Phase I
The Phase I study could potentially result in RP-A501 becoming the first gene therapy for monogenic heart failure.
ABVC Biopharma, based in California, has programs in Phase II trials to treat major depressive disorder, adult attention deficit hyperactivity disorder and myelodysplastic syndrome.
Healx brings the power of artifical intelligence (AI) to rare disease research with an ultimate focus on improving patient outcomes.
Recent gene therapy research has created a durable response in terms of producing normally-shaped red blood cells and reducing the pain of sickle cell disease.
LEXEO Therapeutics has announced positive initial data from its Phase I/II trial of LX1001, a gene therapy intended for the treatment of APOE4 homozygous Alzheimer’s disease (AD).
AbbVie has bolstered its neuropsychiatric and neurodegenerative portfolio with the acquisition of Belgium-based Syndesi Therapeutics in a deal valued at up to $1 billion.
AstraZeneca and Neurimmune have signed an exclusive global collaboration and licensing deal to develop, manufacture and commercialize NI006 for ATTR-CM.
Positive interim data from the global Phase I trial of single-dose NTLA-2001 demonstrated a mean serum TTR reduction of 93% at the highest dose level of 1.0 mg/kg by day 28.
Celyad announced that it is voluntarily pausing a clinical trial it’s conducting with Merck after reports of two patient deaths.
The FDA places holds, companies receive clearance for new studies and Pharma giants release new data in last week’s clinical trial news.
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