Lasting seizure freedom for patients suffering from drug-refractory focal epilepsy

Human
Science and Technology

AAV Gene Therapy

Therapeutic area:
  • Neurological diseases
  • Epilepsy
  • Temporal lobe epilepsy

Problem:

The most common clinical presentaion of focal epilepsy is temporal lobe epilepsy with up to 2M patients in Europe, North America and Japan, and >100.000 new cases every year.

The current standard of care is antiseizure medication which often fails and has severe side effects. Epilepsy surgery as current second-line therapy is invasive and only available for patients with a well-defined and accessible focus. Even then, lasting seizure freedom cannot be guaranteed.

Medical need is high to find a better solution!

Product & USP:

EpiBlok has developed an AAV-based gene vector to suppress focal seizures by minimally invasive, one-time CNS delivery. AAV-derived neuropeptides, called dynorphins are produced and stored in neurons and only released upon high-frequency stimulation, as is the case at the onset of seizures.

EpiBlok’s AAV-pDyn is unique for its “release-on-demand” format of drug delivery, which prevents the long-term risk of habituation.

Team

Regine Heilbronn

Co-founder and CEO of EpiBlok Therapeutics, Berlin

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Until 2020 Director of the Institute of Virology at Charité Universitätsmedizin Berlin, Germany Professor Heilbronn has transitioned as cofounder and CEO of EpiBlok Therapeutics after receiving a €3.9M GO-Bio founders’ award to develop a gene therapy for focal epilepsy. She looks back to 30+ years of research on AAV virology and vector technology starting with Nobel Laureate Harald zur Hausen in Heidelberg, then as Heisenberg Fellow and group leader at the Max-Planck-Institute for Biochemistry in Martinsried before being appointed as Full Professor of Experimental and Clinical Virology at the Free University of Berlin. She authored numerous highly cited publications and a series of patent applications on AAV vectors and gene therapy.

Prof. Heilbronn studied medicine in Düsseldorf, Glasgow UK, and Freiburg i.Br. where she gained an M.D. in biochemistry, and in 2020 an Executive MBA from the European School of Management and Technology (ESMT) in Berlin.

Christoph Schwarzer

Co-founder and CEO of EpiBlok Therapeutics, Berlin

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Professor for Translational Epilepsy Research at the Institute of Pharmacology at the Medical University of Innsbruck, Austria is cofounder and CSO of EpiBlok Therapeutics. He looks back to 30+ years of research on the role of neuropeptides in epilepsy starting with Günter Sperk in Innsbruck, then as senior research fellow at the Garvan Institute of Medical Research in Sydney, Australia. As Full Professor and Deputy Director of the Institute of Pharmacology at the Medical University of Innsbruck he heads the Neurobiochemistry research group. He is an acknowledged expert on the endogenous opioid system and its role in modulating epileptogenesis leading to over 100 highly cited publications and several patent applications.

Prof. Schwarzer studied microbiology and biochemistry at the University of Innsbruck and obtained his habilitation in neurobiochemistry from the Medical University of Innsbruck.