By: The Female Catalyst Founders
The Female Catalysts founders recently participated in a conference centered on improving access to cell and gene therapies (CGTs). The event gathered industry leaders striving to address the challenges within our healthcare system, particularly in covering the costs of one-time treatments.
CGTs encompass both cell therapy and gene therapy, which are transformative fields in biomedical research and treatment. While both aim to treat, prevent, or potentially cure diseases, they operate differently, targeting the root causes of genetic disorders.
Cell therapy involves modifying cells outside the body before reintroducing them to the patient, whereas gene therapy focuses on altering genes within cells, either inside or outside the body. Some therapies combine both approaches, aiming to correct genetic defects.
Currently, there are few FDA-approved CGTs, including Casgevy and Lyfgenia, which are groundbreaking cell-based gene therapies for treating sickle cell disease (SCD) in patients 12 years and older. Casgevy notably utilizes innovative genome editing technology, marking a significant advancement in gene therapy.
Despite their potential, CGTs face hurdles, including high one-time costs and the complexity of manufacturing therapeutic agents to meet pharmacological standards.
There is still much progress needed in research and improving access to CGTs, underscoring the ongoing journey in this transformative field.
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