Intellia Therapeutics Success In Gene Therapy Leaps Ahead
New partnership with Regeneron yields promising results
Intellia Therapeutics (NASDAQ: NTLA) and its strategic partner Regeneron are on to something big in gene therapy and anticipation is building for future breakthroughs.
Intellia is a biopharmaceutical research-driven innovator focused on the development of gene-editing therapies to help patients with autoimmune, genetic and oncology healthcare challenges.
It is refining its use of clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing technology and creatively developing new options for patients and medical professionals.
Ex vivo versus in vivo: Intellia has both capabilities
In a recent clinical trial, Intellia administered gene therapy NTLA-2001 in vivo to a patient and achieved positive outcomes. The in vivo approach is notably different than others.
With ex vivo therapy development the biological material is extracted from a patient’s body, a treatment is applied to it and then it is injected back into the patient. Through using the in vivo approach, Intellia administered the CRISPR-based NTLA-2001 therapy directly to the liver to treat transthyretin (ATTR) amyloidosis.
ATTR amyloidosis is a rare and fatal disease. Hereditary ATTR (ATTRv) amyloidosis is triggered when a patient is born with mutations in their TTR gene. This causes the liver to produce abnormal transthyretin (TTR) proteins. These abnormal proteins accumulate as amyloid in a patient’s system and progressively create more serious health complications in the digestive, heart, nerve and other tissues.
NTLA-2001 is comprised of a two-part genome editing system. The first unit is a guide RNA specific to the disease-causing gene. The second unit is messenger RNA (mRNA) to encode the Cas9 protein and execute the precision gene editing. mRNA technology has been in the headlines lately as it has been used to develop Moderna and Pfizers COVID-19 vaccines.
Results of the clinical trial are quite promising so far. NTLA-2001 demonstrated its effectiveness by lowering serum levels of transthyretin by 87% when patients received a higher dose of the therapy. These outcomes exceeded the standard of care therapy that usually cuts transthyretin by 80%. Part of the clinical trial focus moving forward is to determine is NTLA-2001 is effective enough that results are sustained for a reasonable length of time.
Intellia works on the development of ex vivo and in vivo therapies. In the case of NTLA-2001, it is believed the combination of in vivo administration, the two-part gene-editing system and mRNA technology could be a more effective way of developing and deploying gene therapy to a wider number of patients.
Gene therapy has many promising uses but few solutions
There are many disease candidates for which gene therapy could be an effective answer but it still needs to advance itself scientifically and clinically.
Gene therapy research and development is time-consuming, medically complex and costly. Presently, there are less than 25 FDA-approved gene therapies available for use. They are each highly targeted and not able to treat a wide variety of patients and diseases.
Regeneron and Intellia Therapeutics join forces
Founded in 2014 and headquartered in Cambridge, Massachusetts, Intellia is collaborating with Regeneron, another advanced biotherapy innovator, on the gene therapy development initiatives. Regeneron (NASDAQ: REGN) is based in Tarrytown, New York and was started in 1988.
Regeneron produced about $8.5 billion in sales during 2020. The company has achieved a string of successes in the last 18 months. In addition to its breakthrough work with Intellia, Regeneron’s REGEN-COV2 (a monoclonal antibody used to treat COVID-19) has earned FDA emergency use authorization.
Intellia and Regeneron started working together on in vivo CRISPR/Cas9 gene-editing technology and NTLA-2001 in 2016. Patients, medical professionals and investors all have much to look forward to and gain from in the partnership’s success.
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