Ursule Demael


This episode is a conversation with Professor Bobby Gaspar, the CEO of Orchard Therapeutics, a clinical stage biotech company developing gene therapies for severe diseases. Signal host Ursule Demael speaks to Prof Bobby Gaspar, who holds an MD/PhD and is an honorary professor of Paediatrics and Immunology at Great Ormond Street Hospital in London (United Kingdom). He has decades of expertise across academic research and biotech developing haematopoietic stem cell therapies for patients with immune deficiencies and metabolic disorders. Orchard’s lead therapy Libmedly is approved for the metabolic disorder MLD in Europe.

Show Notes

[02:15] Bobby Gaspar’s background

[05:10] The science of haematopoietic stem cell therapy

[12:30] Spinning out Orchard Therapeutics

[21:15] Reverse partnerships with pharma

[25:55] Balancing regulatory compliance and innovation

[29:45] Value based payments 

[35:15] Recent approval of Casgevy and gene therapy regulation

[39:45] Failures in a biotech’s journey

[43:00] Impact and meaning in therapeutics companies

Orchard Therapeutics Website

FDA Approval of Libmeldy

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