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BridgeBio’s Attruby (Acoramidis): A New, More Potent TTR Stabilizer for ATTR-CM

XTalks

BridgeBio has been highly active in the genetic diseases space. Its investigational therapy infigratinib recently received Breakthrough Therapy designation, becoming the first-ever potential treatment for achondroplasia — a genetic condition affecting bone growth — to achieve this milestone.

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Astellas to support development of Taysha’s gene therapy programmes

Pharmaceutical Technology

Additionally, Astellas will obtain an exclusive option for licencing two clinical-stage programmes of Taysha, namely TSHA-102 and TSHA-120, for Rett syndrome and GAN, respectively. Astellas will also receive specific rights linked to any possible change of Taysha’s control.

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Forge Biologics joins BGTC for new AAV gene therapies development

Pharmaceutical Technology

The BGTC aims to expedite the development, manufacture and delivery of customised or “bespoke” gene therapies to treat rare diseases, including some which are too rare to be of commercial interest. The collaboration creates a reusable standardised method that minimises upfront costs and helps to reduce development barriers.

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Merck and Orna partner for RNA technology-based vaccines and therapies

Pharmaceutical Technology

Merck will also make royalty payments on any approved products developed out of the partnership. The rights to the oRNA-LNP technology platform of Orna will be retained by the company, which will also progress various other fully owned programmes in oncology and genetic disease areas.

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Gene Therapy and Pharmacokinetics

Camargo

When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.

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New White Paper on Advanced Therapies Sets up Developers for Success

XTalks

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. The Xtalks White Paper, The Intersection of Rare Disease and Advanced Therapies: What it Means for Clinical Development , is available for free download. “They called him ‘The Boy.’

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Chiesi’s flagship Fabry drug heads for FDA verdict in early 2021

pharmaphorum

The enzyme replacement therapy (ERT) – also known as PRX-102 – has been granted a priority review by the US regulator, and is the top prospect in Chiesi’s recently formed rare diseases division. Market research firm Optima Insights has predicted that sales of Fabry disease drugs will more than double from around $1.8

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