Phoenix Nest is a collaborative company focused on treating Sanfilippo Syndrome (Mucopolysaccharidosis (MPS) IIIB, IIIC, and IIID).  Their goal is to bring these treatments to market as quickly as possible.  They are working with leading academics and have engaged in a  strategy of licensing key technologies to facilitate this goal.

Biotechnology

Phoenix Nest

Mari-Elaina Neufeld

Dr. Freundlich is an associate professor of Pharmacology & Physiology and Medicine at Rutgers University–New Jersey Medical School. He is a faculty member of the Center for Emerging and Reemerging Pathogens. Prior to his return to academic research in 2006, he spent eight years in the pharmaceutical industry as a medicinal chemist. His undergraduate and master’s degree training were in chemical engineering at Cornell University as a McMullen Dean’s Scholar. He received his doctorate in organic chemistry from the Massachusetts Institute of Technology under the tutelage of 2005 Nobel Prize in Chemistry awardee Richard Schrock. An overarching goal of his current research is the discovery of small molecules that modulate novel biological targets in Mycobacterium tuberculosis – the causative agent of tuberculosis. To accomplish their aims, his lab typically employs computational, chemical, and biological techniques. His laboratory is funded by the National Institutes of Health.

Joel Freundlich

Dr. Alexey Pshezhetsky graduated from the Department of Chemistry, Moscow State University in 1985. In 1989 he obtained Ph.D. degree in chemical kinetics and catalysis from the same university. In 1989-1992 he was a researcher in Moscow State University and Moscow Institute of Medical and Biological Chemistry studying genetic diseases of children caused by the inherited deficiencies of lysosomal enzymes. In 1993 Dr. Pshezhetsky joined Departments of Pediatrics and Biochemistry, University of Montreal where he was subsequently promoted to professor. He is also affiliated with the Department of Anatomy and Cell Biology at McGill University. Since 1998 he is a scientific director of Medical genetics diagnostic laboratory and a director of a Laboratory of lysosomal biology at Ste-Justine Hospital Research Center. The research of Dr. Pshezhetsky has been acknowledged by multiple national and international agencies including the Canadian Institutes of Health Research, Canadian Foundation for Innovation, Genome Canada, Genome Quebec, March of Dimes Foundation, Vaincre les Maladies Lysosomales and others. Dr. Pshezhetsky received many career awards and fellowships including a National Investigator Award from FRSQ and an Award of Excellence in Pediatric Research from the Foundation for the Research in Children’s Disorders. The current research interests of Dr. Pshezhetsky include molecular basis of lysosomal and other inherited metabolic disorders, glycobiology, proteomics and functional genomics of the cell.

Alexey Pshezhetsky

Dr. Steven Gray earned his Ph.D. in molecular biology from Vanderbilt University in 2006, after receiving a B.S. degree with honors from Auburn University. He performed a postdoctoral fellowship focusing on gene therapy in the laboratory of Jude Samulski at UNC Chapel Hill. He is currently an Associate Professor in the Department of Pediatrics at the University of Texas Southwestern Medical Center. Dr. Gray is the director of the UTSW Viral Vector Facility and maintains affiliations with the Department of Molecular Biology, the Department of Neurology and Neurotherapeutics, the Eugene McDermott Center for Human Growth and Development, and the Hamon Center for Regenerative Science and Medicine at UT Southwestern. Dr. Gray’s core expertise is in AAV gene therapy vector engineering, followed by optimizing approaches to deliver a gene to the nervous system. His research focus has also included preclinical studies to development AAV-based treatments for neurological diseases, some of which have translated into clinical trials.

Steve Gray

Dr. Shaywitz is currently Chief Medical Officer-in-Residence at BridgeBio, Inc. Prior to this he worked for five years at BioMarin Pharmaceutical where he played key roles in the development of a number of rare disease programs including but not limited to those targeting lysosomal storage diseases and MPS disorders. He designed and led the early stage clinical studies for the BMN 250 program in Sanfilippo Syndrome Type B and also worked closely with the research and business development teams on portfolio prioritization and in-licensing activities. Before joining BioMarin Dr. Shaywitz spent four years at Amgen in their early clinical development group focused on advancing promising diabetes and metabolism agents into the clinic. He received his MD and PhD degrees from Harvard Medical School and trained in adult medicine at Massachusetts General Hospital, in Boston. He went on to pursue a clinical fellowship in endocrinology, diabetes and metabolism jointly at the Israel-Deaconess Medical Center and Joslin Diabetes Center, both in Boston, where he concurrently pursued postdoctoral studies in molecular physiology.

Adam Shaywitz

Steve is the Chief Medical Officer of Allievex Corporation, a clinical-stage pharmaceutical company developing therapies for Sanfilippo syndrome type B and other pediatric neurodegenerative disorders. He is also the acting Chief Medical Officer of Grace Sciences LLC, a biotechnology company whose aim is to develop a gene therapy for NGly1 deficiency, another devastating neurological disease of childhood. Steve is a board-certified child neurologist and clinical development scientist with experience in rare disease therapeutic development and specific expertise in enzyme replacement and gene therapy. He previously worked in clinical development at BioMarin Pharmaceutical and has served on the Pediatrics and Child Neurology faculty at three world-renowned children’s hospitals. Steve was an NIH- and privately-funded basic science primary investigator who is internationally recognized for research in neural development. He is a recipient of the Child Neurology Society Philip R. Dodge Young Investigator Award for outstanding early career research.

Steve Maricich

Dr. Steve Walkley is a Professor at Albert Einstein College of Medicine. His research interests are concerned with analysis of pathogenic cascades and development of therapeutic strategies for genetic disorders of the endosomal-lysosomal system known as neuronal storage diseases. Disorders include Tay-Sachs, Hurler, Sanfillippo, Niemann-Pick, Batten, and other related conditions. Primary proteins implicated in storage diseases include not only lysosomal hydrolases but also soluble and membrane associated proteins often of unknown function. His studies are focused on the link between the primary protein defect and the abnormal accumulation of substrate (gangliosides, glycosaminoglycans, cholesterol, and so forth) and with subsequent induced changes in trafficking and signaling events within affected neurons.

Steve Walkley

Srikanth Singamsetty is experienced in academic and biopharma industry drug development programs. His most recent positions are at the University of North Carolina Catalyst for Rare Diseases (gene therapy programs) and Charles River Laboratories Discovery and Safety Assessment group.

Srikanth Singamsetty

Jill Wood graduated from Oregon State University with a BS in retail management and apparel design. Jill is a rare disease patient advocate as her son Jonah was diagnosed with Sanfilippo syndrome. She co-founded Jonah’s Just Begun (JJB) with her husband in 2010, a non-profit to raise awareness and support those with Sanfilippo. She built a scientific advisory board, created a comprehensive social media strategy and raised funds to support the research into Sanfilippo syndrome. In 2012 she co-founded Phoenix Nest with the aim to develop treatments for Sanfilippo Syndrome. Jill frequently lobbies on Capitol Hill with the RDLA in support of rare disease initiatives. Jill is also a speaker at major conferences where she tells her story from mom to CEO of a rare disease organization.

Jill Wood

For many years, Dr. Elsa Shapiro specialized in research at the University of Minnesota into numerous neurodegenerative diseases, including metabolic diseases.&nbsp; Over the years she has concentrated her research on the brain outcomes of various neurodegenerative diseases in children. She is particularly interested in the relationship of neuroimaging to cognitive outcomes in the developing child. Currently, she is a semi-retired research consultant to the NIH-supported Lysosomal Disease Network (headquartered at the University of Minnesota), which funds and oversees more than 20 research studies of various lysosomal diseases. She was the original Principal Investigator of the ground-breaking multicenter study “Longitudinal Studies of Brain Structure and Function in MPS Disorders,” examining longitudinal changes in quantitative neuroimaging, neuropsychological markers, quality of life, and biomarkers in more than 100 children around the country with MPS I, II, and VI. The current Principal Investigator of this ongoing study is Chester B. Whitley, Ph.D., M.D., who is also the Principal Investigator of the Lysosomal Disease Network. “Longitudinal Studies of Brain Structure and Function in MPS Disorders” is funded by the NIH, Genzyme Corporation and ShireHGT, as well as the National MPS Society and The Ryan Foundation. In addition, Dr. Shapiro has been co-investigator on ShireHGT studies of the natural history of MPS IIIA and MPS IIIB, studying the cognitive and MRI changes over time to identify sensitive outcome measures that might be needed for treatment trials. In summary, her interests lie in exploring the areas of childhood dementia and cognitive impairment using a variety of tools such as quantitative neuroimaging and neuropsychology to study diseases and conditions that affect the central nervous system. In this era of development of new treatments, identifying assessment methods that are sensitive to treatment outcomes is crucial to the identification of effective new treatments.

Elsa Shapiro

Robert D. Steiner, MD, FAAP, FACMG is Editor-in-Chief of Genetics in Medicine (GIM). He is Professor (Clinical) of Pediatrics at the University of Wisconsin (UW) School of Medicine and Public Health, Chief Medical Officer for PreventionGenetics, and geneticist and Medical Director at Marshfield Clinic Health System. After obtaining his MD from UW, he completed a residency in pediatrics at The University of Cincinnati/Cincinnati Children’s Hospital and Medical Center. Following residency, Dr. Steiner trained in medical genetics at The University of Washington/Seattle Children’s Hospital and Regional Medical Center. He is board certified in Pediatrics, Clinical Genetics, and Clinical Biochemical Genetics.

Steiner’s previous academic positions included Executive Director of the Marshfield Clinic Research Institute and Chief Science Officer at Marshfield Clinic, as well as Executive Associate Director of the UW Institute for Clinical and Translational Research. For most of his career, he was a faculty member at Oregon Health & Science University (OHSU), ultimately named Credit Unions for Kids Professor of Pediatric Research, Professor of Pediatrics and Molecular & Medical Genetics, and (tenured) Vice Chair for Research in Pediatrics. Steiner also served as Chief Medical Officer for Acer Therapeutics. Dr. Steiner is an active clinician with specific interests in inherited metabolic diseases, metabolic bone diseases, laboratory testing, and newborn screening. In addition, he is an accomplished researcher, having served as the Principal Investigator for more than a dozen clinical trials and research studies on Smith-Lemli-Opitz Syndrome (SLOS), Osteogenesis Imperfecta, Phenylketonuria, Lysosomal Storage Diseases, and others. Steiner has authored or co-authored over 150 peer-reviewed original research articles as well as more than 80 reviews, book chapters, and letters. He’s also been a longtime reviewer and award-winning editor for a number of journals.

Dr. Steiner is a member of several professional societies, including the American Society of Human Genetics, American College of Medical Genetics and Genomics (ACMG), Society for Inherited Metabolic Disorders, and the Society for the Study of Inborn Errors of Metabolism. He has had numerous appointments on national committees and advisory boards including as a member and treasurer of the ACMG Board of Directors and of the Health Care System Research Network Board of Governors, the Institute of Medicine advisory panel on accelerating rare disease research, serving as Chair of the ACMG working group on Pompe disease, as a Standing and Steering Committee member of the Newborn Screening Translational Research Network, as an NIH Study Section ad hoc member, and as a member of the ACMG Therapeutics Committee. He was a member of the expert panel convened in 2005 by the US Health and Human Services Health Research Services Agency that reviewed the status of newborn screening in the US and recommended expansion of the program to the US Secretary of Health and Human Services.

Robert Steiner

Matthew is a commercial leader with extensive background in rare disease pipeline marketing and commercial development. He has a broad, strategic global experience with proven execution in complex projects and markets. He thrives in dynamic, fast-paced environments - such as start-ups, launches or high-stakes projects. He is driven to get impactful products to the patients - to as many as possible, as quickly as possible.

Matthew has led the launch of products in the US and overseen global product launches. He, and his team, have prepared markets by liaising closely with advocacy groups, identifying unmet needs, driving diagnosis and ensuring patients have expert care at a defined medical home.

Prior to approval, Matthew has worked on dozens of programs in research and worked with the cross-functional team to bring many of them into and through clinic development At first, his role is providing the business case and pathway to commercialization. He has worked closely with Clinical Development, Clinical Operations, Tech Ops and Regulatory to ensure that once a product is approved, it has all of the right evidence and attributes for success in the market.

Matthew has led the commercial efforts for >50 Business Development deals, focusing on opportunity assessments, revenue forecasts and commercial plans. He has been part of ~15-20 executed BD deals, several of which evolved into leading the commercial integration and launch team.

Matthew is recognized for hiring/building strong teams, driving for results, facilitating decision-making with ambiguous or incomplete information, and collaboration across functions.

Before his career in biotech/ pharmaceuticals, Matthew served in the US Peace Corps doing small business development with a local handicraft operation and, in his free time, started a youth soccer league in the village with his soccer teammates.

His worked experience includes Genentech, BioMarin Pharmaceutical, Chiron Corporation, Depomed Inc, and BioRad Labs in roles from Marketing, Pipeline Marketing, Sales, Sales Operations, Market Planning and Portfolio & Project Management. He has BS in Chemical Engineering from University of Notre Dame and an MBA from Haas School of Business at UC Berkeley.

Matthew Hawe

Vanessa Rangel Miller is certified genetic counselor, with over 15 years' experience in rare disease, patient engagement and genetic programs. Vanessa is currently in Medical Affairs at Ultragenyx. Previously at Invitae, Vanessa served in Biopharma/Advocacy Business Development, bringing together multiple partners in the biopharmaceutical industry to launch genetic diagnostic programs. As a co-founder of PatientCrossroads (d.b.a. AltaVoice), Vanessa brought together technology, genetics, and external partners, and was responsible for >50 patient-centered registry programs in rare and non-rare disease, including partnering in the formation of ConnectMPS, a global patient registry for mucopolysaccharidoses and mucolipidoses, which originated from the Sanfilippo community. Vanessa has previously worked for Emory Genetics Laboratory, Emory University, and completed her master’s in genetic counseling at the University of North Carolina-Greensboro and her MBA at Emory University.

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