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Early trial data supports neurodegenerative disease-modifying drug

Drug Discovery World

Biotechnology company Allyx Therapeutics has reported positive Phase Ib clinical data for its lead compound, ALX-001, a first-in-class, synapse-targeted, disease-modifying oral therapy in development for neurodegenerative diseases. All doses were well-tolerated and there were no serious adverse events.

Disease 130
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Investigating potential for an oral treatment in diabetic retinopathy  

Drug Discovery World

Breye Therapeutics, a clinical-stage biopharmaceutical company developing novel oral therapies for retinal vascular diseases within ophthalmology, has started a Phase Ib/IIa clinical trial to investigate danegaptide, following oral administration, in patients suffering from diabetic macular oedema (DMO).

Treatment 130
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Phase Ib/IIa trial in diabetic macular oedema successfully completed  

Drug Discovery World

Exonate, an mRNA therapy company focused on treatments for diabetic complications, has announced that its lead ophthalmology asset, EXN407, has achieved its prespecified endpoints in a Phase Ib/IIa study. During the trial, EXN407 met all safety and pharmacokinetic parameters and displayed encouraging signals of biological activity.

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Positive initial data on first mRNA therapy for propionic acidemia

Drug Discovery World

The ongoing global Phase I/II clinical trial is a multicentre, open-label study designed to assess the safety, pharmacodynamics, and pharmacokinetics of mRNA-3927 in participants aged one year and older with genetically confirmed PA. Currently, there are no effective therapies for PA that target the underlying root cause of the disease.

Therapies 130
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Latest advances in immunotherapy from SITC 2023

Drug Discovery World

Dense aggregates of immune cells, including CD8 positive granzyme B positive cytotoxic T cells, dendritic cells, and B cells, were observed in PDAC tissue after CAN-2409 treatment, confirming activation of a robust antitumoral immune response.

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FDA grants orphan drug status for uveal melanoma drug

Drug Discovery World

Uveal melanoma is a disease in which cancer originates in the tissues of the eye, causing symptoms such as blurred vision or a dark spot on the iris. . When the cancer metastases, which it does in approximately 50% of patients, there are limited treatment options and projected overall survival is only a year. .

FDA 130
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Validating a revolutionary therapy against lethal haemorrhage 

Drug Discovery World

million from the Spanish Research Agency (AEI), under the call for grants for Public-Private Collaboration Projects, to accelerate the development of an innovative treatment aimed at controlling disabling and lethal haemorrhage.

Therapies 130