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Jupiter Neurosciences Makes Way in CNS Treatments with $11M IPO

XTalks

In addition, preclinical studies of Jotrol in a Parkinson’s disease mouse model at the University of Miami have shown promise, addressing hallmark symptoms like nigral cell loss and dopamine deficits. Proceeds from the IPO will propel key initiatives, including the Phase II clinical trial of Jotrol in Parkinson’s disease.

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Astellas to support development of Taysha’s gene therapy programmes

Pharmaceutical Technology

TSHA-102 is the first gene therapy in the clinical development stage for Rett syndrome currently while TSHA-120 is being analysed in a Phase I/II clinical trial for treating GAN. Astellas will also receive specific rights linked to any possible change of Taysha’s control.

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Neurocrine Biosciences’ Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH

XTalks

Related: Arrowhead Pharmas New Rare Disease Campaign for FCS Highlights Importance of Lowering Triglycerides The FDAs approval of Crenessity was based on two randomized, double-blind, placebo-controlled trials in 182 adults and 103 children with classic CAH. Crenessity marks the third approved drug for Neurocrine.

Genetics 116
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Astellas makes another gene therapy play, takes stake in Taysha

pharmaphorum

The Japanese firm has agreed to make a $50 million investment in Dallas-based Taysha in exchange for a 15% stake in the company, plus exclusive options to license two clinical-stage, single-gene therapies for rare genetic diseases.

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Surrogate Endpoints: How to Choose the Best One for Your Rare Disease Trial

XTalks

In rare disease trials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. Verifying the biomarker’s clinical validity for use as a surrogate endpoint in rare disease research is another hurdle which is generally a longer-term goal. What Is a Surrogate Endpoint?

Trials 98
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Eli Lilly Pursues RNA Editing in New Partnership with ProQR

XTalks

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

RNA 105
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Rare diseases, repurposing and the role of AI

pharmaphorum

In the age of artificial intelligence, no trial data should be going to waste. Findacure’s Rick Thompson looks at how these technologies could bring us closer to treatments for underserved rare diseases. The repurposing of drugs is becoming more common, especially in the field of rare diseases.

Trials 105