Unlocking Potential: Exploring Cell and Gene Therapies in Disease Treatment

Cell and gene therapies potential treatment options for various diseases.
AscellaHealth EU
· 7 min read

Cell and gene therapies (CGTs) offer potentially curative treatments for various diseases by addressing underlying genetic or disease issues. Currently, there are over 19 EMA-approved therapies in the EU, and more CGTs are on their way with approximately 2000 therapies in development worldwide with the majority focused on cancer treatment.

Cell therapy directly fights diseases by restoring or altering cells outside the body before injection. Ebvallo® is used to treat relapsed or refractory Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD), a rare type of cancer.

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.

CAR-T therapy combines CGT's technologies, modifying T-cells to attack cancer cells. Kymriah® is an example of CAR-T therapy used in the treatment of certain types of leukaemia.

The pipeline for CGT is expanding. While CGTs have the potential to address many unmet medical needs, there may be some associated risks, and regulatory agencies such as the EMA, MHRA, and FDA are monitoring these closely. Relatively high treatment costs, often in the region of €2 million, may also pose challenges to widespread adoption. However, while CGTs are expensive, they are potentially curative and prevent or alleviate the need for long-term adherence to often complex maintenance medications and other medical management. AscellaHealth recognises these challenges and remains committed to working collaboratively with manufacturers in addressing these issues to facilitate broader access for patients.

CGTs hold promise to be life-changing medical treatments for a wide range of diseases such as cancer, haemophilia, 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 organisation as a pivotal player in advancing CGTs. Beyond providing crucial financial solutions, we play a key role in streamlining the entire commercialisation 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 is 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.

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