We live in an extraordinary time.

It's a time where technology in life sciences is peeling back the curtain on the underlying causes of disease.

In this note, I'll cover recent developments in gene editing therapies:

• A background on CRISPR
• The impact of CRISPR technology on sickle cell disease
• Prospects for FDA approval and for the future

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Background

First of all, what IS CRISPR?

CRISPR stands for “clustered regularly interspaced short palindromic repeats”.  “Palindromic” means that they “read” the same way – have the same sequence – whether you read them backward or forward. They are the hallmark of a bacterial defense system against virus attacks. 

This revolutionary discovery, for which Jenifer Doudna and Emmanuelle Charpentier received 2020 Nobel Prize in Chemistry, permits editing the genomes of living organisms.  In a sense, it allows us to “edit” who we are.

The revolutionary advancements in gene editing, particularly CRISPR technology, are ushering in a new era of hope for individuals suffering from genetic disorders like sickle cell disease.

Impact of CRISPR

A recent example of this is the experimental therapy exa-cel, developed by Vertex Pharmaceuticals and CRISPR Therapeutics. This therapy, which involves editing a patient's own stem cells, has shown life-changing results for patients. The therapy, however, does not remove the genetic mutation itself but rather mitigates its most harmful effects.

Sickle cell disease is characterized by misshapen red blood cells that cause blockages and severe pain. It has been a significant health challenge, particularly in populations of African ancestry. Traditional treatments like hydroxyurea or stem cell transplants have limitations, either in efficacy or availability. Exa-cel represents a significant leap forward, offering a potential solution to a disease that has long plagued millions.

However, the path to this treatment is not without challenges.

The therapy is customized for every patient, requiring a complex and expensive process of collecting, editing and reinfusing a patient's own stem cells. Additionally, the preparatory chemotherapy regimen needed for the treatment is arduous and poses risks like infertility.  That's a significant consideration for younger patients.

Another concern is the long-term effects and potential risks associated with CRISPR technology. Most experts consider these risks as minimal.  But, in truth, we don't have clinical evidence to prove that.

Despite these challenges, the success of exa-cel in clinical trials is undeniable. Most participants have remained free of pain crises, and the overall quality of life has improved dramatically for those treated. This success has stirred optimism about the future of gene editing in treating genetic diseases.

Prospects – FDA Approval & Beyond

However, it also raises questions about accessibility and affordability, especially given the potential high cost of the therapy and the socio-economic dynamics of the populations most affected by sickle cell disease.  Those are populations less likely to be able to bear the cost of the therapy.

The upcoming FDA decision on exa-cel, expected by December 8, 2023, is not just a turning point for sickle cell treatment but also a significant milestone in the field of genetic medicine. If approved, exa-cel would be the first CRISPR-based therapy in the market, marking a new chapter in medical science. This development, while promising, also brings to the forefront the ethical, emotional, and financial complexities inherent in such groundbreaking treatments.

Patients  who have undergone this treatment not only experience a dramatic shift in their health but also face new identities and life perspectives post-treatment. Their journeys highlight the profound impact of these therapies, beyond the physical, shaping a future where genetic diseases might be a thing of the past, but also presenting new dilemmas in the realm of medical ethics and healthcare equity.

For a more comprehensive discussion of these issues, see the recent article in BIOPHARMADIVE.