Joshua Lilienstein Email and Phone Number
Joshua Lilienstein is a Senior Medical Director at Avidity Biosciences, Inc.. Colleagues describe them as "I am pleased to recommend Joshua for any organization where he can bring his experience and expertise. During my first year at BioMarin, I served as Joshua's line manager. Throughout this time, Joshua was a significant help, particularly during my onboarding process. His meticulous planning and systematic approach to sharing information ensured a smoother transition into the company. Working with Joshua was a very pleasant and positive experience. He consistently demonstrated reliability, delivering his outputs on time and to a good standard of quality. His intellectual acumen and inquisitive mind, coupled with his ability to analyze situations and devise potential solutions, are noteworthy. Additionally, Joshua knows when to seek assistance, which enhances his effectiveness in any role. One of Joshua's notable contributions was his leadership in engaging internal and external stakeholders to re-examine data from existing studies and develop innovative methods for analyzing results. This introduced new perspectives that ultimately benefited patients. Joshua’s ability to lead these efforts highlights his commitment to enhancing outcomes. In conclusion, I recommend Joshua for any position where his skills, knowledge, and leadership can be utilized. His contributions during my time as his line manager were very valuable, and I am confident that he will bring substantial value to any team he joins." and "I had the pleasure of not only working with Joshua for almost 4 years, but being trained by him when I first entered the pharma and medical affairs world. Joshua is a brilliant, ever curious, outside the box thinker. He artfully blends scientific curiosity with business strategy, and somehow always sees the forest for the trees. He communicates elegantly and confidently, yet also humbly and with a desire to understand, which frequently makes him the preferred work-stream spokesman. He demonstrates a strong patient first approach, with genuine compassion and empathy for the patient journey. Joshua is a phenomenal teacher, collaborator and thought partner. Some of the work I’ve been most proud of thus far in my career has been done in partnership with Joshua. Perhaps most notably, he maintains an enviable perspective on what’s most important, both at work, but in life in general. I could not recommend Joshua more highly as an asset to any workplace. "
Avidity Biosciences, Inc.
View- Website:
- aviditybio.com
- Employees:
- 53
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Senior Medical DirectorAvidity Biosciences, Inc. Dec 2024 - PresentNorth American Medical Lead, DMD -
Senior Medical DirectorDelphi (Delphi Circle) Jun 2024 - Dec 2024Delphi Circle is a consultancy and incubator which seeks to develop and integrate psychedelic therapy into the existing mental and behavioral health ecosystem. Potential clients include drug and device developers, organizations building treatment facilities and wellness communities, public and private insurance organizations which would eventually be paying for those treatments; governmental agencies which would be regulating those treatments; and those who would seek to invest in the space. -
Senior Medical Director, Global Medical Lead, Early ProgramsBiomarin Pharmaceutical Inc. Dec 2022 - Jun 2024United StatesThis new role for the organization was created to ensure that the needs of payers and everyday clinicians were heard and prioritized when designing development strategies for the portfolio of early-stage programs• Treatment modalities: small molecule, biologic (ASO, enzyme substitution, antibody), AAV gene therapy• Therapeutic areas: musculoskeletal, cardiovascular, metabolic, non-onc heme• Led Strategic Medical Affairs Teams, Integrated Evidence Teams• Key Member of Asset (Core) Teams, Clinical Development Teams, Value/Access/Pricing Teams• Participated in business development and portfolio optimization assessments by quantifying the unmet medical need, identifying and sizing target populations, assessing the competitive landscape. This led to strategic re-allocation of company resources, including discontinuing several programs, accelerating others, and initiation of asset acquisitions -
Medical Director, Global Medical Lead, Early ProgramsBiomarin Pharmaceutical Inc. Oct 2022 - Mar 2023United States -
Medical Director, Pku Global ProgramBiomarin Pharmaceutical Inc. Sep 2021 - Oct 2022California, United StatesMedical oversight of two approved drugs (one small molecule, one biologic) and one pipeline asset (AAV5 gene therapy) for the treatment of phenylketonuria, a rare inborn error of metabolism with neuropsychological sequelae. • Responsible for the development and execution of the Global Medical Plan (including the External Engagement Plan, the Scientific Communications Plan, and the Evidence Generation Plan)• Primary face of the company for all external facing engagement related to the disease• Key Member of Asset (Core) Team, Value/Access/Pricing Team• Coordinated with regional and country medical leads and field teams to support HCPs, payors, and patients• Responsible for medical and scientific review of regulatory filings (REMS, DSUR) and label changes (PLLR, sBLA)• Clinical Lead for two phase 4 clinical studies and two patient registries.o Obtained FDA agreement to close out a Post-Marketing Requirement (PMR) registry 2 years early, saving the company ~$2M (PKUDOS)o Satisfactorily brought to completion an FDA PMR clinical study (PKU-015), authored final CSR and manuscripto Obtained EMA agreement to close out one Post-Marketing Requirement registry 5 years early, saving the company ~$5M (KAMPER)o Satisfactorily brought to completion an EMA PMR clinical study (SPARK), authored final CSR and manuscript -
Medical DirectorBiomarin Pharmaceutical Inc. Feb 2019 - Sep 2021San Rafael, Ca• Responsible for the development and execution of the PKU Regional Medical Plan• Clinical Lead for one PMR clinical study and a PMR patient registry• Led Medical support of a successful US launch of Palynziq, a first-in-class enzyme substitution therapy -
Associate Medical DirectorBiomarin Pharmaceutical Inc. Aug 2016 - Jan 2019San Rafael, CaPKUNorth America -
Chief Medical OfficerNanox Imaging Jul 2014 - Jun 2016Contributed to the design of novel field effect cathode technology, adapting it to the needs of the medical imaging market. Nanox technology represents the first fundamental re-design of the X-ray tube since its invention, expanding the capabilities of medical imaging.• Worked closely with the CEO and CTO to forge a development plan, defining potential verticals, sizing target markets, and articulating the business plan• Served as the primary liaison to key clinical and industry partners• Represented the company at major industry trade shows• Forged partnerships and licensing contracts with leading international medical imaging companies -
Radiology ResidentTufts Medical Center Jul 2013 - Jul 2014Greater Boston AreaExperience in Interventional Radiology, Body CT, Neuroradiology, Pediatric Radiology, Fluoroscopy, Ultrasound, Nuclear Medicine, and Musculoskeletal Radiology• Participation in multidisciplinary rounds with the departments of Neurosurgery and Vascular Surgery• Initiated a collaboration between the departments of Radiology and Hematology/Oncology to create a cloud-based, HIPAA-compliant image sharing platform• Prepared and delivered lectures to medical students• Instructor in the Tufts School of Medicine “Healer’s Art” course for 2 years -
InternLemuel Shattuck/Tufts Medical Center Jun 2012 - Jun 2013
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Research Aid, Pi: Dr Gary Ulaner, Department Of RadiologyMemorial Sloan-Kettering Cancer Center Jan 2011 - Jun 2011Research projects in the application of PET/CT to the imaging of breast cancer and lymphoma. This effort has produced data for three publications and two presentations at RSNA. -
Md/Phd Student. Pi: Dr. Axel SchôNthal; Department Of PathologyUniversity Of Southern California Mar 2008 - Aug 2008Los Angeles, Ca- Investigated the abilities of chloroquine and non-Cox-2 inhibitory analogs of celecoxib to disrupt autophagy in an effort to develop novel treatments for breast carcinoma, glioblastoma multiforme, and multiple myeloma. -
Md/Phd Student. Pi: Dr. C.P. Reynolds; Developmental Therapeutics ProgramChildren'S Hospital Los Angeles (Chla) Nov 2007 - Mar 2008Los Angeles, Ca- Investigated the cellular biology of ceramide metabolism. Our efforts focused on using novel retinoic acid derivatives as new treatments for neuroblastoma. - My research assessed the effect of various small molecule inhibitors of sphingomyelin metabolism on cell viability, as part of an ongoing effort to identify clinically-relevant drug combinations. -
Research Study Coordinator. Pi: Dr. Bruce AmesChildrens Hospital Oakland Research Institute Jan 2004 - Aug 2005- Coordinated an effort to develop a nutritional supplement to treat and prevent childhood obesity and pre- diabetes. - Wrote the grant proposal which detailed the scientific rationale behind our approach. - Initiated a collaboration between Children's Hospital Oakland Research Institute and the USDA.- Planned a clinical trial to test the effectiveness of our intervention, and co-authored the resultant publication.
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Research Assistant IiChildren'S Hospital Oakland Research Institute Jan 2004 - Aug 2005- Developed a method to measure the functional effect of serum inflammatory mediators on the vascular endothelium. - Techniques include mouse models, high-throughput screening, transfection, Western blot, PCR -
Research InternStanford University School Of Medicine Jan 2004 - Aug 2005Stanford, CaAnalyzed data on a number of studies investigating the use of computer aided detection in breast MRI. Authored a case report on a novel technique to correlate MRI and ultrasound findings. -
Research InternOcean Medicine Foundation Jan 2004 - Aug 2005Palo Alto, CaWrote requests for funding, and developed a website to coordinate telemedicine efforts for a non-profit that seeks to improve the state of health care delivery and medical information systems on remote islands.
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Editorial AssistantCell Death And Differentiation - A Nature Publishing Group Journal Jan 2003 - Oct 2003Rome, Italy- Managed the “Reviews” and “News and Commentaries” sections of the journal. - Organized meetings and symposia.- Edited journal articles submitted by non-English speaking authors. - Aided in the translation of a text book on apoptosis.
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Laboratory ManagerChildren'S Hospital Oakland Research Institute Jan 2001 - Jan 2003- Implemented a small animal study that sought to elucidate the biochemical relationship between a western-style ! diet and the chronic low level systemic inflammation at the root of Western style disease.- Responsible for managing and training undergraduate assistants.
Joshua Lilienstein Education Details
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Doctor Of Medicine (Md) -
Neuroscience -
Certificate In Psychedelic Therapies And Research
Frequently Asked Questions about Joshua Lilienstein
What company does Joshua Lilienstein work for?
Joshua Lilienstein works for Avidity Biosciences, Inc.
What is Joshua Lilienstein's role at the current company?
Joshua Lilienstein's current role is Senior Medical Director.
What schools did Joshua Lilienstein attend?
Joshua Lilienstein attended Keck School Of Medicine Of The University Of Southern California, University Of California, Berkeley, California Institute Of Integral Studies.
Who are Joshua Lilienstein's colleagues?
Joshua Lilienstein's colleagues are Lecya Tyaglo, Eric Horne, Allice Yang, Berta Thimmig, Martin Navarrete, Noah Luther, Stephanie Huynh.
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Joshua Lilienstein
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