on 27th, during Rising Biotech Stars Session (from 14.00 to 16.00)
Retinitis Pigmentosa (RP), Leber’s Congenital Amaurosis (LCA) and Stargardt’s Disease (STGD) are rare eye diseases leading to severe vision impairment or blindness. Within the eye, they cause degeneration of the photoreceptors responsible for conversion of light into signals to be sent to the brain. The conditions are inherited and caused by ~250 different gene mutations. They affect a combined 450,000 people within the EU, Japan and the US. To date, no treatment is available for RP, LCA and STGD. Mireca wants to develop drug candidate LP–DF 003 which uses a novel approach for the treatment of the conditions. In contrast to current gene therapies, which address specific gene mutations, Mireca’s approach is significantly broader and assumed suitable to target at least one third of the various gene mutations. Based on the promising outcomes of the EU (FP7) funded DRUGSFORD project, Mireca benefits from completed pre-clinical pharmacology and initial safety testing, an existing orphan drug designation granted by the EMA, a GMP-compliant production process, 3 patent applications and exclusive licensing agreements. Beyond currently planned product developments, Mireca has access to a platform technology for the potential treatment of several neurodegenerative diseases. Mireca has been set up as a German limited liability company (“GmbH”) located in Tuebingen (~40 kilometers south of DE-Stuttgart). Mireca’s experienced international management team combines broad and deep scientific as well as business experience. Mireca wants to transition LP-DF003 into clinical studies and aims to obtain market authorizations for RP, LCA and STGD treatment products starting in the year 2024.
Musli Thyropeutics is a clinical stage pharmaceutical company addressing high-grade cancers. The company's patented fix-dose combination is based on recent scientific findings. Musli is targeting Glioblastoma Multiforme and Brain Metastases as-well-as Lung cancer and other aggressive high-grade cancers. Musli is preparing for Phase 2/3 clinical trials starting in 2018. Our innovative adjunct approach to current standard of care is expected to facilitate quick penetration to some of the largest markets in oncology, composing a multi-billion opportunity.