Prilenia is a clinical stage biotech company, led by Michael Hayden, MD, PhD., who is a world-renowned scientist in Huntington Disease research and the former President of Global R&D and Chief Scientific Officer at Teva Pharmaceuticals. The company’s lead asset is a highly selective sigma-1 receptor (S1R) which has shown to maintain functional capacity in early HD patients, as measured by Total Functional Capacity (TFC). Furthermore, it was recently selected from an international competition of over 30 potential therapeutics for inclusion in the first ever ALS platform trial, led by the Healey Center for ALS at Massachusetts General Hospital. Pridopidine is currently in late-stage clinical development for HD (Phase III) and ALS (Phase II). www.prilenia.com INVESTMENT IN TARGET: SERIES A OTHER INVESTORS: Forbion, Morningside Ventures, Sectoral Asset Management, Talisman Capital Partners PRODUCT: Small molecule (sigma-1 receptor (S1R) PROGRAMS: Huntington’s Disease, ALS PHASE: PHASE III
Scientific Advisory Board
Based on our Organs-on-Chips technology, we have created a Human Emulation System that provides a high-fidelity window into the inner workings of human biology and disease. The platform offers researchers a new standard for predicting how a human may respond to medicines, chemicals, and foods — with greater precision and control than today’s cell culture or animal-based testing methods. www.emulatebio.com INVESTMENT IN TARGET: SERIES C OTHER INVESTORS: FOUNDERS FUND PRODUCT: ‘ORGAN-ON-A-CHIP’ PLATFORMTECHNOLOGY (GROWING CELLS ON A DEVICE) FOR PRE CLINICAL DEVELOPMENT PROGRAMS: +10 PROGRAMS AMONG WHICH LUNG-, KIDNEY-, LIVER-, AND BRAIN MODELS. A COMBINATION OF MODELS IS RELEVANT FOR ALS PHASE: MARKET ROLL-OUT
Verge was founded by a unique combination of the field’s top machine learning experts and seasoned neuroscience drug developers. They share the vision that exponential advances in computational genomics combined with new insights into neuroscience has created a breakthrough opportunity to discover drugs that dramatically improve the lives of patients suffering from neurodegenerative disease. www.vergegenomics.com
INVESTMENT IN TARGET: SERIES A OTHER INVESTORS: DRAPER FISHER JURVETSON, WUXI PRODUCT: COMBINING ROBUST DATA PLATFORM AND DEEP NEUROBIOLOGY EXPERTISE TO DEVELOP NOVEL THERAPIES PROGRAMS: LEAD PROGRAMS ALS AND PARKINSONS’S, CONTRACT WORK IN OTHER INDICATIONS WITH PARTNERS PHASE: CLINICAL
Orphazyme was founded in 2009 with the objective to develop new therapies for patients suffering from protein-misfolding diseases with no or limited treatment options available.The company was based on a scientific discovery on the function of Heat-Shock Proteins (HSPs) by Thomas Kirkegaard Jensen and Professor Marja Jäättelä that was published in the scientific journal Nature. Since inception, Orphazyme has translated the scientific discovery into a late-stage clinical development program. Protein misfolding is the hallmark of a broad range of diseases and Orphazyme’s strategy is to use its expertise, including proprietary know-how to select and develop new leads for suitable diseases, hence the development of a proprietary suite of New Molecular Entities (NMEs) with improved characteristics. Orphazyme intends to select diseases suitable for the NMEs based on genetic and mechanistic insights into selected protein-misfolding diseases. www.orphazyme.com INVESTMENT IN TARGET: SERIES B OTHER INVESTORS: LSP, NOVO, SUNSTONE, KURMA PARTNERS, AESCAP PRODUCT: SMALL MOLECULE THERAPY PROGRAMS: LEAD PROGRAMS: ALS, NIEMANN PICK, SIBM SEOCNARY:GAUCHER AND PARKINSON’S PHASE: PHASE II (POTENTIALLY PIVOTAL)
Apic Bio Inc. (”Apic”) is a Boston based gene therapy company with a clinical stage program and a new, ground breaking technology platform. Whereas gene therapies normally consist of single vectors that stop or repair genes, Apic’s new platform uses two vectors simultaneously that stop and repair genes. The company has spun-out of University of Massachusetts Medical School which is one of the scientific leading institutes in gene therapy. The scientific leaders, Terry Flottes, Christian Mueller and Bob Brown are world renown in their field. Christian was involved in founding the company with John Reilly who is CEO. The company focuses on two indications, being ALS and Alpha-1 Antitrypsin Deficiency (AATD). In ALS the company targets two gene mutations. INVESTMENT IN TARGET: SERIES A OTHER INVESTORS: MORNINGSIDE VENTURES PRODUCT: GENE THERAPY WITH DUAL VECTOR APPROACH PROGRAMS: ALS MUTATIONS S0D1 AND C9, AND ALPHA 1 ANTI-TRIPSIN DEFICIENCY PHASE: PHASE I
www.amylyx.com INVESTMENT IN TARGET: SERIES A OTHER INVESTORS: MORNINGSIDE VENTURES, HENRI TERMEER PRODUCT: COMBINING TWO REPURPOSED SMALL MLECULES INTO ONE THERAPY PROGRAMS: COMBINATION OF 2 EXISTING DRUGS ALS; ALZHEIMER’S, FRIEDREICH’S ATAXIA PHASE: ON MARKET USA & CANADA
Amylyx is developing a proprietary therapeutic for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases that targets the neuroinflammation and nerve cell death that characterize these disorders. The company’s therapeutic, AMX0035, is a proprietary combination of existing compounds that Amylyx has demonstrated have synergistic effects in preventing nerve cell death and neurotoxic inflammation in multiple preclinical models. Outcome of a Phase II trial for ALS is expected in H2 2019.
Scientific Advisory Board
Scientific Advisory Board
Scientific Advisory Board
Scientific Advisory Board
Advisory Board
Advisory Board
Advisory Board
ALS Investment Fund
ALS Investment Fund
ALS Investment Fund