Cervical cancer (CxCa) is caused by high risk types of the human papillomavirus (a.o. HPV16). Prophylactic HPV vaccination is not the standard of care in all European countries, and where approved it is not sufficiently adopted. Once infected, these vaccines no longer prevent premalignant lesions and CxCa, which can only be treated with surgery or radio-chemotherapy. However, this is not effective in recurrent/advanced CxCa. In underdeveloped countries, CxCa is often detected when it is too late for curative treatment. With an estimated global incidence of 500.000 new cases of CxCa and 274.000 deaths per year, the need for an effective therapy is extremely high. Targeted immunotherapy is an effective approach to induce a tumour-directed immune response. Previously, members of the IMMUNISA consortium have formulated ISA101, a cancer vaccine consisting of overlapping synthetic long peptides covering all epitopes of the HPV16 oncogenic proteins. ISA101 has shown promising Phase 1 clinical trial results in patients with advanced CxCa where it works synergistically with chemotherapy. IMMUNISA now proposes the multi-centre randomised Phase 2 CervISA-2 trial; a careful assessment of the efficacy of the proprietary ISA101b therapeutic vaccine in combination with chemotherapy for the treatment of CxCa, measured by a prolonged progression free survival. A multidisciplinary collaboration between leading clinical EU sites specialised in CxCa, a partner providing strategic and operational regulatory service and a cutting-edge biotech SME will enable the optimal implementation of the CervISA-2 trial, exploitation activities and overall project dissemination. This is pivotal to catapult further development of ISA101b as a commercial vaccine and push it towards clinical implementation as fast as possible. For ISA101b, IMMUNISA will provide, 1) clinical data on the efficacy, 2) a solid exploitation strategy and 3) the regulatory framework for efficient translation to clinical use.

Hearing loss is a chronic non-communicable disease disabling over 328 million adults, and 32 million children worldwide. Sensorineural hearing loss due to loss of auditory hair cells was long thought to be irreversible. However, recent animal studies have demonstrated that pharmacological inhibition of cell signalling via Notch receptors using gamma-secretase inhibitors (GSIs) can regenerate hair cells and partially restore hearing capacity. This novel therapeutic concept provides the first promising lead for actual treatment of hearing loss. Clinical validation of these findings is the next crucial stepping stone in the development of a regenerative therapy for hearing loss. The ambition of REGAIN is to repurpose a GSI molecule for this indication by shifting from systemic to local treatment. The objective of the REGAIN project is to demonstrate and exploit the efficacy of locally administered GSIs to improve hearing through regeneration of inner ear hair cells with a lasting effect. The project will involve 1) the upscaling of GMP production of the clinical GSI candidate, 2) the generation of preclinical data on GSI dosing and local safety, 3) medical ethical clinical trial approval and 4) the demonstration of proof of concept for GSI for treatment of patients with recent onset sensorineural hearing loss. Small molecule drugs targeting the underlying biological causes of hearing loss in a safe way are expected to meet a real medical need for millions of patients, who currently rely on the limited benefits provided by hearing aids or cochlear implants. The partners involved in REGAIN represent the current state of the art in regenerative hearing loss research in the EU. REGAIN will break through that current state of the art, and will advance the first highly promising pharmaceutical treatment of hearing loss through clinical testing.

A recent evaluation of FP7 projects executed by the EC, showed that an estimated 80% of funded Health projects lacks valorisation. The “UTILE” proposal presents the EU-Health Innovation Marketplace, an online tool to better valorise the FP7 Health and Horizon2020 SC1 project results. This marketplace will actively bring together Innovation Providers (i.e. technology push) and the Innovation Developers (i.e. market pull). Moreover, in order to bring technology push and market pull successfully together, all 1200 FP7 Health and Horizon2020 SC1 projects will not only be analysed and defragmented by five high quality TTO’s (including NIH) in a LEAN process. It will also be valued by an elaborate Market & Stakeholder Advisory Committee, existing of Business Developers of 20-35 important pharmaceutical, medtech, biotech companies and Venture Capitalists. Thus here, valorisation of Health results will not be enhanced by just pushing harder, but by defragmenting and presenting these results in a way that the market will better recognize the added value. In addition to the presentation of well valued selected results on the online UTILE Marketplace, technology push and market pull will be actively brought together by a series of offline activities such as specific workshops and roundtables at international and conferences, brokerage events and technology valorisation courses and trainings. Special attention will be given to the participation of SME’s and involvement of Low Performing Countries. UTILE will deliver a self-sustainable platform of functionalities and activities will also enable the valorisation of future Horizon2020 SC1 projects as well as the many more health-related EC-funded health projects (e.g. in the Marie (Skłodowska) Curie actions). The UTILE Marketplace therefore will continue well after the current grant period.

Faecal incontinence (FI) is a common condition affecting ~67 million people in Europe, seriously impairing living and productivity of affected individuals and their families. Women with FI arising from childbirth injury may benefit from regenerative medicine using autologous skeletal muscle derived cells (ASMDC) to restore function of damaged sphincter muscle. However, progress is hindered by sub-optimal manufacturing and delivery techniques contributing to inconsistent results. AMELIE proposes an innovative approach that uses ASMDC attached to implantable microcarriers that will enable delivery of a higher number of viable ASMDC into the damaged sphincter muscle, increasing the likelihood of cell engraftment, regeneration of muscle and improved continence. To achieve this, AMELIE will develop bespoke implantable microcarriers suitable for clinical use; establish robust bioprocessing for manufacture of the cell-microcarrier combination; and for the first time, robustly test, in a randomised clinical trial, the principle that delivery of ASMDC in an anchored, natural state, provides more effective and consistent treatment. AMELIE comprises a highly interdisciplinary, gender balanced, consortium of internationally recognised experts and key-opinion leaders with relevant experience from academia and industry across Europe in the fields of engineering (IST, PS, UCL, NHSBT), biological sciences (NHSBT, UCL, IISFJD), and translational regenerative medicine (QMUL, MUG, AUH, AU, RUH, UHE, IISFJD, CVBF). BCR will lead dissemination activity and stakeholder interaction with patients and the public, healthcare professionals, industry and third party FI charities across Europe to establish reciprocal dialogue with people that will interact with the new technology throughout the lifetime of the AMELIE project. TTOP will lead knowledge management and exploitation to develop a marketing strategy and business plan to facilitate future exploitation of the technology to ensure patient benefit.

PRECIOUS aims at scaling-up biodegradable nanomedicines for multimodal precision cancer immunotherapy. With 3.5 million cancer patients in Europe every year, new cancer medicines are eagerly awaited, notably for prostate and ovarian cancer. Systemically applied new immunotherapies are promising, but their toxicity is a hurdle. Vaccination against cancer is safe but rather the efficacy can be disappointing, i.e. the microenvironment shuts down anti-tumour immunity. PRECIOUS will tackle 2 bottlenecks: 1. Production of non toxic multimodal nanomedicines, which induce vigorous immune responses, and at the same time reverse immunosuppression 2. Large scale GMP production of nanomedicines, and initiate a multimodal immunotherapy Phase I trial. We want to solve these bottlenecks by: Objective 1: Two types of GMP biodegradable nanoparticles: 1) a nanovaccine, containing tumour antigens and immune activators, and 2) a nanoparticle composed of compounds, which reverses the suppression and reactivates immunity in the tumour. Objective 2: Clinical Phase I trial to show efficacy. Nanomedicines will be used by 1) injection of vaccines to evoke immune responses and 2) injection of nanoparticles in the tumour microenvironment, which slowly release compounds that reverse suppression. To achieve this, a platform is formed with 6 leading industrial partners to produce large scale GMP nanomedicines and an excellent immunomodulator track record, together with 5 renowned academic partners to perform clinical studies. Relevance to call: Large scale GMP production, industrial leadership, nanotechnology and advanced manufacturing KET technologies, translation to the clinic, marketing development.