The tailored genome - How enzymes can help to cure hereditary diseases
The start-up project RecTech led by Prof. Frank Buchholz and Dr. Anne-Kristin Heninger has once again succeeded in scoring points in the prestigious Science4Life Venture Cup. This time, RecTech was awarded as one of the ten best teams for its innovative business concept. The participation is highly competitive and offers applicants access to more than 300 industry experts.
The research team of the TU Dresden has developed a promising business concept to establish a flourishing Biotech company in Dresden based on designer recombinase technology. The technology of genome editing applies molecular biological tools to specifically modify the genetic code of organisms. Based on genome editing tools, genetic diseases might be cured in the future by means of "molecular repair". The business idea of the RecTech project is to develop a recombinase platform technology.
Currently, more than 8,000 gene mutations are known to cause genetic diseases that are currently incurable. However, RecTech's designer recombinases enable the development of new curative therapies for such diseases. Sequence-specific recombinases (SSRs) are enzymes that can precisely edit and modify the genome at a specific target sequence. The RecTech team has developed innovative methods to adapt SSRs to any recognition sequence.
In the phase of the Science4Life business plan competition, 81 teams from all over Germany once again participated. This time, the ten best business concepts were awarded within the Venture Cup and the BMBF-GO-Bio-funded start-up RecTech from Dresden was again among them.
Start-up RecTech is one of the winners of the 2021 Science4Life Venture Cup first phase
Within the business plan competition of the 2021 Science4Life Venture Cup, 97 applicants presented their innovative business ideas and 5 teams were awarded with a prize for the best business idea. The BMBF-GO-Bio-funded start-up project RecTech, led by Prof. Dr. Frank Buchholz and Dr. Anne-Kristin Heninger, successfully convinced the reviewers with their idea to develop Designer-Recombinases as versatile genome editing tools for novel healing strategies of genetic diseases.
Science4Life e.V. is an independent founders' initiative, which was already founded in 1998 as a non-profit organization. Initiators and sponsors are the Hessian state government and the healthcare company Sanofi. Once a year, the initiative organizes the largest business plan competition in the fields of life sciences, chemicals and energy in Germany. The winning teams qualify for selected coaching workshops during the Academy Days (link).
The Dresden start-up project RecTech, led by Prof. Dr. Frank Buchholz and Dr. Anne-Kristin Heninger, is one of five start-up projects that received a prize for the best innovative business idea within the prestigious Science4Life Venture Cup (link). Frank Buchholz, Professor of Medical Systems Biology at the Medical Faculty of the TU Dresden and Anne-Kristin Heninger, leader of the RecTech project, are developing methods to correct or remove mutated sequences in the human genome. The introduced innovative genome editing technology is based on many years of research developing sequence-specific recombinases (SSRs) as an efficient and safe genome surgery tool. SSRs are enzymes that can precisely edit the genome of cells at a specific target sequence. In particular, these so-called Designer-Recombinases could be applied for the treatment of monogenetic diseases. Currently, more than 8,000 genetic modifications are known that lead to presently incurable diseases. RecTech's Designer-Recombinases will enable the development of new curative therapies for such diseases. Thus, the establishment of an innovative platform technology for the production of these tailor-made genome editing tools opens up a completely new market segment with promising commercial prospects in Dresden.
The team of Prof. Buchholz and Dr. Heninger has developed a promising business concept to establish a flourishing Biotech company in Dresden based on the recombinase technology. RecTech’s goal is to transfer the technology into broad clinical application in the near future.
Development of a powerful gene editing tool to correct a genomic inversion frequently found in Hemophilia A
Hemophilia A (HA) is the most commonly occurring inheritable deficiency of blood coagulation. Severely affected individuals suffer frequent spontaneous haemorrhaging, which can lead to chronic painful and debilitating arthropathies, hematomas, and potentially life-threatening internal bleeding. About 50% of severe HA cases are caused by large gene inversions between inverted repeats surrounding the F8 gene.
In an attempt to find novel ways to flawlessly revert these gene defects, The Buchholz laboratory has developed a recombinase-based system to correct a 140 kb genomic inversion of the F8 gene causing severe Hemophilia A. The team was able to reverse the defect in patient derived cells at high efficiency, indicating that this approach might be suitable to develop a curative therapy for patients carrying this genetic inversion.