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Okay, well, maybe not quite, but its closer...
(Just as an FYI, I don't represent this company in any way, but I couldn't find any non-company releases on the SUNRISE trial)
Who knows if this will actually work, but I think gene-editing for congenital diseases is really the wave of the future in pediatrics. The highest mortality in pediatrics in under age 1 and due to congenital anomalies. Clearly, not all those anomalies are enzyme specific, but there's a good number that are. Anyway, I think this is incredibly exciting and really just the beginning...
Discuss the pros and cons of gene-editing, or don't, because either way... the future is HERE!
LogicBio Therapeutics Announces First Patient Dosed in Groundbreaking Phase 1/2 SUNRISE Clinical Trial for the Treatment of Pediatric Patients Suffering from Methylmalonic Acidemia | LogicBio Therapeutics, Inc.
- Company believes dosing represents first in vivo gene editing therapy delivered systemically to a pediatric patient - Clinical trial designed to treat patients as young as 6 months old with LB-001 based on proprietary GeneRide™ platform - Company remains on track to announce update on enrollment,
investor.logicbio.com
Who knows if this will actually work, but I think gene-editing for congenital diseases is really the wave of the future in pediatrics. The highest mortality in pediatrics in under age 1 and due to congenital anomalies. Clearly, not all those anomalies are enzyme specific, but there's a good number that are. Anyway, I think this is incredibly exciting and really just the beginning...
Discuss the pros and cons of gene-editing, or don't, because either way... the future is HERE!