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uniQure is a world-leading gene therapy company. Our lead product, Glybera, received a positive opinion from CHMP in July 2012 and is poised to become the first gene therapy approved in the Western World, capping more than 30 years research in this area.

This positive outcome validates uniQure's AAV platform and, most importantly, the only commercially scalable manufacturing system currently available anywhere.

uniQure's plug-and-play platform offers the opportunity to develop many additional therapies on a platform that is now validated and accepted as safe by regulatory authorities.

uniQure's second programme, for Hemophilia B ($1.5bn market) is in a Phase I/II clinical study and has shown up to 30 months stable, therapeutic expression from a single treatment (NEJM Dec 2011).

Over the coming 6 months further programmes for Porphyria, Parkinson's Disease and Sanfilippo B are expected to enter Phase I/II clinical trials.

uniQure is based in Amsterdam, and backed by Forbion, Gilde, Advent, CAPE and other investors. We would be interested in meeting to discuss potential collaborations and also potential investment opportunities.

ORCA Therapeutics B.V. (ORCA) is developing a pipeline of innovative anticancer therapies based on the highly promising approach of oncolytic viruses. ORCA’s value proposition is based on using its proprietary technologies in the field of Oncolytic Replication Competent Agents. ORCA’s lead product ORCA-010 is a new oncolytic adenovirus based on the Company’s proprietary T1 technology. ORCA-010 has up to ten thousand fold higher oncolytic potency as compared to current state of the art oncolytic adenoviruses in a variety of cancer cell lines and ORCA is currently developing ORCA-010 for its first study in man. 

uniQure is a world-leading gene therapy company. Our lead product, Glybera, received a positive opinion from CHMP in July 2012 and is poised to become the first gene therapy approved in the Western World, capping more than 30 years research in this area.

This positive outcome validates uniQure's AAV platform and, most importantly, the only commercially scalable manufacturing system currently available anywhere.

uniQure's plug-and-play platform offers the opportunity to develop many additional therapies on a platform that is now validated and accepted as safe by regulatory authorities.

uniQure's second programme, for Hemophilia B ($1.5bn market) is in a Phase I/II clinical study and has shown up to 30 months stable, therapeutic expression from a single treatment (NEJM Dec 2011).

Over the coming 6 months further programmes for Porphyria, Parkinson's Disease and Sanfilippo B are expected to enter Phase I/II clinical trials.

uniQure is based in Amsterdam, and backed by Forbion, Gilde, Advent, CAPE and other investors. We would be interested in meeting to discuss potential collaborations and also potential investment opportunities.

Corporate

• Ceronco Biosciences is developing new drugs to fight cancer based on the short-chain sphingolipid (SCS) technology discovered in a collaborative research project at the Netherlands Cancer Institute (NKI) and Erasmus MC.
• Proof of principle has been established in combining the SCS-technology with doxorubicin in highly relevant animal models. Addition of the SCS leads to increase in uptake of doxorubicin, specifically in cancer cells, and subsequently a significant reduction in tumor growth and increase in overall survival. At the same time no increase in severity of expected side effects or new side effects were noted.
• Ceronco is now taking this first product (CB001) into clinical trials, with expected start of the first clinical trial early 2013.

Technology

• A major barrier for uptake of chemotherapeutic drugs is the cellular membrane of a cancer cell. A new class of short-chain sphingolipids (SCS) has been identified which insert themselves into the cellular membrane and lead to catalysis of drug-membrane translocation. This results in increased uptake of a large variety of amphiphilic chemotherapeutic drugs.
• Tumor Cell Membrane Modulation using the SCS-technology is a novel and unique method to increase the efficacy of chemotherpaeutic drugs.

Products

• CB001: GluCer-enhanced liposomal doxorubicin
• CB002: SCS-enhanced mitoxantrone

InteRNA Technologies is a Dutch drug discovery and development company engaged in the development of novel breakthrough cancer therapies based on the unique functions of its proprietary miRNAs.

InteRNA well equipped to move quickly towards the clinic

InteRNA's drug development programs aim at developing miRNA-based therapeutics that control genes connected in signal transduction pathways and biological processes that are known to play a role in cancer initiation, progression and metastasis.

InteRNA identified the function of multiple miRNAs in a number of cancer indications through functional screens, using its proprietary lentiviral-based miRNA expression library, which is the largest currently available.

With the Company's proprietary miRNA expression database (generated through deep sequencing) and additional technologies (like RNAseq, PAR-CLIP, bioinformatics, in vivo models) in place to identify the downstream regulated proteins for each miRNA drug candidate, the Company is well equipped to move its novel drug candidates with their biomarkers quickly towards the clinic.