All articles tagged with #intellia therapeutics
Intellia Therapeutics has temporarily paused its Phase 3 clinical trials of Nex-z for transthyretin amyloidosis after a patient experienced severe liver-related side effects, with plans to investigate and consult with regulators before resuming the trials.
Intellia Therapeutics' stock dropped 19% after a safety update revealed a case of elevated liver enzymes in its ongoing Phase 3 trial for gene therapy, though the event is resolving without severe intervention. Despite the incident, analysts remain cautiously optimistic, citing the rarity of the event and ongoing positive trial progress, with some viewing the market reaction as an overreaction.
Wall Street analysts are optimistic about the potential for Ginkgo Bioworks and Intellia Therapeutics, with consensus price targets suggesting significant gains of 144% and 202% respectively over the next 12 months. Ginkgo Bioworks, a synthetic biology company, has faced financial challenges despite its innovative work, while Intellia Therapeutics is developing CRISPR-based gene therapies with promising clinical trial data but faces a long road to commercialization.
Intellia Therapeutics sought to address concerns about accidental germline transmission in CRISPR trials by sharing detailed information about studies conducted to assess the risks. The company stated that there is no evidence of vertical germline transmission of renegade CRISPR components. Intellia is a leading company in the field of in vivo gene editing therapies and has previously demonstrated the successful alteration of DNA in human cells to treat disease. However, during toxicology studies in non-human primates, editing was found to have occurred at low levels in organs other than the liver, including the spleen, adrenal glands, and ovaries of female monkeys.
The FDA has approved Intellia Therapeutics to begin testing a drug that uses CRISPR gene editing in vivo, marking the first time ever that the FDA has approved such testing. The drug would prevent swelling attacks in people with a genetic condition called hereditary angioedema. Typically, treatments and drugs that utilize CRISPR take place outside the body, but Intellia's edited media finds its own way to the liver rather than being injected there.