Das Projekt beinhaltet die Entwicklung von personalisierten Therapien für Betroffene mit seltenen NME, bei denen bisher Behandlungsmöglichkeiten fehlen. Zu den Patient:innen gehören auch Kinder und junge Erwachsene, die bislang keine Aussicht auf Heilung oder zumindest auf Linderung der Erkrankung hatten. Für sie sollen neue therapeutische Ansätze entwickelt werden, die im Sinne eines “Bench-to-Bedside”-Prinzips (aus dem Labor an das Krankenbett) direkt in individuelle Heilversuche überführt werden können. Dabei kommen sogenannte Omics-Technologien sowie Bioinformatik inkl. Künstliche Intelligenz (KI) zum Einsatz, um therapeutisch relevante Krankheitsmechanismen zu identifizieren und in die klinische Anwendung zu überführen.

Ausgangspunkt für diesen Ansatz sind die Patient:innenauswahl, die Analyse von  Hautbiopsien und die nachfolgende Phänotypisierung (Klassifizierung des Erscheinungsbildes) der gewonnenen Hautzellen (genauer: Fibroblasten). Diese Phänotypisierung, die von Wissenschaftler:innen in Düsseldorf, Bochum und Essen durchgeführt wird, wird zum Verständnis der zugrunde liegenden Eigenschaften dieser Zellen führen. Da Hautzellen dieselben genetischen Informationen wie Muskelzellen tragen, lassen sich ihre krankheitsverursachenden Veränderungen mit Omics-

Technologien untersuchen. Omics bezeichnet die gesamtheitliche Charakterisierung unter anderem aller Gene, Proteine oder Metabolite (Stoffwechselprodukte). Omics-Daten liefern einen wichtigen Ansatzpunkt in der personalisierten Medizin, da sie Aufschluss über individuelle Krankheitsvorgänge und mögliche Therapieansätze geben. Die Firma Singleron wird die zugrunde liegenden Genaktivitäten (sogenannte Transkriptomanalysen) durchführen, während ISAS-Forschende die entsprechenden Proteine und Stoffwechselprodukte auf Basis speziell entwickelter Massenspektrometrie-Verfahren analysieren werden.

 Suche nach bereits bekannten und unbekannten Arzneimittelwirkstoffen

Die gewonnenen Omics-Daten werden Bioinformatiker:innen am ISAS anschließend mithilfe von KI auswerten und mit Datenbanken zu vorhandenen Arzneimittelwirkstoffen abgleichen. Das Ziel dieser Analysen ist es, geeignete, bereits bekannte Wirkstoffe zu finden, mit denen sich die im ersten Schritt identifizierten fehlregulierten zellulären Prozesse bei NME adressieren lassen – und die damit für die Behandlung der Patient:innen infrage kommen können. Außerdem wird das Lead Discovery Center nach weiteren potenziell wirksamen Substanzen suchen. Sowohl die neuen, als auch bereits bekannten Wirkstoffe werden anschließend im Labor an den gewonnenen Fibroblasten getestet. In einem weiteren Schritt werden die Wissenschaftler:innen die Relevanz der fehlregulierten zellulären Prozesse in Nerven- und Muskelzellen prüfen. Erfolgversprechende Substanzen sollen danach in den Kliniken direkt für eine personalisierte Behandlung in individuellen Heilversuchen in den Patient:innen eingesetzt werden.

Insgesamt hat das Konsortium aus Kliniker:innen, Grundlagenwissenschaftler:innen und Bioinformatiker:innen bei diesem Forschungsprojekt sowohl den medizinischen, als auch den gesellschaftlichen Nutzen im Fokus. Eine verbesserte Lebensqualität der NME-Patient:innen durch neue personalisierte Therapien wirkt sich nicht nur positiv auf die einzelnen Betroffenen, sondern auch auf ihre Familien und das gesamte gesellschaftliche Umfeld aus.

Übersicht der Projektpartner

Unter der Leitung von Prof. Dr. Tobias Ruck von der Universitätsmedizin Düsseldorf vereint das Konsortium führende medizinische Forschungsinstitutionen und Industrieunternehmen. Dieser synergistische neuartige Ansatz ermöglicht innovative Therapiestrategien zur schnelleren Diagnostik und Behandlung von NME. Zentral für diese Ziele ist eine intensive Zusammenarbeit der Projektpartner:innen:

• Universitätsklinikum Düsseldorf, Klinik für Neurologie
• Universitätsmedizin Essen, Abteilung für Neuropädiatrie
• BG Universitätsklinikum Bergmannsheil Bochum
• Leibniz-Institut für Analytische Wissenschaften – ISAS e.V.
• Lead Discovery Center GmbH (LDC), Dortmund
• Singleron Biotechnologies GmbH, Köln

Weitere Informationen: b2b-rare.de

###

Über die Klinik für Neurologie des Universitätsklinikums Düsseldorf

In der Klinik für Neurologie des Universitätsklinikums Düsseldorf (Klinikleitung: Prof. Dr. med. Dr. rer. nat. Sven Meuth; Stellvertretung: Prof. Dr. med. Tobias Ruck) wird das gesamte Spektrum neurologischer Erkrankungen bei Erwachsenen ab 18 Jahren diagnostiziert und behandelt. Insgesamt werden in der Klinik für Neurologie jährlich ca. 15000 Patient:innen stationär und ambulant behandelt (ca. 2000-2500 Patient:innen davon mit neuromuskulären Krankheitsbildern). Die Klinik ist Teil des Neuromuskulären Zentrums Nordrhein. Zudem ist die Klinik für Neurologie ein zertifiziertes Myasthenie-Zentrum. In der Klinik wird das Konzept einer engen Vernetzung von Patient:innenversorgung, klinischer Forschung und Grundlagenwissenschaft verfolgt. Das Team möchte, dadurch die Ursachen neurologischer Erkrankungen besser verstehen und neue Therapiemöglichkeiten entwickeln. Die intensive Einbindung in internationale Studien gibt die Möglichkeit, Patient:innen   Zugang zu neuesten innovativen Therapien zu verschaffen.

Weitere Informationen: www.uniklinik-duesseldorf.de

Über die Neurologische Klinik und Poliklinik des BG-Universitätsklinikums Bergmannsheil

Die Neurologische Klinik und Poliklinik des BG-Universitätsklinikums Bergmannsheil (Interimsleitung Prof. Dr. med. Matthias Vorgerd) befasst sich neben der klinischen Versorgung sämtlicher neurologischer Erkrankungen von Patient:innen ab einem Alter von 18 Jahren mit einem breiten Spektrum klinischer und grundlagenwissenschaftlicher Forschung. Die Klinik ist Teil des Muskelzentrums Ruhrgebiet und von der Deutschen Gesellschaft für Muskelkranke e.V. (DGM) zertifiziertes Neuromuskuläres Zentrum. Sie ist an verschiedenen nationalen und internationalen Studien beteiligt. Zudem verfügt die Klinik über ein eigenes neuromuskuläres Labor, wo diagnostische und wissenschaftliche Untersuchungen am entnommenen Skelettmuskel, Nerven und Haut durchgeführt werden.  

Ein besonderer Schwerpunkt liegt auf der Erforschung von neuromuskulären Erkrankungen (NME), die sich vor allem auf die Untersuchung von Muskel-, Nerven- und Hautbiopsien stützt. Die Forschungsaktivitäten auf dem Gebiet der NME werden durch das Heimer Institut für Muskelforschung gefördert, welches in die Organisationstruktur der Neurologischen Klinik eingeordnet ist. Diese enge Vernetzung von klinischer Arbeit, spezialisierter Labordiagnostik und grundlagenwissenschaftlicher Forschung soll zur Entwicklung neuer Behandlungsmöglichkeiten beitragen.

Über die Abteilung für Neuropädiatrie der Universitätsmedizin Essen AöR

Die Abteilung für Neuropädiatrie beschäftigt sich neben der routinediagnostischen Analyse der Muskulatur von Patient:innen auch mit der Erforschung der molekularen Ursachen der jeweiligen Krankheitsbilder. Dazu zählen die Identifikation neuer Gene, sowie die Analyse der biochemischen Ursachen neurodegenerativer und muskulärer Erkrankungen. Die Forschungsaktivitäten des neuromuskulären Labors werden hierbei durch Herrn PD Dr. rer. Nat. Andreas Roos (Adjunct Professor der University of Ottawa) und Frau Prof. Dr. med. Ulrike Schara-Schmidt geleitet. Forschungsinteressen des Muskellabors liegen auf der Identifikation von Pathomechanismen und Biomarkern für neuromuskuläre Erkrankungen mit einem ausgesprochenen Fokus auf die Nutzung von Biomaterial. Dabei steht auch die Nutzung von Biomaterialien, welche minimal-invasive gewonnen werden können, im Fokus.

Im Rahmen von translationalen Prozessen ist das neuromuskuläre Labor Bestandteil zahlreicher nationaler und internationaler Studien (clinicaltrials.gov) sowie an übergreifenden nationalen und internationalen Studien zur Genotyp-Phänotyp Korrelationen beteiligt.

Über das Leibniz-Institut für Analytische Wissenschaften – ISAS e.V. –

Das Leibniz-Institut für Analytische Wissenschaften – ISAS – e.V. entwickelt leistungsfähige Analyseverfahren für die Gesundheitsforschung. Mit seinen Innovationen trägt es dazu bei, die Prävention, Frühdiagnose und Therapie von Erkrankungen zu verbessern. Ziel des Instituts ist es, die personalisierte Therapie voranzutreiben. Dafür kombiniert das ISAS das Wissen aus Chemie, Biologie, Medizin, Pharmakologie, Physik und Informatik. Das Institut kooperiert mit nationalen und internationalen Partnern aus der Wissenschaft und Industrie.

Weitere Informationen: www.isas.de

Über die Singleron Biotechnologies GmbH

Singleron Biotechnologies fördert die Präzisionsmedizin und die menschliche Gesundheit durch wegweisende Lösungen zur Analyse von Einzelzell-Multi-Omics. Das aktuelle Produktportfolio umfasst Hochdurchsatzgeräte für die automatisierte Verarbeitung von Einzelzellen und Gewebedissoziation, Reagenzien, Bioinformatik-Software sowie eine umfassende Wissensdatenbank für Einzelzellen.

Gegründet im Jahr 2018, ist Singleron weltweit tätig und verfügt über Büros, Labore und Produktionsstätten in Deutschland, Singapur, China und den USA. Die Produkte des Unternehmens werden in über 3000 Laboren in Krankenhäusern, Forschungsinstituten und Pharmaunternehmen in mehr als 20 Ländern eingesetzt.

Weitere Informationen: www.singleron.bio. Folgen Sie uns auf LinkedIn für aktuelle Updates.

Über die Lead Discovery Center GmbH

Die Lead Discovery Center GmbH wurde 2008 von der Technologietransfer-Organisation Max-Planck-Innovation gegründet, um das Potenzial exzellenter Grundlagenforschung für die Entwicklung neuer, dringend benötigter Medikamente besser zu nutzen. LDC nimmt vielversprechende Projekte aus der akademischen Forschung auf und entwickelt sie typischerweise weiter bis zu pharmazeutischen Leitstrukturen (Proof-of-Concept in Modellsystemen) oder bis zu präklinischen Kandidaten. In enger Zusammenarbeit mit führenden Partnern aus der akademischen Forschung und Industrie entwickelt LDC ein umfangreiches Portfolio an Projekten im Bereich niedermolekularer Wirkstoffe sowie therapeutische Antikörper mit außergewöhnlich hohem medizinischem und kommerziellem Potenzial.

LDC unterhält eine enge Partnerschaft mit der Max-Planck-Gesellschaft und dem KHAN Technology Transfer Funds I + II (KHAN-I, -II). Es hat weltweit zahlreiche Kooperationen mit verschiedenen Organisationen geschlossen, u.a. AstraZeneca, Bayer, Boehringer Ingelheim, Merck KGaA, Qurient, invIOs, Cumulus Oncology, Nodus Oncology, JT Pharmaceuticals, KinSea AS und den Helmholtz-Zentren, für Infektionsforschung (HZI) und dem Deutschen Krebsforschungszentrum (DKFZ). Das LDC arbeitet außerdem mit führenden akademischen Wirkstoffforschungszentren und Investoren zusammen, um Firmengründungen zu unterstützen.

Weitere Informationen: www.lead-discovery.de

less

Scope of Collaboration

Antibody-based inhibitors have gained  relevance in the field of drug discovery due to their exceptional affinity, selectivity and physicochemical properties. Within this collaboration, Icosagen will generate, identify, and discover potent antibodies against innovative and therapeutically relevant targets, supplying them to LDC for further development.

Utilizing its proprietary technology, Icosagen will produce the proteins of interest and enhance their display on the surface of virus-like particles, which serve as vehicles for the production of antibodies with affinity for the targets. Subsequently, an extensive library will be established to facilitate the selection of high-affinity monoclonal antibodies, which will undergo further analysis at LDC through a streamlined process of high-throughput screening. This partnership highlights Icosagen’s capability to scale its proprietary platforms and synergize effectively with LDC’s workflow.

“Partnering with LDC will further help expand the opportunities for antibody therapeutics, especially in the challenging field of GPCR-targeted treatments” said professor Mart Ustav, CEO of Icosagen. “This will not only highlight our technological expertise but also perfectly aligns with our mission to transform scientific discoveries into life-changing treatments.”

“We are excited to partner up with Icosagen, a competent and highly experienced player in the field of antibody research” remarked Bert Klebl, CEO and CSO of LDC. “This project will greatly benefit from Icosagen’s expertise and state-of-the-art capabilities as we pursue our shared goal: the development of novel therapeutics that can make a meaningful difference in healthcare.”

Future Prospects

LDC takes on promising early-stage projects from academia and transforms them into innovative pharmaceutical leads and antibodies that reach initial proof-of-concept in animals as well as candidate nomination. In close collaboration with high-profile partners from research and industry, LDC is building a strong and growing portfolio of small molecule and antibody leads with exceptional medical and commercial potential. The partnership between Icosagen and LDC holds promise in advancing targeted therapeutics, with the potential to revolutionize treatment approaches and improve outcomes for patients worldwide. The successful development of these monoclonal antibodies is anticipated to significantly advance the understanding and treatment of diseases.

###

About Icosagen

Icosagen is a renowned biotech company with over 20 years of experience in pioneering antibody research and production. With a strong commitment to innovation, Icosagen has developed a range of proprietary platforms for the efficient discovery, development and manufacturing of high-quality antibodies, serving the global research community and pharmaceutical industry.

Further information available at: www.icosagen.com

About LDC

Lead Discovery Center GmbH (LDC) was established in 2008 by the technology transfer organization Max Planck Innovation, as a novel approach to capitalize on the potential of excellent basic research for the discovery of new therapies for diseases with high medical need. LDC takes on promising early-stage projects from academia and transforms them into innovative pharmaceutical leads and antibodies that reach initial proof-of-concept in animals as well as candidate nomination. In close collaboration with high-profile partners from research and industry, LDC is building a strong and growing portfolio of small molecule and antibody leads with exceptional medical and commercial potential.

LDC sustains a long-term partnership with the Max Planck Society and its institutes as well as with KHAN-I, and has formed alliances with AstraZeneca, Bayer, Boehringer Ingelheim, Merck KGaA, Daiichi Sankyo, Qurient, InvIOS, Novo Nordisk, Cumulus Oncology, Nodus Oncology, JT Pharmaceuticals, KinSea Lead Discovery AS, HLB Pharma, the Helmholtz Center for Infection Research, e.g. In addition, LDC also works with leading translational drug discovery centers and with various investors to provide its assets for company creation.

Further information available at: www.lead-discovery.de

###

Contact:

Lead Discovery Center GmbH
Otto-Hahn-Straße 15
44227 Dortmund, Germany
pr@lead-discovery.de
www.lead-discovery.de

###

less

Tromsø, Norway, and Dortmund, Germany – April 25th, 2024. KinSea Lead Discovery AS, a biopharmaceutical start-up pioneering the use of marine bioactives for the treatment of human diseases, announces the successful first closing of its seed financing round. It includes an equity investment from KHAN Technology Transfer Fund I GmbH & Co KG (KHAN-I), an early-stage life sciences venture fund based in Germany, and welcomes the new investor Berners AS, a North Norwegian investment company. A year ago, KinSea had already secured a convertible loan from KHAN-I, which was recently converted into shares.

The financing enables the company to further develop its lead program, a FLT3 kinase inhibitor based on unique chemistry from marine sources, towards preclinical and clinical testing. Data from in vivo proof-of-concept studies suggest superior properties over existing FLT3 inhibitors, including potential broad activity against known drug-induced and drug-resistant FLT3 mutations, improved selectivity, and outstanding in vivo potency. The program originates from the successful collaboration of the founding partners, Arctic University of Norway (UiT), University of Bergen (UiB), Norinnova, and Lead Discovery Center GmbH (LDC).

‘We are grateful for the continued confidence and support from KHAN-I, and delighted to welcome Berners AS on board’, says Jeanette Hammer Andersen, CEO of KinSea. ‘This first closing validates the transformative potential of our approach. We are very committed to take our FLT3 inhibitors through the next stages of drug discovery and development in order to provide entirely new treatment options for AML patients that are safer and more effective’.

KinSea also plans to gradually expand its drug discovery pipeline and establish a diversified portfolio of high-potential drug candidates based on novel chemical scaffolds from the Arctic Ocean for the treatment of cancer and other diseases.

‘We are excited to reaffirm our commitment to KinSea and its groundbreaking work in the field of marine-derived therapeutics. The team has made significant progress over the last year, and we are convinced that their unique approach and capable team will continue to drive the maturation and expansion of their pipeline, and eventually make a meaningful impact on patients’ lives, in particular with regard to urgently needed, improved therapies for AML patients’, comments Bert Klebl, Managing Director of KHAN-I.

Mats Sæverud, CEO of Berners AS, adds: ‘In KinSea, we found an ambitious startup company that wants to make an important impact by employing natural products from the Arctic Ocean. The KinSea team has bold visions, scalable solutions, excellent expertise and skills, and fits well with Berners´ ambitions.’

KinSea has already started discussions with further investors for a second and final closing.

###

Contact
Lead Discovery Center GmbH
Otto-Hahn-Straße 15
44227 Dortmund
Germany
Phone: +49 231 97 42 70 00
Mail: pr@lead-discovery.de

###

About KHAN-I

KHAN Technology Transfer Fund I GmbH & Co KG (KHAN-I) is an early-stage life sciences venture fund with €70 million under management. Our mission is to create value through cooperative drug development partnerships with academic innovators in Europe. KHAN-I focuses on first-in-class therapies for attractive markets with a high unmet medical need. The fund is managed by Khanu Management GmbH, an experienced team of professionals with proven track records in early-stage drug development and academic spin-offs as well as pharma licensing and partnering. KHAN-I received an investment from the European Investment Fund (EIF) with the support of InnovFin Equity, and with the financial backing of the European Union under Horizon 2020 Financial Instruments and the European Fund for Strategic Investments (“EFSI”) under the Investment Plan for Europe. KHANI is also supported by Austria Wirtschaftsservice GmbH (AWS with funds provided by the Austrian

Federal Ministry for Digital and Economic Affairs and the Austrian Foundation for Research, Technology, and Development), Max Planck Foundation, and Thyssen’sche Handelsgesellschaft mbH. In addition, KHAN-I sustains a preferred partnership with the Max-Planck Society (Max-Planck Gesellschaft e.V.).

Further information at www.khanu.de

About Berners AS

Berners AS is a newly established, privately owned investment company, based in Tromsø, Norway. Through investments and active ownership, Berners will contribute to the development of profitable and sustainable businesses, especially within the seafood and marine sector. We aim to be a supporter of the development of brands and competence clusters in Northern Norway. Our investment strategy is based on our knowledge and love for the coast and the sea, and the opportunities that exist there. Berners is owned by Triko AS (80%) and Larren Invest AS (20%).

About Norinnova

Norinnova is one of Northern Norway’s most competent and experienced agencies for research commercialization. Norinnova connects researchers, start-up environments, companies and commercial actors to develop and utilize the region’s innovation power. For more than 30 years, Norinnova has worked closely with researchers and leading research communities in Northern Norway to harness the power of innovation in this region. This collaboration has contributed to the creation of brand-new businesses and has reinforced existing companies through new products and services. Norinnova secures rights, helps provide funding, investigates market potential, finds relevant partners, and contributes so that the scientists can get their product or service to the market.

Further information available at: www.norinnova.no

About LDC

Lead Discovery Center GmbH (LDC) was established in 2008 by the technology transfer organization Max Planck Innovation, as a novel approach to capitalize on the potential of excellent basic research for the discovery of new therapies for diseases with high medical need. LDC takes on promising early-stage projects from academia and transforms them into innovative pharmaceutical leads and antibodies that reach initial proof-of-concept in animals as well as candidate nomination. In close collaboration with high-profile partners from research and industry, LDC is building a strong and growing portfolio of small molecule and antibody leads with exceptional medical and commercial potential.

LDC sustains a long-term partnership with the Max Planck Society and its institutes as well as with KHAN-I, and has formed alliances with AstraZeneca, Bayer, Boehringer Ingelheim, Merck KGaA, Daiichi Sankyo, Qurient, InvIOS, Novo Nordisk, Cumulus Oncology, Nodus Oncology, JT Pharmaceuticals, KinSea Lead Discovery AS, HLB Pharma, the Helmholtz Center for Infection Research, e.g. In addition, LDC also works with leading translational drug discovery centers and with various investors to provide its assets for company creation.

Further information available at: www.lead-discovery.de

less

Dortmund, October 12, 2016 – Lead Discovery Center GmbH (LDC) and the biotech company UGISense AG are about to initiate a collaboration to jointly develop innovative drugs using the proprietary Ugimeres^TM antisense platform. Additionally, LDC will acquire a minority stake in UGISense AG during a first 1.2 million Euros financing round involving several private investors. The partnership aims at combining the potential of the innovative Ugimeres antisense platform with LDC’s drug discovery expertise and target know-how to maximize synergies and jointly commercialize successful projects.

“We see the Ugimeres platform as a highly exciting addition to our own in-house drug discovery technologies. We believe that the drug class of peptide nucleic acids (PNAs) offers enormous potential especially in the innovative field of microRNAs and for so far ‘undruggable’ targets”, says Dr. Bert Klebl, CEO of the LDC. “We look forward to working closely with the UGISense team and to jointly advance our first pilot projects”.

“We are very excited about the successful financing of UGISense AG and welcome our new partner LDC. This partnership will allow us to focus closely on the high-quality development and commercialization of Ugimeres as innovative drugs for targets with high unmet medical need”, says Dr. Thomas Lindhorst of UGISense AG’s executive board.

About UGISense AG

UGISense AG is a biotech company dedicated to developing new and innovative antisense drugs in collaboration with partners from the industry and academia. The developments are made on the basis of a proprietary platform technology, i.e. the Ugimeres^TM. The company, which was first established in 2016, is being financed by private investors and has been accredited by the Federal Office of Economics and Export Control (BAFA) (within the scope of their Venture Capital Grants program).

About Ugimeres^TM

Ugimeres^TM are short oligonucleotide sequences capable of interacting with single-stranded DNA or RNA by forming a double strand. Thanks to said double-strand formation, disease-relevant proteins will be specifically prevented from forming altogether or will be reduced to only a modified stage. This makes it possible to therapeutically influence the development of diseases at a very early stage of intervention. Structurally, Ugimeres^TM are derived from peptide nucleic acids (PNAs) to which important pharmacological functions have been added by way of chemical modifications.

For more details please go to: www.ugisense.com

Contact

Dr. Thomas Lindhorst
T. +43 (0)664 9123146
E. thomas.lindhorst@ugisense.com

UGISense AG
c/o Nordwind Capital GmbH
Residenzstrasse 18
80333 Munich

less

Dortmund, June 7, 2016 – Researchers from the Lead Discovery Center (LDC) in Dortmund, Germany led by Dr. Bert Klebl and McGill University in Montréal, Canada led by Dr. David Y. Thomas will work closely together to identify novel tools for protein trafficking and folding diseases drug discovery.The 3-years research initiative between LDCand McGill University will be funded for a total of $1M.

The project is the first to be funded under the Canada/Germany Program, a joint collaboration between the German Federal Ministry for Economic Affairs and Energy (BMWi), through its ZIM Program,and the CQDM consortium in Canada.Falling under the broader Canada/Europe initiative, the Canada/Germany Program aims to support novel and potentially transformative next‐generation technologies with the potential to improve, enhance or accelerate the state of the art and drug development process.

“We are proud of the fruits that bear our partnership with BMWi. The creative and innovative aspects of this project are the reasons we fulfill our missions and organize international funding programs,” said Diane Gosselin, President and CEO at CQDM. “This public‐private partnership is willing to develop new expertise and to expand into new markets, and these collaborative efforts will certainly strengthen research in Canada and Germany.”

“This project is the outcome of a great collaboration with LDC scientists on an important research topic. The project aims at addressing a need in biopharmaceutical research that is still understudied and unmet. ER stress is implicated in many diseases and Dr. Klebl and I will work at developing new tools for its study thanks to the funding initiative of CQDM and ZIM,” said Dr. David Y. Thomas at McGill University.

“We are very excited to embark on this international collaboration together with Dr. Thomas from McGill University. It truly is a team effort that draws its strength from our complementary expertise and deep insights into the biology,”said Dr. Bert Klebl, CEO at LDC. “This public private partnership pulls together multidisciplinary scientific resources and may lead to the discovery of new targets, a better understanding of off targets of known drugs and eventually to the identification of novel drug candidates for protein trafficking diseases.”

About the Project: Protein trafficking and misfolding under scrutiny

One third of the proteins encoded in our DNA transit through the endoplasmic reticulum (ER) inside cells. The ER has several distinct mechanisms to check the integrity and proper folding of these proteins. Some protein trafficking respiratory diseases result from an overzealous quality control system that recognizes mutant proteins that are otherwise functional and tags them for degradation before they reach their correct location. This complex quality control system is achieved through a network of interacting enzymes called kinases. ER quality control is an understudied field of biology and could represent a goldmine of new targets to treat many diseases including neurodegenerative disorders, diabetes and cancer. We have shown that inhibitors of some of these kinases affect the accuracy of the ER quality control system and are able to correct trafficking defects responsible in some diseases. The aim of this project is to generate a platform to study the most important players responsible for the proper trafficking of proteins. This collaborative project will leverage the expertise of each researcher to generate a set of validated tools and chemical probes to interrogate the protein kinases involved in protein trafficking which could allow to identify new targets for the development of novel ER related diseases therapies.

About CQDM

CQDM is a pharma-based consortium active in early research whose mission is to fund the development of innovative tools and technologies to accelerate drug discovery. Unique in the world, CQDM’s business model is based on a collaborative approach where all stakeholders share the costs of biopharmaceutical research and benefit from its results. CQDM also provides a common meeting ground where academia, governments, and the pharmaceutical and biotechnology industries converge to address numerous complex medical challenges. CQDM receives financial support from Merck, Pfizer, AstraZeneca, Boehringer Ingelheim, GlaxoSmithKline, Eli Lilly Canada, Janssen, Novartis Pharma Canada, Sanofi Canada, as well as from Quebec’s Ministry of Economy, Science and Innovation (MESI) and from the Government of Canada under the Business-Led Networks of Centres of Excellence Program (BL-NCE).

For more information:www.cqdm.org

less

Dortmund, May 11, 2016 – The Lead Discovery Center GmbH (LDC), Max Planck Innovation GmbH and Boehringer Ingelheim International GmbH have signed an agreement providing Boehringer Ingelheim with the option to receive the exclusive rights to a new lead compound for the treatment of schizophrenia to be discovered and developed at the LDC.

The novel approach builds on ground-breaking research results from Prof. Moritz Rossner and his team at the Max Planck Institute of Experimental Medicine in Göttingen. He will work closely together with the LDC team to identify and optimize novel compounds with strong therapeutic potential and develop it further to the stage of a validated pharmaceutical lead with in vivo efficacy. Moritz Rossner holds also a Professorship at Ludwig Maximilians University, Munich and is a co-founder of Systasy Bioscience GmbH.

Schizophrenia is a chronic, severe and disabling mental disorder ranked among the 12 most debilitating diseases by the World Health Organization. It affects about 1 in 100 people worldwide, changing the way they behave, think and perceive the world. Although anti-psychotic medications and psychosocial interventions can effectively reduce symptoms and improve patients’ lives, there remainsa strong need for new drugs truly addressing causative mechanisms and cognitive impairment.

Schizophrenia is a severe mental disorder

which affects how a person behaves,

thinks and perceives the world.

“Schizophrenia is an incredibly complex disorder which dramatically changes the life of the individual affected,” says Prof. Rossner. “We believe our approach holds strong potential to improve the treatment options for patients, and this collaboration with the LDC and Boehringer Ingelheim is a great opportunity to advance it from our laboratory into pharmaceutical development.”

In this early discovery project Boehringer Ingelheim will take a seat on the project development team and will pay an option fee. In addition, the company will allocate internal resources to the programand support collaborating partners to strengthen the early development work. Once the project has attained proof-of-concept in relevant invivo models Boehringer Ingelheim can exclusively license the lead at pre-defined terms for subsequent preclinical and clinical development. Any revenue the LDC may receive from commercialization will be shared with the academic inventors and collaborating institutions.

“Since the LDC’s inception, we have been in close contact with Boehringer Ingelheim, establishing a relationship based on mutual trust over the years. We very much appreciate their expertise and commitment to driving forward innovative therapeutic approaches,”says Dr. Bert Klebl, CEO of the LDC. “It is a great pleasure for us to be launching our first joint project in the field of mental disorders, which we believe is often underestimated. We see strong commercial opportunities here, and there is already a strong line-up of promising projects in this area from our academic network.”

About Max Planck Innovation
Max Planck Innovation is responsible for the technology transfer of the Max Planck Society and, as such, serves as a link between industry and basic research. With its interdisciplinary team it advises and supports scientists in evaluating their inventions, filing patents, and founding companies. Max Planck Innovation offers the industry unique access to the innovations of the Max Planck Institutes, and therefore performs an important task: the transfer of basic research results into products, which contributes to economic and social progress.

Further information at: www.max-planck-innovation.de

less

LDC and Roche will jointly advance innovative drug discovery projects

Dortmund, January 21, 2016 – The Lead Discovery Center GmbH (LDC) and Roche will collaborate to identify and leverage innovative therapeutic opportunities that address diseases of unmet medical need across several disease areas.

Projects will be sourced from the LDC’s academic partners, including leading universities and institutes from the Max Planck Society and the Helmholtz Association, as well as from Roche’s innovation network. The partners will work together to advance projects from as early as target level up to the identification of a preclinical candidate. Over an initial three year period, LDC will act as a translational incubator for Roche and carry out small molecule projects in close collaboration with the scientific inventors and their academic institutions.

“We are delighted to team up with Roche, the world’s largest biotech company,” says Dr Bert Klebl, CEO of the LDC. “We are impressed by their deep expertise across many disease areas and the perfect cultural fit with our team. We are definitely on the same wavelength regarding our approach to early drug discovery and share a mission to transform academic innovation into tangible benefits to patients.”

Upon attainment of a predefined milestone, Roche has option rights to an exclusive license. The terms and conditions of collaboration and licensing will be agreed on a project-by-project basis to ensure a fair balance of investments and potential returns between the partners. Any revenue the LDC may receive from a potential future commercialization will be shared with the academic inventors and collaborating institutions. Roche’s contributions will include research funding and possibly in-kind support required for optimal project progress, such as access to compound libraries.

less