disease

Intellia Announces First Clinical Evidence from Ongoing Phase 1 Study that Nexiguran Ziclumeran (nex-z), an In Vivo CRISPR/Cas9-Based Gene Editing Therapy, May Favorably Impact Disease Progression inTransthyretin (ATTR) Amyloidosis

Consistently rapid, deep and durable reduction in serum TTR accompanied by evidence of disease stabilization or improvement after a one-time…

1 month ago

Intellia Announces First Clinical Evidence from Ongoing Phase 1 Study that Nexiguran Ziclumeran (nex-z), an In Vivo CRISPR/Cas9-Based Gene Editing Therapy, May Favorably Impact Disease Progression inTransthyretin (ATTR) Amyloidosis

Consistently rapid, deep and durable reduction in serum TTR accompanied by evidence of disease stabilization or improvement after a one-time…

1 month ago

Numab Therapeutics and Kaken Pharmaceutical Enter Collaborationand Option Agreement for Multi-specific Antibody ND081 for Treatment of Inflammatory Bowel Disease

Numab to receive R&D funding for the preclinical development of ND081 Kaken Pharmaceutical receives an option to in-license ND081 in…

1 month ago

AlveoGene Receives Rare Pediatric Disease Designation (RPDD)from FDA for AVG-002, its Novel, Inhaled Gene Therapy forLethal Neonatal Surfactant Protein B (SP-B) Deficiency

Inherited SP-B deficiency is an ultra-rare monogenic cause of fatal respiratory distress syndrome in newborn infantsClinical estimates for SP-B deficiency…

1 month ago

deCODE genetics/Amgen: New Study Explores the Role of BMI in Disease Risk

REYKJAVIK, Iceland, Nov. 12, 2024 /PRNewswire/ -- New study from deCODE genetics/Amgen highlights the importance of BMI in pathogenesis of disease,…

1 month ago
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