SPARK Success Stories

Prof. Dr. Simone Spuler, Dr. Verena Schöwel-Wolf, ECRC

MyoPax develops an innovative autologous muscle stem cell therapy to treat muscle wasting. The team’s technological innovation enables highly standardized manufacturing of pure, native and highly regenerative muscle stem cells from small human muscle tissue specimens to treat acquired and inherited muscle diseases. The team prepares to set up the startup MyoPax to clinically pursue the development of their approach to fight muscle diseases. They have successfully been awarded follow-on funding from the BMBF for their First-in-Human clinical trial, which starts in 2022. The team is currently raising their first investment round to establish the muscle stem cell platform MyoPax.

Project Summary | Website

Prof. Dr. Alessandro Prigione, Prof. Dr. Markus Schülke-Gerstenfeld, MDC & Charité

Leigh syndrome is a rare incurable mitochondrial disease affecting children where treatment options are lacking. The team has developed a novel assay to identify compounds for treating Leigh syndrome. Using this assay, a class of drugs applicable for repurposing that restore the cellular disease phenotype has been identified. The team has initiated a compassionate use treatment for a terminal ill patient. The patient has recovered significantly.

Project summary

Prof. Dr. Regine Heilbronn, Prof. Dr. Christoph Schwarzer, Charité & Medizinische Universität Innsbruck

The project aims at developing a gene therapy for the treatment of drug-resistant focal epilepsy. An adeno-associated viral (AAV) vector will be delivered minimally invasively to the epileptic focus, re-expressing a neuropeptide that will be released in an activity-dependent manner, i.e. in periods of high neuronal activity which precedes the onset of a seizure. Suppression of neuronal excitability thereby suppresses the epileptic event. The team was supported by GO-Bio funding with 3.9 Mio EUR, founded the startup EpiBlok Therapeutics GmbH and has acquired follow-up funding to further pursue the strategy and develop the gene therapy for the use in patients.

Project summary

PD Dr. Boris Schmitt, Charité

For babies born with a congenital heart valve defect there is no dedicated child valve on the market. Instead, they often receive xenogenic animal valves which degrade over the following years urging for risky open-heart re-surgery. The aim of the project is the production and testing of an anchoring mechanism of a personalized, autologous heart valve for children. This enables growth in a once-in-a-lifetime point-of-care minimally invasive implantation. The novel anchoring mechanism facilitates placement of the valve without hindering growth of valve and vessel.

Project summary | Website

Prof. Dr. Achim Kramer, Charité

The circadian clock is a biological program that structures physiology and behaviour according to the time of day. It is active in practically all cells of our bodies. The circadian clock is thus a cell-based program that is essential to health and well-being. The team around Professor Kramer has developed a new diagnostic tool to probe human internal time and rhythm and founded the startup Bodyclock Technologies in 2021.

Project summary | Website

Dr. Felix Lorenz, Dr. Julian Clauss, Dr. Inan Edes, Prof. Dr. Wolfgang Uckert, MDC

Immunotherapy currently holds the most potential for cancer treatment, with T-cell therapies as one promising approach. The team develops a high throughput platform to identify T cell receptors (TCRs) specific for cancer antigens for a novel and effective T-cell therapy for untreatable blood cancer patients. The team is setting up the startup Captain T-Cell and has acquired 4.9 Mio follow-on funding from the GO-Bio program to further pursue the strategy.

Project summary

Dr. Panagiotis Fikatas, Charité

Dr. Fikatas and his team have developed a device and method for the generation of extra corporally pre-tied surgical knots. The device consists of a yarn carrier with a pre-tied but still open knot ready to use during surgery. It is easy to use even by non-surgeons without special training. Knots produced are stronger and more stable than other sliding knots and tying is faster. The team founded the startup Clouz in early 2020.

Project Summary | Website

Prof. Dr. Alexander Meyer, Prof. Dr. Volkmar Falk Charité & DHZB

The large number of concurrent patient data in critical care units goes well beyond the capacity of the intensive care physician and may lead to treatment delays or clinical errors. The team applies deep machine learning methods in a critical care scenario to provide timely and highly accurate decision support to the clinical staff. They have developed a set of forward-facing real-time prediction models for severe post-cardiothoracic surgery complications. Primary focus is the prediction of postoperative bleeding. The team founded the startup x-cardiac and obtained CE certification for their first

Project summary | Website

Prof Dr. Peter Kühnen, Prof. Heike Biebermann, Charité

Obesity is an increasing problem with immense socioeconomic burden and severe suffering for the individual patients. The team has identified a novel intracellular pathway via
the Melanocortin-4 receptor which plays a pivotal role in weight regulation. When this signaling pathway is disturbed, the patients experience a constant hunger
feeling irrespective of how much they eat. The aim of this project is to identify patients that benefit from a MC4R agonist treatment resulting in a normal hunger feeling, and thus reducing the weight naturally. Prof. Peter Kühnen received the Paul Martini Award 2020 for studies on normalization of body weight in hereditarily obese patients. The prize is awarded annually by the Paul Martini Foundation for outstanding achievements in clinical therapeutic drug research.

Project summary