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534,154 Results for "trucode gene repair inc".
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Neuroscience
UniQure’s chronic epilepsy gene therapy ‘should be on investors’ radar’
As uniQure prepares its closely watched Huntington’s disease gene therapy for FDA review, the biotech is also making waves in chronic epilepsy with another gene therapy that Stifel says is off to a “promising start.”
June 22, 2026
·
3 min read
·
Tristan Manalac
Press Releases
Repair Biotechnologies Granted Rare Disease Evidence Principles (RDEP) Eligibility by the FDA for REP-0003 in Homozygous Familial Hypercholesterolemia
May 12, 2026
·
3 min read
Rare diseases
Intellia’s gene editor ‘keeps pace’ with Ionis in hereditary angioedema
With a one-time dosing profile, Intellia Therapeutics’ gene editing asset could be “paradigm-shifting” for hereditary angioedema, according to Jefferies. The biotech anticipates market approval next year.
June 16, 2026
·
2 min read
·
Tristan Manalac
IPO
Scribe reels in nearly $129M in rare early-stage IPO for a gene editing biotech
Jennifer Doudna–founded Scribe Therapeutics intends to use the proceeds from the public offering—the first for a gene editing outfit in over two years—to support development of its epigenetic silencing therapy for atherosclerotic cardiovascular disease.
July 24, 2026
·
3 min read
·
Tristan Manalac
Duchenne muscular dystrophy
REGENXBIO bets on FDA flexibility with filing plans for Duchenne gene therapy
A few short days after announcing an FDA pivot on a separate asset, REGENXBIO is planning to test the agency’s apparent newfound rare disease outlook on another late-stage gene therapy.
June 24, 2026
·
2 min read
·
Gabrielle Masson
gene editing
Gene therapy leaders aim to help more Baby KJs with novel regulatory models
As cell and gene therapy leaders gathered in Maryland to discuss accelerating clinical trials in children, one “cutting edge” session focused on the need to expedite more bespoke gene editing treatments like the one that saved young KJ Muldoon.
April 27, 2026
·
5 min read
·
Heather McKenzie
Press Releases
Nyra Medical Announces First Patient Enrollment in ENHANCE Early Feasibility Study, Advancing Next Generation Mitral Valve Repair Platform
July 7, 2026
·
5 min read
Press Releases
GRI Bio Delivers Compelling New Phase 2a Gene Expression Data Demonstrating Improvements in Key Drivers of Fibrosis and Lung Repair in IPF
January 29, 2026
·
8 min read
Editorial
BIO 2026: Cell and gene therapy sector must break the mold to thrive
If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
June 22, 2026
·
4 min read
·
Jef Akst
Opinion
The next FDA commissioner must be a champion for gene therapies
The FDA must provide consistent and predictable regulatory frameworks if the U.S. is to maintain its leadership in gene therapy, one of the most consequential therapeutic fields of our generation.
June 5, 2026
·
4 min read
·
Erik Paulsen
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