Biomapas Insights: From Hope to Global Impact – How Elpida Accelerated Gene Therapy for Rare Diseases

In this Biomapas Insights episode, Martijn van de Leur, Chief Commercial Officer, speaks with Terry Pirovolakis, CEO of Elpida Therapeutics, the organisation that grew out of the CureSPG50 advocacy movement. Together, they explore how a parent-led mission to help children with SPG50 advanced into a gene therapy organisation now developing multiple programs across rare neurodevelopmental disorders.

In this episode, you will hear:

  • The origin story of CureSPG50 and the creation of Elpida Therapeutics
  • What enabled Melpida’s outstandingly rapid clinical progress
  • How Elpida selects and collaborates with partners like UTSW and NIH
  • Insights into navigating FDA, EMA, and global regulatory pathways
  • The biggest unmet needs and long-term challenges in paediatric gene therapy

This conversation offers a rare and inspiring look at how patient advocacy, science, and collaboration with the right partners can redefine what’s possible for children with rare diseases.

Choosing the Right QPPV Partner for EU and Global Compliance

Choosing the Right QPPV Partner for EU and Global Compliance

Pharmacovigilance outsourcing has become crucial for biotech companies to remain compliant and safeguard patients. When you bring a drug to market in Europe or globally, your Qualified Person for Pharmacovigilance (QPPV) serves as your first line of defense. The right...

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Big Enough To Cover All Your Needs.
Small Enough To Care.

Discover how Biomapas provides all the support to accelerate your clinical trials and optimize your drug development process.

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Reliable support for every phase of your clinical trial, from recruitment to results.

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