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Regeneron, UPenn to use gene therapy tools to deliver a COVID-19 antibody drug

Dive Brief:

  • Regeneron is investigating the possibility that its antibody-based treatment for COVID-19 – an infusible drug recently used to treat President Trump – may protect against coronavirus infections when given as a nasal spray.


  • The biotech is teaming up with gene therapy pioneer James Wilson of the University of Pennsylvania on a new way to deliver the drug combination that might make it useful as a convenient, preventive treatment. The FDA recently cleared the treatment, known as REGEN-COV-2, to treat patients with mild- to moderate cases of COVID-19. But it must be administered at a clinic or hospital and its effects are temporary.


  • The researchers aim to spur a “sustained expression” of antibodies in the nose, where the respiratory infection is often introduced, Wilson said in a press release. A quick and long-lasting response could represent an alternative to vaccination and be critical for health-care workers on the front lines and people who have weaker immune systems.

Dive Insight:

Regeneron is exploring new ways of using its combination treatment after winning an emergency to treat patients who have mild to moderate symptoms but are at high risk of severe disease or hospitalization.

Clinical results have shown the drug to be particularly helpful for patients early on in their disease course whose immune systems haven’t yet mounted a defense against the novel coronavirus. But the company is hoping for broader use, with ongoing studies in hospitalized patients and in uninfected people who are at high risk of exposure, such as a family member of someone who has contracted the virus.

The work with Wilson, a pioneer in the use of adeno-associated viruses for gene therapy, is part of that effort, albeit at a much earlier stage. The idea is to load up an AAV with the genetic blueprints for the neutralizing antibodies in REGEN-COV2, and then spray them into the nose. The hope is doing so will lead to a fast-acting immune response that protects against infection, an effect that kicks in more quickly than a vaccine.

Previous studies have shown that Regeneron’s therapy, given intravenously or through an injection, produces antibody levels that last a month or more. Wilson and his team are hopeful that a single dose delivered by a nasal spray will offer protection for a longer period of time, making it a potential alternative to those who might not respond to a vaccine.

The research team will start by giving the antibody combination to animals and then exposing them to the coronavirus. The next step would be seeking FDA approval to begin research on humans.

AAVs, meanwhile, are now routinely used in gene therapy. Penn said Wilson’s AAV vectors are currently being used in at least 90 preclinical and 40 clinical programs throughout the biotech industry.