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It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.
What are the main challenges currently faced in the treatment of chronic liver diseases, and how does Resolution Therapeutics aim to address these challenges? Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years.
As clinical trials become increasingly complex, particularly in decentralized trials and rare disease studies, sponsors experience increased challenges in site selection, forecasting and resourcing, and patient recruitment and enrollment.
Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.
to enhance outcomes, new treatments which will target kidney-specific disease mechanisms are highly desired,” said Hiddo L. Christian Rommel, Member of the chief Committee of Bayer AG’s Pharmaceutical Division and Head of Research and Development. 1 in 3 adults is at risk for the disease. In the U.S.,
Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. KIF1A-Related Research In the rare disease space, a common and straightforward question is: How rare is the disease?
Prior to joining Gannex, Dr. Palmer was Head of Liver DiseaseClinicalDevelopment at Takeda Pharmaceuticals. She has also held senior leadership positions at Shire Pharmaceuticals and Kadmon Corporation, where she was in charge of the global development of NASH and other liver disease programs.
He has contributed to more than 130 granted patents and advanced over 12 clinical drug candidates across various therapeutic areas, including cognition, depression, pain and other disorders. Now in Phase II development at Gate for depression and other synaptopathies, zelquistinel represents a key part of his current work.
Bayer announced today the initiation of the CONFIDENCE study, a Phase II, three-arm study that will investigate simultaneous initial combination therapy with finerenone and the SGLT2 inhibitor empagliflozin, compared with finerenone alone and empagliflozin alone respectively in patients with chronic kidney disease (CKD) and type 2 diabetes (T2D).
I was part of building up this small biopharma company and learn a lot about rare diseases, regulatory interactions and challenges related to that, challenges related to out licensing drug candidates and having new people joining the company every month as we were growing so fast.
By Mike Cloonan, Chief Executive Officer of Sionna Therapeutics, as part of the From The Trenches feature of LifeSciVC The drug development process in rare diseases is rife with challenges especially when companies target significant differentiation or first-in-class targets.
Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinicaldevelopment program will be provided at a future time following further discussions with health authorities. This guidance provides clarity on the path to registration for AVXS-101 IT.
From a very young age, I was drawn to science and medicine. This early exposure to bench science had a long-lasting impact. Through further research and work as a principal investigator on clinical studies for several central nervous system indications, I laid the groundwork for a career in gene therapy drug development.
Gilead Sciences, Inc. He completed his postdoctoral training at the University of Alabama at Birmingham in the Division of Developmental and Clinical Immunology. He has published numerous papers on the role of immune cells in driving inflammatory diseases and cancer, and holds a number of patents related to his discoveries.
This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinicaldevelopment, offering promising advancements in innovative cancer treatments.
Laboratory professionals in all areas of science have experienced significant changes in recent years, and 2022 was no exception. Additionally, chronic disease, cancer and neurodegenerative disorders like Alzheimer’s were top of mind last year for diagnostics and drug R&D teams alike.
Top 10 Life Science Resources pmjackson Mon, 10/30/2023 - 16:16 There’s a lot of life science content out there, which is why we’ve curated a selection of our expert insights, tips, case studies, and scientific and regulatory information for you. Catch up on what you may have missed below! Read it now. The Altascientist : Issue No.
PRA Health Sciences selected for the study which will position Awakn at the Forefront of Psychedelic Research for Addiction Treatment. Alcohol use disorder is the most prevalent substance use disorder, with alcohol use as one of the top five causes of disease and disability in almost all countries throughout Europe. Source link.
Spending several years with Sanofi in Paris, France as a Global safety officer, he then relocated to New Jersey, USA to work in Early and Late Stage ClinicalDevelopment in Immunology, when he became the Global Project Head of Dupixent, a medicine that has revolutionized the field of allergic and atopic diseases.
Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. Like hereditary spastic paraplegia, CMT is an umbrella disorder, associated with mutations in over 140 genes, but many diagnosed patients do not know their disease-causing mutation.
and Shionogi Limited as shareholders, today announced the positive findings of a pooled analysis of COVID-19-related impacts across the investigational long-acting cabotegravir and rilpivirine clinicaldevelopment programme. These findings were presented today at the 2020 Infectious Diseases Society of America (IDSA) IDWeek.
What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? But commitment to neuroscience drug development is critically important given the significant unmet medical needs and the ways in which patients, caregivers, and families suffer.
How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? Essentially, our CNPs reprogramme the immune system by restoring balance and returning the body to a homeostatic state, offering a precise approach to treating immune-mediated diseases.
Together, these nine companies have collectively developed more than 70 novel vaccines that have helped to eradicate some of the world’s most complex and deadly public health threats, underscoring their experience in clinicaldevelopment and regulatory rigor, as well as their longstanding commitments to patient safety and public health.
18 December 2020 — The US Food and Drug Administration (FDA) has requested further clarifying analyses of clinical data, to complete its review of the New Drug Application (NDA) for roxadustat, an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor for patients with anemia of chronic kidney disease (CKD).
Making a little tangle of DNA visible to the eye and understanding that this is the basis of complex organisms, which might be altered in disease, was the defining moment for my future path in life sciences. Communication and networking are key in any career, with science being no exception.
Based on Nobel Prize-winning research, IRLAB has grown rapidly to become recognised and respected as a world-leader in understanding the complex neuropharmacology of CNS disorders, especially Parkinson’s disease (PD). The third project in clinical phase, IRL757, is in Phase I and is being developed for the treatment of apathy.
Together, these nine companies have collectively developed more than 70 novel vaccines that have helped to eradicate some of the world’s most complex and deadly public health threats, underscoring their experience in clinicaldevelopment and regulatory rigor, as well as their longstanding commitments to patient safety and public health.
–( BUSINESS WIRE )– Gilead Sciences, Inc. While HIV treatment has advanced dramatically over the past three decades, people living with HIV still face a lifetime of therapy,” said Diana Brainard, MD, Senior Vice President, Virology Therapeutic Area, Gilead Sciences. 1, 2021 11:00 UTC. FOSTER CITY, Calif. &
The US Food and Drug Administration (FDA) has issued a complete response letter (CRL) regarding the New Drug Application (NDA) for roxadustat for the treatment of anaemia of chronic kidney disease (CKD), in both non-dialysis dependent (NDD) and dialysis-dependent (DD) adult patients. Anaemia is particularly prevalent in patients with CKD.
– More than 30 Abstracts Across HDV, HCV, HBV, NASH and PSC Reflect Gilead’s Ongoing Commitment to Addressing Challenges Facing People Living With Liver Diseases –. About Gilead Sciences in Liver Disease. For more than 20 years, Gilead has sought to address some of the biggest challenges in liver disease.
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P otential to be a safer and effective alternative to the intravenous immunotherapies currently used for Crohn’s Disease.
Crohn’s Disease Therapeutics Market Size $4.7
Phase1b/2 clinical study to be conducted in the United States and several European countries. Billion by 2025.
and Europe.
18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD).
NEW YORK , Jan.
NYSE: PFE) today reported positive Phase 2 pediatric data for their Lyme disease vaccine candidate, VLA15. About VLA15 VLA15 is the only Lyme disease vaccine candidate currently in clinicaldevelopment. Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company, and Pfizer Inc.
Shailubhai will provide updates on the Company’s proposed move from AIM to the Standard Segment of the Main Market of the London Stock Exchange, ongoing clinical trials, as well as other recent developments in Tiziana Life Sciences. About Tiziana Life Sciences. NEW YORK and LONDON, Dec.
+44 (0)20 7495 2379.
BIIB122 is an inhibitor of LRRK2, a potential novel target intended to impact the underlying biology and slow the progression of Parkinson’s disease Phase 2b LUMA to enroll approximately 640 participants with early-stage Parkinson’s disease; most advanced clinical study of a LRRK2 inhibitor. Denali Therapeutics Inc.
AbbVie (NYSE: ABBV) and Calico Life Sciences today announced an extension of their leading-edge collaboration to discover, develop and bring to market new therapies for patients with age-related diseases, including neurodegeneration and cancer. This is the second extension of the collaboration originally established in 2014.
Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company focused on the development and commercialization of prophylactic vaccines for infectious diseases with significant unmet medical need, and Pfizer Inc. VLA15 has demonstrated strong immunogenicity and safety data in pre-clinical and clinical studies so far.
A healthy immune system defends the body against disease and other conditions. Autoimmune disease impacts different parts of the body, weakening functionality. Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. It may be life-threatening.
Additionally, they compared their long COVID analysis with known genetic associations in various chronic diseases, particularly neurological and metabolic disorders. The combinatorial analysis revealed that 73 genes were significantly associated with long COVID development.
the completion of enrollment of its Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease (PD). Ph1 clinical trial is to evaluate the safety, tolerability, and?preliminary?efficacy?of of BRT-DA01 in patients with Parkinson’s disease.?. The purpose of the?Ph1
Food and Drug Administration (FDA) has granted Fast Track designation to VTX-801, Vivet’s clinical-stage gene therapy for the treatment of Wilson Disease – a rare, genetic disorder that reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurological symptoms, and potentially death.
Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Now, FDA’s CBER is setting its sights on making ambitious strides toward tackling rare diseases.
Finerenone 10 mg or 20 mg is indicated to reduce the risk of sustained estimated glomerular filtration rate (eGFR) decline, end-stage kidney disease, cardiovascular death, non-fatal myocardial infarction, and hospitalization for heart failure in adult patients with chronic kidney disease (CKD) associated with type 2 diabetes (T2D).
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