Wainua (eplontersen)
Research Synopsis
- Eplontersen, branded as Wainua, is an antisense oligonucleotide designed to lower transthyretin (TTR) protein levels, primarily indicating its role in treating hereditary transthyretin amyloidosis (ATTRv) with polyneuropathy.* -
- The NEURO-TTRansform trial demonstrated eplontersen's efficacy, showing significant reductions in serum TTR levels and improved patient quality of life, solidifying its potential effectiveness in managing ATTRv.* -
- Recent research highlights eplontersen's use in combination with imaging techniques like Technetium-99m-Pyrophosphate SPECT/CT to monitor cardiac health changes in patients with hereditary transthyretin amyloid cardiomyopathy.* -
- Eplontersen has received FDA approval as of December 21, 2023, which marks a significant advance in therapeutic options for ATTRv patients, with ongoing discussions for European review.* -
- The drug's mechanism involves targeting and silencing the TTR gene, aiming to halt the progression of amyloid deposits that contribute to disease symptomatology.* -
- Clinical studies have shown eplontersen's effects on reducing amyloid burden, suggesting avenues for reversing damage to organs primarily affected by ATTR, including the heart and nerves.* -
- Comparisons between historical placebo groups indicated that patients taking eplontersen showed improved cardiac function, better left ventricular ejection fraction, and other key measurements.* -
- Ongoing trials aim to further explore eplontersen's safety, effectiveness, and potential use in broader applications for different types of amyloidosis.* -
- The emergence of eplontersen reflects a growing trend towards RNA interference therapies, which represent a transformative approach in the treatment landscape for hereditary amyloidosis and related conditions.*
Wainua (eplontersen)
RNA-targeting and gene editing therapies for transthyretin amyloidosis.
London, UK
2 hours ago
1 Received
- Transthyretin (TTR) is a protein produced mostly by the liver, but it can misfold and form harmful amyloid fibrils, leading to a condition called ATTR amyloidosis, particularly affecting the heart and nerves.
- Treatment strategies for ATTR amyloidosis involve blocking TTR production, stabilizing its structure, or disrupting the amyloid fibrils, with promising therapies like siRNA (patisiran) and antisense oligonucleotides (inotersen) already approved for certain patients.
- Current clinical trials are exploring further treatments with other siRNA and ASO formulations, as well as innovative genome editing technologies like CRISPR-Cas9, to enhance the management of ATTR amyloidosis
$100 - $150
Hourly Rate
The treatment of amyloidosis is being refined.
London, UK
2 hours ago
1 Received
- - The treatment for transthyretin (TTR)-related cardiac amyloidosis involves both supportive therapies to manage complications and disease-modifying therapies aimed at stopping the production and accumulation of amyloid fibrils.
- - Various approaches include gene silencing techniques (like patisiran and inotersen), stabilizing the TTR protein to prevent fibril formation (like tafamidis), and targeting existing amyloid deposits using experimental antibody treatments.
- - Current research focuses on several stages of amyloid development, with gene editing technologies such as CRISPR being explored, while existing antibody therapies are still undergoing early clinical trials.
$100 - $150
Hourly Rate
Population pharmacokinetic/pharmacodynamic modelling of eplontersen, an antisense oligonucleotide in development for transthyretin amyloidosis.
London, UK
2 hours ago
1 Received
- This study focuses on eplontersen, a treatment for transthyretin-mediated amyloidosis, by creating a pharmacokinetic/pharmacodynamic model to understand how it works in the body.
- Researchers analyzed data from two clinical trials to determine how factors like lean body mass and injection site influence the drug's effectiveness.
- The findings revealed that while the method of administration and body weight impacted drug exposure levels, they did not lead to significant changes in how well the treatment worked.
$100 - $150
Hourly Rate
Overview of Current and Emerging Therapies for Amyloid Transthyretin Cardiomyopathy.
London, UK
2 hours ago
1 Received
- - Recent advances in basic science have revealed the mechanisms behind amyloid transthyretin (ATTR) amyloidosis and led to the development of new targeted therapies, the first being tafamidis for ATTR-cardiomyopathy (CM).
- - Several promising agents, including acoramidis and patisiran, are being developed to stabilize or silence transthyretin (TTR) to prevent amyloid fibril formation, with some also exploring options for affordable off-label use.
- - Anti-amyloid treatments, which aim to remove existing amyloid deposits using therapies that activate the immune system, are crucial due to the high incidence of significant amyloid burden in patients with advanced ATTR-CM, with several investigational drugs
$100 - $150
Hourly Rate
Treating hereditary transthyretin amyloidosis: Present & future challenges.
London, UK
2 hours ago
1 Received
- Hereditary transthyretin amyloidosis (ATTRv) is a severe genetic disorder caused by mutations in the TTR gene, leading to lethal outcomes.
- Traditional treatments were limited to liver transplants and TTR-stabilizers, but recent advancements have introduced effective small interfering RNA (siRNA) and antisense oligonucleotide (ASO) therapies like patisiran and inotersen.
- Ongoing studies are exploring further siRNA and ASO options, as well as CRISPR-Cas9 gene editing, showcasing a transformative shift in treatment approaches for ATTRv and other gain-of-function genetic disorders.
$100 - $150
Hourly Rate
Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen.
London, UK
2 hours ago
1 Received
- - Hereditary transthyretin (ATTRv) amyloidosis is a rare and severe disease caused by the buildup of transthyretin amyloid fibrils, affecting both men and women and leading to debilitating symptoms.
- - The NEURO-TTRansform study is a pivotal phase 3 clinical trial evaluating the drug Eplontersen for treating patients with ATTRv-PN, focusing on individuals who are ambulatory with specific disease stages and genetic variants.
- - A total of 168 patients were enrolled from 15 countries, with an average age of 52.8 years, predominantly male, and most had early disease onset, emphasizing the need for effective treatments in this population.
$100 - $150
Hourly Rate
RNA Targeting and Gene Editing Strategies for Transthyretin Amyloidosis.
London, UK
2 hours ago
1 Received
- Transthyretin (TTR) is a protein primarily made by the liver, and its misfolding leads to conditions like polyneuropathy and cardiomyopathy due to amyloid fibril deposits.
- New therapies focus on stabilizing TTR or reducing its production, with drugs like patisiran, vutrisiran, and inotersen approved for treating ATTR-related conditions.
- Ongoing trials are exploring the effectiveness and safety of novel treatments, including gene-editing therapy, which offers hope for transforming ATTR amyloidosis from a fatal disease to a manageable one, though concerns about long-term effects remain.
$100 - $150
Hourly Rate
Treating amyloid transthyretin cardiomyopathy: lessons learned from clinical trials.
London, UK
2 hours ago
1 Received
- Growing awareness and advancements in diagnostic tools have significantly improved the management of amyloid transthyretin cardiomyopathy (ATTR-CM) in recent years.
- While traditional supportive therapies offer limited relief for heart failure symptoms, new disease-modifying treatments have emerged, targeting the amyloid proteins involved in the condition.
- Tafamidis is the only approved drug for ATTR-CM, with additional treatments like patisiran and inotersen showing promise for hereditary ATTR polyneuropathy, and ongoing trials exploring innovative therapies like vutrisiran and eplontersen.
$100 - $150
Hourly Rate
Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy.
London, UK
2 hours ago
1 Received
- The study evaluates the efficacy of eplontersen, a new treatment targeting hereditary transthyretin amyloidosis (ATTRv), specifically in its polyneuropathy form, through a phase 3 trial known as NEURO-TTRansform.
- Conducted across 40 sites globally, the trial included 168 adults with specific eligibility criteria, comparing results from patients treated with eplontersen to historical data from placebo participants.
- Main outcomes measured improvements in serum transthyretin levels and quality of life scores, showing a significant reduction in transthyretin levels in eplontersen-treated patients compared to placebo, indicating potential effectiveness of the treatment.
$100 - $150
Hourly Rate
Use of Technetium-99m-Pyrophosphate Single-Photon Emission Computed Tomography/Computed Tomography in Monitoring Therapeutic Changes of Eplontersen in Patients With Hereditary Transthyretin Amyloid Cardiomyopathy.
London, UK
2 hours ago
1 Received
- Hereditary transthyretin amyloid cardiomyopathy (hATTR-CM) is a severe and progressive heart disease, and this study investigates how eplontersen therapy affects the monitoring of this condition using bone scintigraphy.
- The research analyzed data from the NEURO-TTRansform trial, comparing patients taking eplontersen with a control group not on this treatment, and noted significant reductions in heart and lung volume ratios in those receiving eplontersen.
- The results indicate that eplontersen may effectively treat hATTR-CM, and the use of technetium-99m-pyrophosphate imaging could be crucial for assessing treatment effectiveness.
$100 - $150
Hourly Rate
2023 FDA TIDES (Peptides and Oligonucleotides) Harvest.
London, UK
2 hours ago
1 Received
- In 2023, the FDA approved nine TIDES, including four oligonucleotides targeting disorders like ALS, geographic atrophy, primary hyperoxaluria type 1, and hereditary transthyretin-mediated amyloidosis, all with enhanced stability and effectiveness.
- The approved peptides exhibit a variety of structures (linear, cyclic, and lipopeptides) and applications, including the first orphan drug designation for a peptide-based chemokine antagonist.
- Notably, a peptide-based treatment was approved for core symptoms of Rett syndrome, with the analysis of TIDES focusing on their chemical structure, medical targets, and potential side effects.
$100 - $150
Hourly Rate
Eplontersen: First Approval.
London, UK
2 hours ago
1 Received
- - Eplontersen (Wainua™) is an antisense oligonucleotide designed to lower TTR protein levels in patients with TTR-mediated amyloidosis (ATTR), developed by Ionis Pharmaceuticals and AstraZeneca.
- - In a phase III clinical trial, eplontersen demonstrated efficacy by reducing serum TTR levels, slowing neuropathy progression, and enhancing quality of life for patients with hereditary ATTR polyneuropathy (ATTRv-PN).
- - Following positive trial outcomes, eplontersen received FDA approval for the treatment of ATTRv-PN in the US on December 21, 2023, and is also being reviewed for approval in Europe and other regions, with ongoing trials
$100 - $150
Hourly Rate
Correction: Eplontersen: First Approval.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
A mini-review of Vutrisiran and Eplontersen in hereditary transthyretin-mediated amyloidosis with polyneuropathy.
London, UK
2 hours ago
1 Received
- * Recent advancements in treatment include new RNA interference therapies, Vutrisiran and Eplontersen, which have shown potential in improving symptoms, quality of life, and biological markers in clinical trials.
- * Ongoing research is essential to enhance understanding of these therapies and to explore new treatment options, aiming to better meet the diverse needs of patients affected by ATTRv amyloidosis globally.
$100 - $150
Hourly Rate
Evolution of Disease-modifying Therapy for Transthyretin Cardiac Amyloidosis.
London, UK
2 hours ago
1 Received
- Transthyretin cardiac amyloidosis (ATTR-CA) is a severe and progressive heart condition caused by the accumulation of misfolded transthyretin proteins in the heart.
- Recent advances have led to the development of several therapies targeting different stages of the disease, with Tafamidis being the first approved treatment that stabilizes the transthyretin tetramer to prevent further amyloid formation.
- Other emerging therapies include gene-silencing agents and anti-amyloid treatments, which aim to reduce protein synthesis or help remove amyloid deposits, offering hope for reversing the disease even in late-stage patients.
$100 - $150
Hourly Rate
RNA Interference Therapeutics for Hereditary Amyloidosis: A Narrative Review of Clinical Trial Outcomes and Future Directions.
London, UK
2 hours ago
1 Received
- Hereditary transthyretin amyloidosis (ATTR) is a serious genetic disorder caused by a mutation in the transthyretin gene, leading to harmful misfolding and buildup of amyloid in organs, resulting in severe nerve and heart issues.
- Existing treatments like liver transplantation and stabilizers have limitations, but RNA interference (RNAi) has emerged as a promising new approach, with the drug patisiran introduced in 2018 showing significant potential.
- The study reviews various RNAi-based therapies for ATTR, including patisiran, vutrisiran, inotersen, and eplontersen, focusing on their effects on patients' quality of life,
$100 - $150
Hourly Rate
Mechanisms of Action of the US Food and Drug Administration-Approved Antisense Oligonucleotide Drugs.
London, UK
2 hours ago
1 Received
- - Antisense oligonucleotides (ASOs) are single-stranded nucleic acids that specifically target RNA and have been approved by the FDA for various diseases, utilizing three main mechanisms of action: mRNA degradation, exon skipping, and mRNA function inhibition.
- - ASOs are structurally modified for stability and resistance to degradation, and examples include drugs like inotersen for hereditary transthyretin amyloidosis and nusinersen for spinal muscular atrophy.
- - The design of ASOs is largely based on mRNA sequence knowledge, making the development process quicker compared to traditional protein-targeted drugs, which enhances their potential for therapeutic applications.
$100 - $150
Hourly Rate
Recent advances in the diagnostic methods and therapeutic strategies of transthyretin cardiac amyloidosis.
London, UK
2 hours ago
1 Received
- Cardiac amyloidosis is a serious condition where abnormal amyloid fibrils accumulate in the heart, causing it to function poorly. It's mainly classified into two types: immunoglobulin light chain amyloidosis and transthyretin amyloidosis (ATTR), which comes in non-hereditary and hereditary forms.
- The rise in cases of ATTR cardiac amyloidosis is partly due to better diagnostic methods, and survival rates are improving thanks to new treatment options like tafamidis and emerging therapies currently in trials.
- New treatments such as acoramidis, eplontersen, vutrisiran, patisiran, and an experimental antibody NI006 are being tested to enhance cardiac function, with results expected in a few
$100 - $150
Hourly Rate
Effect of Eplontersen on Cardiac Structure and Function in Patients With Hereditary Transthyretin Amyloidosis.
London, UK
2 hours ago
1 Received
- Hereditary transthyretin amyloidosis (ATTRv) can lead to complications like polyneuropathy and cardiomyopathy, with eplontersen being investigated for its effects on heart health.
- A study (NEURO-TTRansform) involved 144 participants with ATTRv, some of whom had cardiomyopathy, and found that eplontersen led to significant improvements in heart function compared to a historical placebo group.
- The results showed better left ventricular ejection fraction and stroke volume in the eplontersen group, prompting further research in the ongoing CARDIO-TTRansform trial.
$100 - $150
Hourly Rate
Eplontersen: a promising breakthrough in treating hereditary transthyretin amyloidosis-related polyneuropathy.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate