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Are gene editing therapies the next step in treating rare genetic diseases?

Are gene editing therapies the next step in treating rare genetic diseases?

The gene editing field has evolved significantly in the past decade, from the advances in genetic tools within the field – CRISPR and zinc-finger nucleases – to the interest around its vast potential applications.



Research has indicated that approximately 10% of the global population (475 million people) are affected by a rare condition, though there is a lack of combined data in this area.

Only 5% of the people with a rare disease have a treatment, which can explain why gene editing therapies are rising on the agenda when it comes to the treatment of rare genetic diseases.

Are gene editing therapies the next step in treating rare genetic diseases, and what implications might arise in the future?

Cutting-edge collaborations and partnerships

As with many advances in the life sciences industry, anticipation is tied heavily to collaborations in the gene editing space.

One key example of this is a collaboration between Moderna and Life Edit Therapeutics, which is a strategic research and development collaboration focused on discovering and developing in vivo mRNA gene editing therapies.

This partnership aims to combine Life Edit’s suite of proprietary gene editing technologies with Moderna’s mRNA platform to advance in vivo gene editing therapies against a select set of therapeutic targets.

Under this agreement, Life Edit Therapeutics and Moderna will collaborate on research and preclinical studies, funded by Moderna, with the hope of harnessing the power of gene editing technologies as part of their broader R&D efforts in the future.

Treatment milestones

In the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) research communities, excitement around the therapy based on this technique is climbing as it gets closer to FDA approval.

CRISPR gene editing in particular has long attracted attention and anticipation in both research and wider communities, in part due to the greater level of accessibility it offers compared to other methods.

The implications for treatment milestones in this area are vast, and though no therapies using this approach have been approved yet, this could change soon.

Exa-cel (exagamglogene autotemcel), made by CRISPR Therapeutics and Vertex Pharmaceuticals for the treatment of sickle cell disease and transfusion dependent beta thalassemia, is an area close to potential approval.

These clinical successes are immensely significant to the future of gene editing therapy – the first approval will be a milestone for CRISPR therapies, and further establishes regulatory precedents in the area.

Gene editing is the next step

The traditional gene therapies that have been in development for decades contrast considerably with the current developments in the gene editing space.

CRISPR-based therapies, for example, have shifted from a primarily ex vivo model in earlier generations – tweaking cells removed from the patient and reinfusing them – to in vivo candidates, which are delivered directly into the patient like a gene therapy.

These developments are extremely significant to the future of gene editing because it signifies how much gene editing has adapted.

Whilst traditional gene therapy is most effective in tissue where cells aren’t dividing and reproducing themselves, CRISPR-based edits to the genome can be used in tissues with a higher turnover rate.

Investor interest is high 

The future of a therapeutic area doesn’t rest solely on investor interest, particularly at a time when there is such high uncertainty in public biotech markets.

However, since 2012, the number of venture capital (VC) deals for companies developing gene editing technologies has increased significantly.

The total value of VC deals since 2012 has reached over $3.2bn, with over $1.3bn raised in 2021 alone (over 250% more than what was raised in 2020).

The potential for this industry is also what draws interest – the global genome editing market is expected to hit $21.36bn by 2030, with a CAGR of 17.33% during the forecast period.

The European gene editing market is projected to grow at 14.9% CAGR between 2020 and 2026, catalysed by considerable R&D expenditures across biopharmaceutical and pharmaceutical companies.

CRISPR is anticipated to be the leading segment by technology, with cell line engineering being the leading segment by application.

As investments increase in enhancing the current technologies in gene editing therapy, the market will continue to expand rapidly.

Factors continue to drive the growth of gene editing

From the increasing prevalence of infectious diseases and genetic disorders to the rising expenditures on research and development, there are multiple factors influencing the growth of gene editing.

Personalised medicine has become a huge area of interest for many in the industry, which is only furthering the growth of the gene editing market as a result.

Germany’s genome editing market in particular contributed more than 18.5% of the total market share in 2019 and is expected to continue this trend into 2023 and beyond.

One of the reasons behind Germany’s contributions to the market is the significant increase in genetic disorders and chronic illnesses (e.g. cancer and diabetes) which have increased the adoption of and interest in gene editing.

What does the future hold?

In spite of continuing uncertainty in the biotech market, gene editing therapy and technology has continued to generate investor interest, and as new developments continue to unfold and treatments start to gain approval, this will only increase.

Gene editing is also likely to niche down, particularly against gene therapy – gene editing may be more appropriate for certain patients, or it might be the case that gene editing finds a niche that is further removed from the competition (traditional gene therapy).

The progress in this area has already been considerable, but there is a long way to go to truly measure the impact that gene editing could have on the treatment of rare genetic diseases.

Only time will tell!

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