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PHARMALEX GMBH

Country: Germany

PHARMALEX GMBH

2 Projects, page 1 of 1
  • Funder: European Commission Project Code: 825925
    Overall Budget: 15,396,000 EURFunder Contribution: 14,959,800 EUR

    Low back pain (LBP) is a leading cause of disability and morbidity worldwide. It is widely accepted that a major contributor to LBP is intervertebral disc degeneration (IDD). IDD account for at least 40% (~280 million) of all LBP cases, leading to an EU-economic burden of ~€240 billion. These patients receive conservative treatment (e.g. pain relief medication and physiotherapy). When the latter is unsatisfactory, the only option left are invasive and costly surgical intervention. To date, no treatments halt or reverse IDD. Despite the profound socioeconomic burden and impact of IDD, decreasing the quality of life of millions of people, a game-changing treatment strategy for IDD-induced LBP is almost non-existent. The iPSpine consortium was formed to initiate a European-led research effort to identify a future advanced therapeutic strategy that results into a radical new treatment of IDD-induced LBP. With their multi-disciplinary expertise in the development of advanced therapies and their translation from bench to bedside, the aim of the iPSpine team is to investigate and develop a new advanced therapy medicinal product (ATMP) of the future, based on a novel developmental biology-based therapeutic strategy employing pluripotent stem cells (iPSC) and smart biomaterials. The iPSpine consortium will develop and demonstrate Proof-of-concept with the aid of novel and extended knowledge, tools and technology platforms. Hereby, iPSpine has the ambition to make a significant contribution by reducing translational bottlenecks through open innovation and take European leadership in the development of ATMPs. The iPSpine impact: iPSpine seeks to offer novel technologies and ATMPs for the advanced therapy research and development community. IDD will be the showcase, offering improved quality of life for millions of patients with IDD-induced LBP, through long-lasting reduction of LBP, reduced LBP-related premature retirement, and improved socio-economic contribution.

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  • Funder: European Commission Project Code: 101057777
    Overall Budget: 13,269,100 EURFunder Contribution: 13,268,000 EUR

    Lung cancer (LC) treatments have advanced in recent years with the advent of genetic profiling and immunotherapy. However, LC is a complex heterogeneous disease and survival rates remain poor. RNA (mRNA, microRNA, other non-coding RNAs and nucleic acid based modulators of same) and gene therapies (DNA or gene editing) for delivering nucleic acid-based therapeutics have curative potential for a host of indications previously untreatable. They have yet to enter the mainstream, due to safety concerns and difficulties delivering them efficiently to areas other than the liver, kidney and circulatory system. Aerosol delivery allows direct targeting of lung tissues but viscous mucus in the lung is a significant barrier to gene transfer to the target cells of the lungs. Even if the mucus layer can be penetrated, inefficient penetration through the cell membrane further impedes access of these vectors to the underlying target cells, thus preventing successful gene transfer. Delivery is a major barrier to successful pulmonary gene therapy for competing viral and non-viral gene transfer vectors and the vast promise of gene therapy has many challenges to overcome. OMNI's novel solution is pioneering the use of genetically modified MSC EVs with a proprietary surface engineering technology to further enhance delivery through the mucus barrier and into the targeted lung cells. This platform technology also combines efficient aerosol delivery of the EVs via AERO's proprietary state of the art vibrating mesh nebulizer technology. This unique solution solves the problems associated with lung targeted delivery of RNA based advanced therapies.

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