Gene-Editing shot aims to fix rare lung and liver disease
NCT ID NCT07227207
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a new gene-editing medicine called TSRA-196 in 72 adults with a severe form of alpha-1 antitrypsin deficiency (the PiZZ genotype) who have lung or liver problems. The treatment is given by IV infusion and aims to raise levels of a protective protein. The study will check safety and whether the protein levels increase enough to help.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
TSRA-196 (a gene-editing medicine given by IV infusion)
What this could lead to
If successful, this could lead to a one-time or infrequent treatment that raises protective protein levels, potentially slowing or stopping lung and liver damage in people with severe alpha-1 antitrypsin deficiency.
What could go wrong
This is an early-phase trial with only 72 participants, so safety and effectiveness are not yet proven. Gene-editing therapies can have unexpected side effects, and the long-term risks are unknown.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Clinical Study Center
RECRUITINGBoston, Massachusetts, 02118, United States
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Clinical Study Center
RECRUITINGCharleston, South Carolina, 29425, United States
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Clinical Study Center
RECRUITINGFitzroy, Victoria, Australia