NF2 BioSolutions Chief Scientific Officer (CSO)
Edgar Rodríguez-Lebrón, Ph.D.
Dr. Rodríguez-Lebrón has over 18 years of experience in the use of AAV as a CNS gene delivery vector. His research has relied on the use of model systems to test novel gene-based therapies that prevent, halt or reverse neurodegenerative processes caused by the abnormal accumulation of misfolded proteins. He has pioneered AAV-based gene therapies that make use of microRNA molecules and gene editing tools to suppress the expression of these proteins in CNS resident cells. Before joining Lacerta as CSO, Dr. Rodríguez-Lebrón held a faculty appointment in the Department of Pharmacology and Therapeutics at the University of Florida. He received his Ph.D. in Neuroscience from the University of Florida where he trained in the laboratories of Drs. Ron Mandel, Alfred Lewin and Nicholas Muzyczka.
Scientific Advisory Board:
Krizelle Alcantara
Krizelle obtained her bachelor’s and master’s degrees in Molecular Biology and Biotechnology from the University of the Philippines in Manila, Philippines. Her diagnosis with NF2 in 2007 when she was 15 years old led her to pursue NF2 research for her BS and MS theses. For her project, she demonstrated the inactivation of the neurofibromin 2 tumor suppressor gene by deregulated miRNAs in cancer cells leading to enhanced oncogenicity.
Upon graduating cum laude with her bachelor’s degree in 2012, she initially worked in a private laboratory providing genetics and cell therapy services to cancer patients. She then joined the Philippine Genome Center (PGC) in 2013 as part of the team that established the center’s DNA sequencing core facility. Afterwards, she led a project under the Health Program of the PGC which aimed to detect and characterize novel ethnicity-specific mutations among Filipino colorectal cancer patients which may serve as predictive biomarkers for response to anti-EGFR therapy. Before leaving the Philippines in 2019, Krizelle worked briefly under a cell-based ADMET assays project as part of the drug discovery program for priority disease indications of the Philippines’ Department of Science and Technology.
In October 2019, Krizelle relocated to the United States to fully focus her research efforts on the pilot pre-clinical NF2 gene therapy study funded by NF2 BioSolutions. She currently works as a research assistant under Dr. Kathrin Meyer at the Center for Gene Therapy of the Abigail Wexner Research Institute at Nationwide Children’s Hospital in Columbus, Ohio.
Krizelle hopes to use her experience as both a molecular biologist and an NF2 patient to accelerate the molecular understanding and development of treatment strategies of neurofibromatosis type 2.
Gary Jay Brenner, M.D., Ph.D.
Dr. Brenner currently sees patients at the Massachusetts General Hospital Center for Pain Medicine and has been the Director of the MGH Pain Medicine Fellowship since 2002. He is an Associate Professor of Anesthesia at Harvard Medical School and has authored more than 50 publications including peer-review articles, reviews, and book chapters; these have related to the pathophysiology of pain, treatment of neurofibromatosis, neuro immunology and clinical approaches to chronic pain. The major focus of his laboratory is the development of novel treatments for schwannoma.
As part of his long-standing commitment to pain medicine education Dr. Brenner currently serves on the ABA Pain Medicine Committee and has leadership positions in the Association of Pain Program Directors (APPD), the Society of Academic Anesthesia Associations (SAAA) and the American Society of Regional Anesthesia and Pain Medicine.
Dr. Brenner attended Wesleyan University where he received a BA in Biology with a minor concentration in East Asian Studies. After college he worked in Wuhan, China for one year teaching university students and studying the delivery of traditional Chinese medicine. Upon returning to the United States, he studied at the University of Rochester School of Medicine and Dentistry, New York, where he earned MD and PhD (immunology/microbiology) degrees. Dr. Brenner completed an internship in internal medicine in Rochester, followed by anesthesiology residency and pain medicine fellowship at the Massachusetts General Hospital.
Frank Buono, Ph.D.
Frank Buono is an Associate Research Scientist in the Department of Psychiatry at Yale School of Medicine. His training as a behavioral psychologist with expertise on decision making over the last decade has allowed me to develop a diverse background of research within various populations. His current research focuses on empirically-based psychosocial interventions that embeds technology to facilitate treatment to underserved populations and those with genetic disorders. He has received several grants from the Department of Defense, Children’s Tumor Foundation, and private grants for my research on Neurofibromatosis. Additionally, he was diagnosed with NF2 when he was nine years old, and having the experience of living with NF2 has allowed him to provide treatment both clinically and through research for those suffering with NF2.
Jonathan Chernoff, Ph.D.
As Chief Scientific Officer, Dr. Chernoff coordinates and charts the future course of research at Fox Chase Cancer Center.
Dr. Chernoff joined the staff in 1991 as an associate member and was promoted to member with tenure in 1996. In 2002 he was promoted to be a senior member in Fox Chase Cancer Center’s Basic Science division, the equivalent of a full professor in a university.
A molecular oncologist as well as a board-certified medical oncologist, Dr. Chernoff has a special interest in factors that control cell growth and movement, including oncogenes and anticancer or tumor-suppressor genes, and has made fundamental contributions in this research. His group helped establish a link between the Rac/Pak signaling system and the Merlin protein, and also a connection between fatty acid synthesis and growth of Merlin-deficient Schwann cells.
Dr. Chernoff earned his MD and his PhD in biochemistry in 1984 at Mount Sinai School of Medicine in New York City. He completed his residency in internal medicine at the University Health Center of Pittsburgh and a clinical fellowship in medical oncology at Johns Hopkins Oncology Center. He then held a postdoctoral fellowship in cellular and developmental biology at Harvard University before coming to Fox Chase.
In recognition of his national reputation in molecular oncology and biochemistry, Dr. Chernoff also holds the Stanley P. Reimann Chair in Oncology.
Gareth Evans, M.D., PhD
Professor Evans has established an international reputation in clinical and research aspects of cancer genetics, particularly in neurofibromatosis and breast cancer. He has developed a clinical service for cancer genetics in the North West Region of England, UK which is nationally regarded. He is an important opinion leader nationally through membership of committees and was chairman of the NICE Familial Breast Cancer Guideline Development Group and is now the clinical lead. Prof Evans has raised millions of pounds in NHS provisions and research grants, and successfully started the National NF2 service that began in 2010 in the UK.
Professor Evans sits on the board of trustee of NF2 BioSolutions UK chapter.
Rick Friedman, M.D., Ph.D.
Dr. Friedman is a professor of surgery and vice chief of otolaryngology at the University of California San Diego. In collaboration with Dr. Marc Schwartz he is the co-director of the UC San Diego Health Acoustic Neuroma Center and Center for Auditory Brainstem Implants. Dr. Friedman is an internationally recognized skull base surgeon (neurotologist) and has served all of the major organizations professionally including approximately 10 years as the co-chair of the medical advisory board of the Acoustic Neuroma Association. He is an NIH funded clinician scientist committed to discovery science and making the lives of patients better. He is married with three children and spends some of his time surfing the San Diego coast.
Michel Kalamarides, M.D., Ph.D.
Michel Kalamarides is Professor of Neurosurgery at Pitié-Salpetrière Hôpital (Sorbonne- Université), Paris France. He is specialized in vestibular schwannoma, meningioma and spinal ependymoma surgery.
He is leading the French NF2 reference clinical center located at Pitie-Salpetriere Hospital and the French NF2 network (4 affiliated NF2- clinics across France). The clinic has been organized to allow the NF2 patients to have their annual check-up during a single half-day visit and to be able to make a fast-track complete assessment for new patients.
Michel Kalamarides is involved within the French NF patients association-Fondation CAP NF (scientific advisory board member).
Within a large INSERM unit- U 1127 at Brain and Spine Institute (Pitié-Salpetrière), he is leading a research group on meningioma (one of the NF2 type of tumor), particularly using mouse models to dissect the mechanisms of meningioma initiation and progression.
Joseph Kissil, Ph.D.
Dr. Joseph Kissil earned his Ph.D. in molecular biology from the Weizmann Institute of Science in Rehovot, Israel. He completed a post-doctoral fellowship at the Massachusetts Institute of Technology, where he specialized in the development of mouse models of cancer. During this time Dr. Kissil’s work focused on understanding the biological processes involved in Neurofibromatosis type 2. In 2004 Dr. Kissil joined the faculty at the Wistar Institute in Philadelphia where his work on NF2 continued. His group identified key functions of Merlin, the protein encoded by the NF2 gene, which offered potential targets for therapeutic intervention. Since 2012 Dr. Kissil is at the Scripps Research institute where he is a Professor in the Department of Molecular Medicine. His group continues to work on understanding the molecular basis of NF2 and developing advanced tools and models to assess therapeutics.
Dione Kobayshi, Ph.D.
Accomplished executive scientist and a proven value creator with a successful record of drug development and company creation. Dr. Kobayashi has led or participated in raising $69.5M in capital for seed, Series A, or Fund entities. She is passionate about developing new medicines for serious diseases, and deeply knowledgeable in several rare disease fields. Dr. Kobayashi is experienced in leading groups across numerous disease areas and therapeutic modalities including antibodies, antisense oligonucleotides, enzymes, gene therapy, and small molecules. She has roles in entities spanning seed/venture-backed startups, venture philanthropy, and large biotech and pharma environments. Dr. Kobayashi Co-Founded 1787 Bio, Imara, and Eperia, and has held leadership roles at Cydan Development, Alector, the Spinal Muscular Atrophy Foundation, and Rinat/Pfizer. Dr. Kobayashi received her PhD in Neuroscience at the University of Edinburgh and began her career at Genentech and Elan Pharmaceuticals. Currently Dr. Kobayashi is an advisor or board member with the Bluefield Project to Cure Frontotemporal Dementia, Muscular Dystrophy Association Venture Philantrophy, NF2 Biosolutions, RDMD, and Save Lydia.Randall D. Learish, Ph.D.
Dr. Learish received his degree in Biology from Westminster College, his Ph.D. in Molecular Genetics and Biochemistry from the University of Pittsburgh School of Medicine, and did his postdoctoral work at the University of Wisconsin-Madison.
He worked for Promega Corporation until 2008 when he joined Cellular Dynamics International as a senior scientist. He has led projects that include the creation of human induced pluripotent stem cell (iPSC) lines, genetic engineering of iPSC lines with lineage specific promoters, differentiation of iPSCs into cardiomyocytes (CM) or neurons, and development of large scale processes to manufacture cells. He is currently setting up a facility to manufacture dopaminergic neurons in a GMP environment, with the intention of supporting an investigational new drug (IND) application for the treatment of Parkinson’s disease. He and his wife, Lisa, have two children. Lisa was diagnosed with mosaic NF2 in 2013. Randy hopes to use his experience in translational research to advance treatment strategies for NF2, and to find a cure.
Cara O’Neill, M.D.
Cara O’Neill, MD graduated Cum Laude with a degree in Biological Sciences from the University of Delaware where she was inducted into Phi Beta Kappa Honor Society in 1997. She went on to receive her medical degree from West Virginia University School of Medicine and subsequently her Pediatric Residency training at the University of South Carolina. She has worked both in private practice and academic settings. During her tenure as an Assistant Professor of Clinical Pediatrics at the University of South Carolina, she helped train medical students and residents and practiced in a clinic specific to children with special healthcare needs.
Dr. O’Neill now serves as the Chief Science Officer at Cure Sanfilippo Foundation where she oversees the foundation’s basic science and clinical trial grants, collaborates in clinical trial protocol design, and leads internal research efforts. She became passionately involved in rare disease research and advocacy after her daughter’s diagnosis of Sanfilippo syndrome (MPS III) in 2013. Her uniquely paired career and life experiences allow her to bridge gaps between scientists, clinicians, industry and families, helping foster patient-centered research and translational paths forward for rare disease treatments.
Cara lives in Columbia, SC with her husband Glenn, son Beckham and daughter Eliza. She enjoys travel and exploring the world with her family.
Marc Otten M.D.
Dr. Marc L. Otten earned his medical degree at Cornell University, where he graduated with honors. He completed a neurological surgery internship, residency, and chief residency at the Columbia University Neurological Institute of New York. In addition, he completed a fellowship at the Minimally Invasive Cranial Base Surgery Center at Thomas Jefferson University in Philadelphia, specializing in endoscopic, microsurgical and nonoperative approaches to brain and spinal disorders. Dr. Otten’s practice is devoted to tumors of the brain, skull base tumors, gliomas, meningiomas, acoustic neuromas, pituitary tumors, craniopharyngiomas, and microvascular decompression (MVD) for trigeminal neuralgia, and hemifacial spasm. He also treats hydrocephalus and neurotrauma.
He uses endoscopy, laser ablation, awake craniotomy, and other specialized techniques to minimize risk to patients with disorders of the brain. He has been published in leading, peer-reviewed journals about his work on noninvasive methods for mapping brain function in patients with brain tumors, which helps maximize tumor resection while maintaining optimum safety.
Aziz Rehman, M.D.
Dr. Rehman graduated with Honors, Magna Cum Laude, in Biochemistry from Temple University, and was selected for Phi Beta Kappa in 2002. During his undergraduate years, he obtained a National Science Foundation grant to perform hematology research in the laboratory of Dr. Deane Mosher at the University of Wisconsin-Madison School of Medicine. He studied the effects of thrombospondin on fibronectin extracellular matrix assembly. In 2009, he earned his medical degree from St. Matthew’s University School of Medicine.
He has worked in biotech and medical device industries and obtained a regulatory affairs dual certificate in pharmaceuticals and medical devices from Regulatory Affairs Professionals’ Society (RAPS). He worked at MediMedia Health Inc., a contract research organization, Merck & Co., Inc., and DePuy Synthes companies of Johnson & Johnson. While at Merck, he supported a Phase 3b trial in CNS/Neurology as well as a Phase 2 trial in Immuno-Oncology.
Dr. Rehman is a performance driven physician executive with extensive experience in clinical trial management. He is knowledgeable of full life cycle product development of biopharmaceuticals and medical devices in the functional areas of clinical development, pharmacovigilance, regulatory affairs, and medical affairs for investigational medicinal products and combination of drug/device products. Additionally, he has worked in multiple therapeutic areas including CNS/Neurology, Immuno-Oncology, Endocrinology, Rheumatology, Surgery (including trauma, orthopedics, neurosurgery, spine, and craniomaxillofacial surgical subspecialties) and molecular biochemistry.
Dr. Rehman’s interests include developing novel therapies for unmet genetically based diseases utilizing modern day molecular biology techniques such as RNAi for gene silencing and CRISPR Cas 9 genome editing complexes. He would like to develop gene therapies for Neurofibromatosis Types 1 & 2, Diabetes Type 1 & 2, autoimmune diseases, lysosomal storage diseases, Tay Sachs disease, Niemann-Pick, Pompe’s disease, and Gaucher’s disease.
Lyndi M. Rice, Ph.D.
Dr. Rice graduated with a degree in Biology and Chemistry from the Indiana University of Pennsylvania and received her doctorate degree in Molecular Cell Biology and Genetics from the Drexel University College of Medicine. She completed her post-doctoral training at the Wistar Institute.
Dr. Rice then joined Genesis Biotechnology Group, LLC , where she directed a team of scientists in multiple cancer drug discovery programs that spanned target identification/validation, ultra-high throughput assay development and validation, hit-to-lead and lead optimization, through preclinical development. More recently, she has moved into the field of gene therapy and worked at the Gene Therapy Program at the University of Pennsylvania where she served as the Director of Analytics. In that role, she oversaw potency assay development and qualification, all vector release and characterization assay development and qualification, analytical technical transfer to CMO, CTO, and sponsors, stability programs, compatibility studies, and contributed extensively to CMC sections of regulatory filings. She has recently joined BioMarin Pharmaceuticals where she will contribute to multiple gene therapy programs. Lyndi lives in San Francisco with her husband and daughter and she is committed to advancing research in gene therapy for rare diseases.
William H. Slattery III, M.D.
William H. Slattery, M.D. works in private practice at the House Clinic in Los Angeles, California. With over twenty years of experience in the field, he specializes in the medical and surgical treatments of hearing disorders, facial nerve disease, vestibular schwannoma and Neurofibromatosis type 2.
Dr. Slattery received his medical degree from the University of Missouri-Columbia. He completed his residency in Otolaryngology at the University of Florida and subsequently,
his fellowship in neurotology/skull base surgery at the House Clinic. Dr. Slattery joined the House Clinic in 1993.
In addition to his busy surgical practice, he has maintained a strong presence in the field as a respected clinical researcher. As the recipient of over $10 millions in federal funding (NIH and DOD), he continues to demonstrate his commitment to improving the understanding of and treatment for patients with acoustic neuroma and NF2 as well as enhancing current methods of drug delivery to the inner ear.
D. Bradley Welling, MD, PhD, FACS
Dr. Welling is the Walter Augustus Lecompte Professor and Chair of the Harvard Department of Otolaryngology Head and Neck Surgery and Chief of Otolaryngology Head and Neck Surgery at Massachusetts Eye and Ear and Massachusetts General Hospital.
Dr. Welling received his MD from the University of Utah, his residency in Otolaryngology-Head and Neck Surgery at the University of Iowa, and fellowship in Otology, Neurotology, and Cranial Base Surgery at the EAR Foundation in Nashville, Tennessee. He completed a PhD in pathobiology at The Ohio State University.
Dr. Welling has a long-standing interest in finding better treatment solutions for NF2. He and his collaborators have defined phenotype-genotype relationships, characterized the promoter region of the NF2 gene, and contributed relevant pathway mechanisms for merlin-deficient tumors. He has conducted and participated in multiple clinical trials intent on finding better treatment options for NF2. His clinical practice has focused on the surgical removal of vestibular schwannomas and on hearing preservation and restoration.
Dr. Welling has served on the Board of Directors of the American Board of Otolaryngology Head and Neck Surgery, and on the Residency Review Committee of the Accreditation Council on Graduate Medical Education (ACGME). He serves as the inaugural Editor-in-Chief of Laryngoscope Investigative Otolaryngology.
Kaleb Yohay, M.D.,Ph.D.
Associate Professor of Neurology and Pediatrics (Clinical) at NYU Langone Health. My career is centered on improving the lives and health of adults and children with Neurofibromatosis 1, Neurofibromatosis 2, Schwannomatosis and related conditions. I am the Director of the Comprehensive Neurofibromatosis Center at NYU Langone.
I have an interest in the development of best practices for symptom screening, treatment, and age appropriate anticipatory guidance for children and adults with NF. The NYU Langone NF Center is part of a national clinical trial consortium for NF, and as such, we are actively participating in several clinical trials for treatment of tumors in NF1 and NF2. I am a member of the Programmatic Review Panel for the Department of Defense, Congressionally Directed NF Research Program. Additionally, I recently joined the Clinical Research Award Review Panel and the Clinical Care Advisory Board for the Children’s Tumor Foundation.
In addition to my interests in Neurofibromatosis, I have a longstanding interest in undergraduate and graduate medical education and training. From 2007 until 2014 I was the Residency Program Director for the Child Neurology Residency at WCMC/NYP.