ALS Investment Fund

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 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

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 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

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

OTHER INVESTORS: MORNINGSIDE VENTURES PRODUCT: Gene Therapy with Dual Vector Approach PROGRAMS: Als Mutations S0D1 and C9, AND ALPHA 1 ANTI-TRIPSIN DEFICIENCY 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

• Chief, Neurology Service, Massachusetts General Hospital
• Julieanne Dorn Professor of Neurology, Harvard Medical School
• Director, Amyotrophic Lateral Sclerosis Clinic, Massachusetts General Hospital
• Co-Director, Neuromuscular Division, Massachusetts General Hospital
• Director Neurological Clinical Research Institute (NCRI)
• Founder and former co-directors of the Northeast ALS Consortium (NEALS)
• Former MD, Neurologist, MSc Neurochemistry, PhD Physiology and Biophysics