diseases by restoring or altering cells outside the body before injection. Omisirge® helps blood cancer patients recover more quickly from treatments. CAR-T therapy combines CGTs technologies, modifying T-cells to attack cancer cells. Kymriah® is an example, in treating leukemia.
The pipeline for cellular and gene therapy is expanding, with products like fidanacogene elaparvovec and lifileucel advancing in clinical development. Fidanacogene aims to treat hemophilia B, while lifileucel is being tested as a treatment for advanced melanoma.
Although CGTs have the potential to address many unmet medical needs, there may be some associated risks such as fatal reactions and T-cell malignancies. High costs, ranging from $2 million to $3 million for gene therapy and up to $1 million for cell therapy, may also pose challenges to widespread adoption. However, while CGTs are extremely expensive, they are potentially curative and prevent or alleviate the need for long-term adherence to often complex maintenance medications. AscellaHealth recognizes the challenges and remains committed to working collaboratively with manufacturers in addressing these issues to facilitate the broader accessibility of these groundbreaking therapies.
Gene modification and cellular therapies hold promise to be life-changing medical treatments for a wide range of diseases such as cancer, hemophilia, sickle cell disease, muscular dystrophy, cystic fibrosis, and many others. These groundbreaking therapies are bringing hope to patients with previously incurable rare conditions.
AscellaHealth's dedication to innovation, patient-centric care and comprehensive end-to-end solutions positions the organization as a pivotal player in advancing CGTs. Beyond providing crucial financial solutions, we play a key role in streamlining the entire commercialization process of CGTs. Our services extend beyond funding, encompassing strategic support that accelerates the integration of CGTs into mainstream healthcare practices. By facilitating seamless communication between patients, prescribers and pharmacies, AscellaHealth continues to be a reliable and trusted partner, actively contributing to positive outcomes and enhancing overall access to cutting-edge treatments.
Access the latest AscellaHealth Specialty & Rare Pipeline Digest™ here.
For more information on CGTs, click here to read this issue's clinical bulletin.