viernes, 30 de septiembre de 2022
Human Gene Therapy - September 2022
Human Gene Therapy - September 2022
SPECIAL ISSUE
29th Annual Congress of the ESGCT
Guest Editor:
Andrew Baker, PhD
Enjoy complimentary access to the latest digital edition of Human Gene Therapy.
The Editorial Staff of Human Gene Therapy is pleased to present this Special Issue on the occasion of the 2022 Congress of the European Society of Gene and Cell Therapy and the British Society of Gene and Cell Therapy. This issue includes a number of timely original research and review articles, providing insights into the ongoing progress in the field, as the world moves out of the global COVID-19 pandemic and the gene therapy community begins to assemble in-person once again to present, discuss, and debate new scientific and clinical findings.
Read featured articles below and you can access the full digital edition.
ESGCT 2022: The New Normal for the Gene Therapy Community
Terence R. Flotte Read Now
[ Open Access ]
Update on Viral Gene Therapy Clinical Trials for Retinal Diseases
Shun-Yun Cheng and Claudio Punzo Read Now
Central Nervous System Gene Therapy: Present Developments and Emerging Trends Accelerating Industry-Academia Pathways
Margareta Rybarikova, Amanda Almacellas-Barbanoj, Stéphanie Schorge, and Nicole Déglon Read Now
Liver Gene Therapy
Amit C. Nathwani, Jenny McIntosh, and Rose Sheridan Read Now
Progress in Respiratory Gene Therapy
Gerry McLachlan, Eric W.F.W. Alton, A. Christopher Boyd, Nora K. Clarke, Jane C. Davies, Deborah R. Gill, Uta Griesenbach, Jack W. Hickmott, Stephen Hyde, Kamran M. Miah, and Claudia Juarez Molina Read Now
Long-Term Disease Prevention with a Gene Therapy Targeting Oligodendrocytes in a Mouse Model of Adrenomyeloneuropathy
Yasemin Özgür-Günes, Malha Chedik, Catherine Le Stunff, Claire-Maëlle Fovet, and Pierre Bougnères Read Now
Methods for Extrapolating Survival Analyses for the Economic Evaluation of Advanced Therapy Medicinal Products
Will A.S. Hardy and Dyfrig A. Hughes Read Now
[ Open Access ]
Insight and Development of Advanced Recombinant Adeno-Associated Virus Analysis Tools Exploiting Single-Particle Quantification by Multidimensional Droplet Digital PCR
Jeanette Zanker, Sara Lázaro-Petri, Daniela Hüser, Regine Heilbronn, and Adrien Savy Read Now
Virtual Lunch & Learn: SAMHSA’s Office of Behavioral Health Equity Celebrates Hispanic Heritage Month Thursday, October 6, 2022 12:00 PM – 1:00 PM ET
Iniciar reunión - Zoom
Join SAMHSA’s Office of Behavioral Health Equity for a Lunch & Learn to celebrate Hispanic Heritage Month. This virtual learning opportunity will discuss successes of the Familia Adelante intervention with its developer, Dr. Richard Cervantes and spotlight NNEDLearn 2022 Familia Adelante participants through a digital story viewing. The Familia Adelante participants will share about their efforts with implementation of this intervention. In partnership with the National Latino Behavioral Health Association executive director, Mr. Fred Sandoval, MPA, this session will also host an exchange about how SAMHSA can increase behavioral health equity for the Hispanic/Latinx community.
Please click the link below to join this virtual session:
https://www.zoomgov.com/j/1616697635
Webinar ID: 161 669 7635
Passcode: 679743
Or One tap mobile:
US: +16468287666,,1616697635#,,,,*679743# or +15512851373,,1616697635#,,,,*679743#
Or Telephone Dial:
US: +1 646 828 7666 or +1 551 285 1373
jueves, 29 de septiembre de 2022
miércoles, 28 de septiembre de 2022
martes, 27 de septiembre de 2022
lunes, 26 de septiembre de 2022
domingo, 25 de septiembre de 2022
sábado, 24 de septiembre de 2022
viernes, 23 de septiembre de 2022
jueves, 22 de septiembre de 2022
miércoles, 21 de septiembre de 2022
Registration for AHRQ NRSA T32 Funding Opportunity Technical Assistance Call
Registration for AHRQ NRSA T32 Funding Opportunity Technical Assistance Call
AHRQ Seeks Applicants for National Research Service Award Institutional Research Training Program
AHRQ is seeking applications for its National Research Service Award (NRSA) Institutional Research Training Program. The NRSA program offers advanced training to predoctoral and postdoctoral fellows at eligible institutions with programs in health services research. AHRQ encourages all institutions, including minority-serving institutions, to apply for this T32 grant program. Letters of intent are due Oct. 14, and applications are due Dec. 1. Access more information. A technical assistance call is scheduled Oct. 5 from 1 to 2 p.m. ET. Registration is required.
Federal Register :: Request for Information on Person-Centered Care Planning for Multiple Chronic Conditions (MCC)
Federal Register :: Request for Information on Person-Centered Care Planning for Multiple Chronic Conditions (MCC)
Input Sought on How To Support Better Care for People With Multiple Chronic Conditions
AHRQ is requesting public comments to inform future planning and the agency’s research agenda to improve care for people at risk for, or living with, multiple chronic conditions (MCC). A Request for Information seeks input on the current state of shared, person-centered care planning for people with MCC, including insights into current and innovative models of shared care planning used in practice, scale of implementation, barriers and facilitators to implementation, and promising strategies and solutions to overcome implementation barriers. Input is sought on care models across a variety of care settings, including health systems, primary care, home and other ambulatory practices. Comments are due Nov. 15. Access the Request for Information for more information and submit responses to MCC@ahrq.hhs.gov.
martes, 20 de septiembre de 2022
lunes, 19 de septiembre de 2022
Webinar: SAMHSA Office of Behavioral Health Equity Recovery Month Thursday, September 22, 2022 1:30 PM – 3:00 PM ET
Iniciar reunión - Zoom
Webinar: SAMHSA Office of Behavioral Health Equity Recovery Month
Thursday, September 22, 2022 1:30 PM – 3:00 PM ET
The purpose of this webinar will be to discuss the need for behavioral health equity to ensure individuals living in underserved communities have access to recovery, treatment, and support.
Join us as our presenter, Dr. Victoria Cargill, will discuss:
The impact of discrimination, racism, and racial trauma on marginalized individuals living with substance use disorders;
Intersecting effects of the social determinants of health;
Effective and successful interventions that support substance use disorder treatment for underserved populations;
Barriers to equitable treatment, recovery, and support services; and
How we move forward together as a nation.
Please click the link below to join the webinar:
https://www.zoomgov.com/j/1614017737?pwd=RjNHcW9SWmR1RVJqRW10bFlJQ0hYZz09
Webinar ID: 161 401 7737. Passcode: 393593
Or One tap mobile :
US: +16468287666,,1614017737#,,,,*393593# or +15512851373,,1614017737#,,,,*393593#
Or Telephone:
US: +1 646 828 7666 or +1 551 285 1373
Presenter:
Headshot of Dr. Cargill
Victoria Cargill, MD, MSCE IAS-USE
Assistant Commissioner, Baltimore City Health Department
Dr. Victoria Cargill is a Boston and Harvard University trained board-certified internal medicine and HIV physician, and former Assistant Commissioner of Health at the Baltimore City Health Department, overseeing the Ryan White program and Community Risk Reduction/Syringe Support Services. Prior to becoming an Assistant Commissioner, she served as a senior policy analyst and program director for 20 years at the National Institutes of Health (NIH) in both the Office of AIDS Research (OAR) and the Office of Research on Women’s Health (ORWH). At OAR, she was the Director of Minority Research and Clinical Studies, and Acting Director of HIV Therapeutics Research. At ORWH, she served as the Associate Director for Interdisciplinary Research, and grew the U3 program focused on stimulating research on understudied, underreported, and underrepresented women to over $3 million dollars. She has also served as the acting chair of the FDA Antiviral Advisory Committee, including chairing the hearings of the first two U.S.-approved HCV antivirals.
A former co-investigator at the Case Western AIDS Clinical Trials Unit and the creator of an AIDS education and prevention adolescent-focused program that reached over 100,000 teens in five Ohio cities, she has extensive public health and HIV prevention research experience. Currently the principal investigator of a HRSA-funded Ending the HIV Epidemic grant, and the author of over 150 scientific articles, research presentations, book chapters and popular press educational materials, she has been nationally recognized as one of America’s Top Doctors. Dr. Cargill continues to consider patients and communities as her best teachers.
domingo, 18 de septiembre de 2022
sábado, 17 de septiembre de 2022
viernes, 16 de septiembre de 2022
NCTR Research Highlights | FDA
NCTR Research Highlights | FDA
FDA NanoDay Symposium 2022 — Register for the Free Event
The Center for Drug Evaluation and Research’s Small Business and Industry Assistance (CDER SBIA) group will host the FDA NanoDay Symposium 2022 on October 11th from 8:50 AM to 3:10 PM EASTERN. This event is free to the public, but registration is required.
Dr. Anil Patri, NCTR Nanocore Director and chair of the FDA Nanotechnology Task Force, will present the welcome message and discuss nanomaterial standards development at FDA. Dr. Patri will also participate in the Question & Answer panel at the end of the symposium.
For event information, including the agenda and virtual attendance, please visit FDA NanoDay Symposium 2022.
jueves, 15 de septiembre de 2022
miércoles, 14 de septiembre de 2022
RDCA-DAP | Critical Path Institute
RDCA-DAP | Critical Path Institute
FDA and NIH Launch Public-Private Partnership for Rare Neurodegenerative Diseases
Today, the U.S. Food and Drug Administration and the National Institutes of Health (NIH) announced the launch of the Critical Path for Rare Neurodegenerative Diseases (CP-RND) – a public-private partnership aimed at advancing the understanding of neurodegenerative diseases and fostering the development of treatments for amyotrophic lateral sclerosis (ALS) and other rare neurodegenerative diseases. The FDA and NIH have selected the Critical Path InstituteExternal Link Disclaimer (C-Path) as the convener of this partnership.
“There is a crucial need to develop new treatments that can improve and extend the lives of people diagnosed with rare neurodegenerative diseases, including ALS. Collaboration across public and private sectors can accelerate the progress to address this urgent need,” said FDA Chief Medical Officer, Hilary Marston, M.D., M.P.H. “The partnership we are announcing today will leverage the shared expertise of all participants to create a path towards new breakthroughs in treating these diseases. We look forward to working with NIH, C-Path, and other public and private partners to carry out this important effort.”
“This public-private partnership will convene the entire ALS community to develop novel strategies and approaches to therapy development and clinical testing with the goal to finally produce a treatment that stops the tragic progression of ALS,” said Walter Koroshetz, M.D., director of the National Institute of Neurological Disorders and Stroke (NINDS), part of the NIH.
C-Path will convene the partnership, bringing together experts in rare neurodegenerative diseases, including, but not limited to, patient communities, advocacy organizations, and private entities. The direction and priorities for the effort will be determined with input from the partners. Areas of focus will include patient-focused drug development, and utilization of the FDA-funded Rare Disease Cures Accelerator-Data and Analytics PlatformExternal Link Disclaimer (RDCA-DAP) to bring together scientific data on rare neurodegenerative diseases to facilitate the characterization of neurodegenerative diseases and their natural history, the identification of molecular targets for neurodegenerative disease, and increased efficiency, predictability, and productivity of clinical development of therapies.
Building on and leveraging the shared expertise of the participants, the goal of this partnership is to generate actionable solutions that can tangibly accelerate drug development for rare neurodegenerative diseases.
The Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act (Act for ALS) was signed into law on December 23, 2021 by President Biden and requires HHS, through FDA and NIH, to establish and implement a public-private partnership not later than one year after enactment. This partnership is a key component of the FDA’s Action Plan announced in June.
To learn more about the Critical Path for Rare Neurodegenerative Diseases, contact the C-Path team at CP-RND@cpath.org.
Critical Path Institute
Critical Path Institute
FDA and NIH Launch Public-Private Partnership for Rare Neurodegenerative Diseases
Today, the U.S. Food and Drug Administration and the National Institutes of Health (NIH) announced the launch of the Critical Path for Rare Neurodegenerative Diseases (CP-RND) – a public-private partnership aimed at advancing the understanding of neurodegenerative diseases and fostering the development of treatments for amyotrophic lateral sclerosis (ALS) and other rare neurodegenerative diseases. The FDA and NIH have selected the Critical Path InstituteExternal Link Disclaimer (C-Path) as the convener of this partnership.
“There is a crucial need to develop new treatments that can improve and extend the lives of people diagnosed with rare neurodegenerative diseases, including ALS. Collaboration across public and private sectors can accelerate the progress to address this urgent need,” said FDA Chief Medical Officer, Hilary Marston, M.D., M.P.H. “The partnership we are announcing today will leverage the shared expertise of all participants to create a path towards new breakthroughs in treating these diseases. We look forward to working with NIH, C-Path, and other public and private partners to carry out this important effort.”
“This public-private partnership will convene the entire ALS community to develop novel strategies and approaches to therapy development and clinical testing with the goal to finally produce a treatment that stops the tragic progression of ALS,” said Walter Koroshetz, M.D., director of the National Institute of Neurological Disorders and Stroke (NINDS), part of the NIH.
C-Path will convene the partnership, bringing together experts in rare neurodegenerative diseases, including, but not limited to, patient communities, advocacy organizations, and private entities. The direction and priorities for the effort will be determined with input from the partners. Areas of focus will include patient-focused drug development, and utilization of the FDA-funded Rare Disease Cures Accelerator-Data and Analytics PlatformExternal Link Disclaimer (RDCA-DAP) to bring together scientific data on rare neurodegenerative diseases to facilitate the characterization of neurodegenerative diseases and their natural history, the identification of molecular targets for neurodegenerative disease, and increased efficiency, predictability, and productivity of clinical development of therapies.
Building on and leveraging the shared expertise of the participants, the goal of this partnership is to generate actionable solutions that can tangibly accelerate drug development for rare neurodegenerative diseases.
The Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act (Act for ALS) was signed into law on December 23, 2021 by President Biden and requires HHS, through FDA and NIH, to establish and implement a public-private partnership not later than one year after enactment. This partnership is a key component of the FDA’s Action Plan announced in June.
To learn more about the Critical Path for Rare Neurodegenerative Diseases, contact the C-Path team at CP-RND@cpath.org.
Bridging Efficacy and Safety to the Obese: Considerations and Scientific Approaches - 11/09/2022 | FDA
Bridging Efficacy and Safety to the Obese: Considerations and Scientific Approaches - 11/09/2022 | FDA
FDA and M-CERSI Announce a Virtual Public Workshop on Bridging Drug Efficacy and Safety to Obese Patients
On November 9, 2022, the U.S. Food and Drug Administration (FDA) in collaboration with the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI) will hold a virtual public workshop entitled "Bridging Efficacy and Safety to the Obese: Considerations and Scientific Approaches." The purpose of this workshop is to review the implications of obesity in adult and pediatric patients on safety, efficacy, drug dosing and disposition.
Besides the major health and socioeconomic implications of obesity, there are great challenges in ensuring the optimal use and development of drugs for the growing population of obese persons. Obese patients are a large segment of the U.S. population and should be included in drug development studies. There are presently no clear dosing guidelines for obese pediatric patients. Although smaller clinical studies have begun to examine drug pharmacokinetics in obese pediatric patients, the relationship between pediatric obesity and drug response, including efficacy and safety, has not been thoroughly elucidated. Advancing this field requires identifying key knowledge gaps and barriers to the conduct of such studies and potential innovative solutions to improve data generation and analysis.
Workshop Objectives:
Review the implications of obesity in adult and pediatric patients on safety, efficacy, drug dosing and disposition.
Discuss: If adult and pediatric patients should be identified and studied as a special population in new drug development programs, and when it may be appropriate to include obese patients in drug development studies to develop specific dosing guidelines for obese patients.
Date/Time of the Workshop:
Date: November 9, 2022
Time: 8:30 a.m. – 4:15 p.m.
This virtual workshop is open to the public; however, registration is required at: https://bioeumd.wufoo.com/forms/m16xw7q80ra8tsz/.
More information about this event can be found at: https://www.fda.gov/drugs/news-events-human-drugs/bridging-efficacy-and-safety-obese-considerations-and-scientific-approaches-11092022.
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