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A new study provides a list of the wildlife species present at the market from which SARS-CoV-2, the virus responsible for the COVID-19 pandemic, most likely arose in late 2019. The study is based on a new analysis of metatranscriptomic data released by the Chinese Center for Disease Control and Prevention (CDC).
The landscape of genetic medicine is undergoing a profound transformation, driven by innovative approaches that challenge the traditional, disease-specific paradigms. My transition to biotech and rare diseases was deeply personal – my son was diagnosed with Duchenne muscular dystrophy in 2020. “My
A broadened clearance for Reblozyl in myelodysplastic syndromes should help Bristol Myers offset the looming loss of revenue from top-selling medicines set to soon lose market exclusivity.
The deal “fits like a glove” for BioMarin’s business, according to an analyst who also thinks there could be a “valuable” market opportunity for Inozyme’s main drug.
The pharmaceutical industry is undergoing a significant shift, with emerging markets offering the next growth opportunity. This growth is driven by several factors, including the increasing prevalence of chronic diseases, cost-effectiveness, and patent expirations of branded drugs.
Rallybio is headed into a mid-stage trial with enough cash to last it through 2026, but biotech's down market has made the company's journey difficult.
With new medications on the market or in the works for Alzheimer's disease and other kinds of dementia, a new study suggests that getting the diagnosis needed to access these new treatments may depend on where you live.
As we delve into the intricate world of biosimilar market dynamics, we’ll explore how these complex molecules are reshaping treatment paradigms across diverse patient populations. ”[1] The global biosimilars market is experiencing exponential growth, with projections indicating it will reach $69.4 from 2020 to 2025[1].
Multibillion-dollar buyouts from Bristol Myers Squibb and Gilead could yield new drugs for brain and liver diseases, while a new cell therapy may reach market.
For years, many manufacturers have assumed that pre-market physician education was not strictly necessary unless their brand was the first to market or had a novel mechanism of action. With the advent of precision medicine, however, ongoing disease state education has become increasingly critical.
5, 2025 -- Hopes that GLP-1 drugs such as Ozempic and Wegovy could help slow Parkinson's disease have taken an hit.A new study found that a drug in the same class called exenatide, marketed as Byetta, had no effect on slowing the. WEDNESDAY, Feb.
The search for effective treatments for neurodegenerative diseases like Parkinson’s disease has long been hindered by the brain’s complexity and the absence of adequate models for drug discovery. “For diseases like Parkinson’s, it’s more than sufficient,” he explains.
Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.
The integration of AI in drug discovery is revolutionizing the way researchers approach the development of new treatments for various diseases. The integration of AI in drug discovery is revolutionizing the way researchers approach the development of new treatments for various diseases. Traditional.
On the last day of February every year is Rare Disease Day , a dedicated day to celebrate the rare disease community, including patients, families, caregivers, clinicians, researchers, regulators, and more. The goal is to build community, share ideas and resources, and advance research and innovation.
These innovations have started to shift industry perceptions, positioning AI as a transformative tool that could alter how drugs are developed, tested, and brought to market. Their work focuses on creating ‘digital twin generators’ – AI-driven models that predict how a patients disease may progress over time.
Placebo arms, in particular, create ethical and logistical hurdles, especially in areas like rare disease and oncology. They are patient-specific outcome predictions, generated using disease-specific machine-learning models trained on large, longitudinal clinical datasets. Digital twins offer a way forward.
In preparation for World Orphan Drug Congress Europe, we interviewed Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease, and Derek Ansel, MS, LCGC, Vice President, Therapeutic Strategy Lead, Rare Disease, to hear their insights into the current progress in rare disease research and their hopes for 2025.
These new findings reinforce the company’s growing leadership in rare hematologic diseases, with a focus on transformative treatments for immune thrombocytopenia (ITP) and hemophilia. Rilzabrutinib is currently under regulatory review in major markets including the United States, European Union, and China for the treatment of ITP.
This approach not only helps maintain market exclusivity but also ensures a steady revenue stream for pharmaceutical companies. Understanding the Pharmaceutical Market Dynamics The pharmaceutical industry is a complex ecosystem where branded drugs and generics coexist, each playing a vital role in patient care and market dynamics.
Tobolowsky — The Rare Pediatric Disease Priority Review Voucher program has had a bit of a tumultuous history in its 12 short years of existence. Over 50 rare pediatric disease priority review vouchers have been awarded to date. We heard and felt the anxiety from many of our clients about the uncertainty this caused.
While new gene therapies for the blood disease grabbed headlines at ASH, Pfizer presented fresh data for an oral drug meant to build on its marketed therapy Oxbryta.
While individual rare diseases affect populations that are small in numbers, collectively they impact millions globally, posing significant health and research challenges. Real-world data (RWD) and real-world evidence (RWE) have emerged as transformational tools in addressing these hurdles, advancing rare disease drug development.
Unlocking European market access for rare disease therapies The European orphan drug market presents a significant opportunity for biotech firms. Learn more about early access programs (EAPs), and how they are emerging as a critical strategy for successfully launching rare disease therapies in this complex and diverse market.
Here we explore the evolution and impact of market exclusivity policies in the EU and US, highlighting their role in fostering innovation and accessibility in rare disease treatment. Market exclusivity for orphan drugs traces back to the early 1980s in the United States, with the landmark Orphan Drug Act of 1983.
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.
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. Click to enlarge Six AI and machine learning considerations for effective clinical trial management 1.
Similarly, the December 2024 draft guidance reiterates prior positions that confirmatory trials should generally be underway at the time the marketing application is submitted but also states that this recommendation becomes a requirement by the time of approval except in limited circumstances. rare disease settings).
Credit: Aston University Aston University and local industry to develop technology to convert organic material into commercially valuable products Sawdust, diseased trees and dried chicken litter among what can be transformed into sustainable bioproducts […] Aston University is to receive £1.8
CNS Market Outlook, 2024 This paper assesses the current state of the broad CNS market, reviewing key expectations for 2024. It addresses Alzheimer’s disease, biomarkers, and advances in psychiatry in detail. It also provides a high-level look at the pain market and the evolving regulatory environment.
A number of areas represent significant opportunities for Antibody Drug Conjugates (ADCs) beyond oncology, leveraging their ability to deliver therapeutic agents specifically to diseased cells or tissues while minimising off-target effects. Oncology also offers high returns and the opportunity to address severe, life-threatening conditions.
In fact, it has a whole page dedicated to “CDRH Innovation,” which states that CDRH “is committed to advancing public health by helping to bring innovative technologies to market.” Manufacturers can expect increased interaction with the review team and prioritized review of the marketing submission.” So what can be done?
“I was increasingly drawn to understanding the underlying biology of disease at a molecular level – the ‘why’ – and how we might harness science to create better medicines for patients,” Lepore says. Today’s broad-spectrum drugs often miss the mark, especially for chronic diseases like COPD or heart failure.
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Monitoring biomarkers can help assess changes in a disease, its level of expression, or the extent of its progression.
Novartis Finalizes Acquisition of Regulus Therapeutics, Strengthening Its Renal Disease Portfolio with Promising ADPKD Therapy Novartis AG, a leading global pharmaceutical company, has officially completed its acquisition of Regulus Therapeutics Inc. , a biotechnology firm known for its expertise in microRNA-targeting therapies.
The next-generation sequencing (NGS) market is experiencing rapid growth, which is expected to continue for the foreseeable future. Research by Markets and Markets projects the market to grow from $12.13 Next generation Sequencing Market Size, Share | 2022 – 2026 | MarketsandMarkets [Internet]. References: 1.
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Inhaled combination products (ICP) have emerged as a significant advancement in the treatment of respiratory diseases such as asthma, chronic obstructive pulmonary disease (COPD), and other pulmonary conditions. The goal of ICPs is to improve the quality of life for patients suffering from chronic respiratory diseases.
Rare Diseases: With a growing number of treatments emerging for rare diseases, companies in this space are seeking legal expertise to safeguard their investments and protect their market share. What do these trends say about the future of the pharmaceutical industry? And what can companies do to stay ahead of the curve?
no comparator arm), FOENIX-CCA2 was not capable of establishing improvement on time-to-event efficacy endpoints such as PFS or OS and, without an appropriate comparator, it is not possible to determine if the observed effect is attributable to LYTGOBI or to other factor(s), such as the natural history of the disease.
Pharma Marketing Trends: Key Takeaways from Digital Pharma East 2024 swheeler Mon, 08/26/2024 - 14:05 Thu, 09/26/2024 - 13:00 Resource Type Webinar Stephanie Butler Promotion Start Tue, 08/27/2024 - 14:15 Promotion End Thu, 02/27/2025 - 14:15 Duration 60 minutes Did you miss the buzz at Digital Pharma East 2024?
Given macro healthcare influences (eg, economic uncertainty, environmental changes) and the numerous available treatments for major diseases, drug developers may need to reassess their therapeutic strategies. This is especially relevant with today’s heavier focus on enhancing personalised medicine via broader emerging scientific findings.
By enabling a more direct assessment of drug interactions with human tissue, bioprinting can streamline the path from lab to market. They help scientists to better understand organ function and disease mechanisms. These are used for drug testing, disease modelling, and personalised medicine. Tissue and organ models.
Its therapeutic platform aims to go beyond traditional symptom management to directly tackle the genetic roots of diseases. The company’s lead candidate, NCX-L2 , is specifically designed to slow or even halt the progression of Parkinson’s disease—a neurodegenerative condition that currently has no disease-modifying therapies on the market.
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