ASGCT Addresses Challenges and Potential Solutions to Development in LMICs

Johnny Mahlangu, BSc, MBBCh, MMed, FCPath - July 14, 2021

While there has been significant clinical advancement of gene therapy in the US, EU, and Japan, low- and middle-income countries (LMICs) do not have the same access to resources needed for development.

Given the prevalence of diseases in LMICs that could potentially be treated with gene therapy, an ASGCT strategic goal is to foster equitable access to gene therapy in these countries. To address this goal, ASGCT hosted two virtual events last month focused on identifying solutions to the development challenges in LMICs. 

ASGCT hosted a half day program on June 29—Advancing Gene and Cell Therapies in South Africa—as part of ASGCT’s partnered educational programming to advance clinical development of gene therapies in middle-income countries that have burgeoning medical or regulatory systems. Stimulating such additional clinical research is an important step in expanding availability and access to these therapies for patients in need in the area. ASGCT partnered with five professional societies and other nonprofits to host the event for participants in South Africa. 

Key insights from the program include the need for collaboration and partnerships to bring similar educational webinars to Africa. It was clear from this webinar that ASGCT, with its global partners in all continents, is the right vehicle to convene meetings of this nature. The webinar was a huge success by all counts, and it highlighted the need for more focused working groups to discuss such topics as the ethics of gene and cell therapies, funding models, manufacturing groups, and creating empowering partnerships.  

A session of the Forum on Gene Therapy for Underserved Populations last month addressed some additional elements needed to overcome challenges to gene therapy development—a regulatory framework specific to gene therapies and financial investment. 

Speakers discussed the possibility of utilizing local manufacturing of CAR T-cell therapy to reduce costs, and funding of research on in vivo gene therapies for HIV and sickle cell disease to develop treatments that are more transportable. Both methods could increase access to these potentially curative therapies in LMICs. 

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