Working to Improve Gene Therapy for Adrenoleukodystrophy and Other Rare Conditions

Rare disease research is often underrepresented in the field of science. However, recent developments have made researchers more interested in studying this group of diseases. For instance, investments in gene therapy have skyrocketed recently. This is amazing news for the rare community. But, it does bring about a new set of concerns.

One of the questions that’s come up as gene therapy research has progressed is- will manufacturers be able to keep up with the growing demand?

Manufacturing dilemmas

Last year, the first directly administered gene therapy was approved by the FDA to treat a form of vision loss. Since then, gene therapy developments have increased at a rapid pace. Bluebird bio for instance, is currently working on gene therapies for Beta Thalassemia, multiple myeloma, sickle cell disease, and cerebral adrenoleukodystrophy among others.

One of the biggest issues in manufacturing new gene therapies is also one of the best things about this innovative treatment. Each treatment is not only specific to the rare disease, but specific to the individual patient. So, since it uses the patient’s own cells, it takes more time to develop than a general drug. Additionally, because the researchers rely on the patient so much during the manufacturing process, scheduling appointments that work for everyone involved can be difficult. Not only that, but it can be a burden for patients who have to travel far distances to a treatment center or for patients who have difficulty traveling.

The goal is for all patients to receive the treatment within 2 to 4 weeks of when they start the gene therapy process. But it’s a system researchers are still working to improve and make faster.

Looking Forward

The best thing about gene therapy is that it works. The worst thing is that it’s inefficient and expensive. But, the fact that researchers know it has flaws and are actively trying to reform the system is what’s important. The tricky part is not messing up the progress they’ve made as they do this. Unfortunately, in order to do that, they have to move much slower than we’d like.

That said, new approaches are constantly being analyzed and there are ongoing discussions regarding new ideas and partnerships. Despite the issues with gene therapy manufacturing, we all know rare communities will be better off in the future now that the idea is being developed. It’s just sometimes difficult to stay patient through the process.

You can read more about gene therapy manufacturing and the newest developments concerning this treatment method here.


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