Currents of disruption: gene therapy offers rare hope

22/10/2018

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Our understanding of the genetic causes of disease has reached a critical stage, putting us on the cusp of being able to develop functional cures for a large number of rare genetic disorders, says Global Life Sciences Co-Portfolio Manager, Andy Acker.
 
Key takeaways:
  • Gene therapy has the potential to provide “one and done” treatments that can correct genetic defects that cause life-threatening diseases.
  • Biopharmaceutical companies are working on treatments for the more than 7000 known genetic disorders, including haemophilia, muscular dystrophy and spinal muscular atrophy – the biggest genetic cause of infant death.
  • As investors, we recognise the importance of looking not only at large, public companies working on gene therapies, but also at the smaller innovators that have the potential to be disruptive.

 Video recorded on 1 September 2018.

 

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