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The pharmaceutical industry is currently experiencing a significant transformation. The convergence of real-world data (RWD), technology and artificial intelligence (AI) is playing a vital role in accelerating drug development. Ready to learn more about convergence of real-world data and technology for clinical trials?
Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. This will, in turn, slow the progression of, or even halt, the disease.
By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare disease research. Common goals empower change.
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.
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.
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.
Orphan drug designation is a regulatory status granted to pharmaceuticalsdeveloped for the treatment of rare diseases. It provides incentives to encourage the research, development, and approval of therapies targeting small patient populations.
Sandoz, a Novartis division, today announced progress in the late-stage clinicaldevelopment program for its proposed biosimilar aflibercept. Sandoz will begin enrolling the first patient in MYLIGHT, a clinical Phase III confirmatory efficacy and safety study, shortly 1. About aflibercept. Disclaimer. About Sandoz.
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.
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.
By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare disease research. Common goals empower change.
“We are constantly working to discover novel ways to treat this devastating disease that affects millions of people worldwide. Bayer’s established collaboration with the Broad Institute has already resulted in three clinical oncology candidates over the past decade.
Advancing drug candidates across key therapeutic areas Dr John Donello brings over 25 years of experience in pharmaceutical drug discovery, development and collaborations. Now in Phase II development at Gate for depression and other synaptopathies, zelquistinel represents a key part of his current work.
AI is being used to find the targets themselves, design the drugs to manipulate that biology, and thirdly, AI is being used to support validation of those targets and drugs as part of pre-clinicaldevelopment. With support from pharmaceutical companies, ultimately, the answer to this question should be yes.
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.
11, 2020 (GLOBE NEWSWIRE) — Lexicon Pharmaceuticals, Inc. Food and Drug Administration (FDA) for the development of LX9211 in diabetic peripheral neuropathic pain. executive vice president of research and development. “The executive vice president of research and development. “We About Lexicon Pharmaceuticals.
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.
15, 2020 (GLOBE NEWSWIRE) — Eloxx Pharmaceuticals, Inc. About Eloxx Pharmaceuticals. Eloxx Pharmaceuticals, Inc. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases. ELX-02 is in the early stages of clinicaldevelopment focusing on cystic fibrosis. WALTHAM, Mass.,
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.
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.
Holzkirchen, May 3, 2021 – Sandoz, a Novartis division, today announced progress in the late-stage clinicaldevelopment program for its proposed biosimilar aflibercept. Sandoz will begin enrolling the first patient in MYLIGHT, a clinical Phase III confirmatory efficacy and safety study, shortly 1. About aflibercept. Disclaimer.
01, 2020 (GLOBE NEWSWIRE) — Allena Pharmaceuticals, Inc. About Allena Pharmaceuticals
Allena Pharmaceuticals, Inc. is a late-stage biopharmaceutical company dedicated to developing and commercializing first-in-class, oral enzyme therapeutics to treat patients with rare and severe metabolic and kidney disorders.
Dr. Ken Kobayashi, CMO, ASLAN Pharmaceuticals, commented: “The emerging safety profile of ASLAN004 has allowed us to move directly into the expansion cohort at the highest dose, 600mg weekly, based on blinded safety data, accelerating the completion of the study.
SINGAPORE, Jan. Media and IR contacts. Forward looking statements.
17, 2020 /PRNewswire/ — CNS Pharmaceuticals, Inc.
“Since becoming a public company, our clear focus has been on advancing the clinicaldevelopment of Berubicin.
About CNS Pharmaceuticals, Inc.
In this trial the overall response rate of stable disease or better was 44%.
ASLAN believes ASLAN003 has the potential to bethe most potent oral inhibitor of DHODH currently in development for autoimmune disease, more than 30 times more potent at inhibiting the DHODH enzyme than teriflunomide.
SINGAPORE, Oct.
SINGAPORE, Oct. The company expects to share further details in early 2021.
03, 2021 (GLOBE NEWSWIRE) — EyePoint Pharmaceuticals, Inc. NASDAQ: EYPT), a pharmaceutical company committed to developing and commercializing innovative ophthalmic products, today announced that Ocumension Therapeutics, a China-based ophthalmic pharmaceutical company traded on the Stock Exchange of Hong Kong (1477.HK),
(NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases , a business unit of Chiesi Farmaceutici S.p.A.,
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.
The companies announced in January 2019 a collaboration agreement to develop, manufacture and commercialize SPR206 in the Territory. SPR206 is in clinicaldevelopment as an innovative option for the treatment of multi-drug resistant (MDR) Gram-negative bacterial infections. SHANGHAI , Jan. SHANGHAI , Jan. ” About SPR206.
Drugs developed under the Fast Track program are afforded increased access to FDA staff and may qualify for other programs to further expedite their clinicaldevelopment, such as priority review and accelerated approval. Alerts
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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. This was a turning point, igniting a wave of research and development into treatments for rare diseases that had previously been neglected.
How has nucleolar stress (NS) been linked to age-related diseases, and what approach did researchers use to investigate its toxicity? The nucleolus (and nucleolar stress) had been previously linked to aging and age-related diseases such as cancer or degeneration.
27, 2021 /PRNewswire/ — Armata Pharmaceuticals , Inc.
About Armata Pharmaceuticals, Inc.
In addition, in collaboration with Merck, known as MSD outside of the United States and Canada , Armata is developing proprietary synthetic phage candidates to target an undisclosed infectious disease agent.
15, 2020 /PRNewswire/ — Armata Pharmaceuticals , Inc. Patrick , Chief Executive Officer of Armata Pharmaceuticals.
About Armata Pharmaceuticals, Inc.
Armata is committed to advancing phage with drug development expertise that spans bench to clinic including in-house phage specific GMP manufacturing. .
02, 2020 (GLOBE NEWSWIRE) — Telix Pharmaceuticals Limited (ASX: TLX, ‘Telix’, the ‘Company’) announces it has entered into a strategic licence and commercial partnership with China Grand Pharmaceutical and Healthcare Holdings Limited (‘China Grand Pharma’) for Telix’s portfolio of Molecularly-Targeted Radiation (‘MTR’) products. .
–( BUSINESS WIRE )– Concert Pharmaceuticals, Inc. President and Chief Executive Officer of Concert Pharmaceuticals. Both of our clinical candidates represent important opportunities for new medicines that we hope will have a meaningful impact on the lives of patients.”. 5, 2020 11:00 UTC. LEXINGTON, Mass.–(
At its annual Pharma Media Day 2022, Bayer presented the latest developments in the ongoing transformation of its pharmaceuticals business, which is aimed at delivering long-term, sustainable business growth by bringing forward new options for patients.
2 The use of Xolair across allergic asthma, CIU and nasal polyps is based on its well-established efficacy and safety profile and supported by a robust clinicaldevelopment program, including 10 Phase III studies. Approximately 460,000 patients have been treated in the U.S. with Xolair since its initial approval in 2003. In the U.S.,
Human derived induced pluripotent stem cells (hiPSCs) have revolutionised research and are increasingly used for toxicology screening and disease modelling. 1-4 This approach saves time, money, and resources, and ultimately leads to safer medicines going forward to the clinic. Regul Toxicol Pharmacol RTP. 2023 Mar;139:105345.
Janssen is now moving its dengue program into clinicaldevelopment. We look forward to working with our collaborators to accelerate clinicaldevelopment.”. “As Global Head of Global Public Health R&D at Janssen Research & Development, LLC. Janssen Pharmaceutica, N.V.
13, 2021 (GLOBE NEWSWIRE) — Tiziana Life Sciences plc (Nasdaq: TLSA / AIM: TILS) (“Tiziana” or the “Company”), a biotechnology company focused on innovative therapeutics for oncology, inflammation, and infectious diseases, announces the appointment of Dr Neil Graham MBBS, MD, MPH as Chief Medical Officer. NEW YORK and LONDON, Jan.
Short will be responsible for overseeing the clinicaldevelopment of VarmX’s pipeline, including progression of its lead product, VMX-C001, into clinical proof of concept and registrational studies. Further, he advanced gene therapy clinical programmes in haemophilia B and Fabry’s disease. Notes to Editors.
SK Chemicals has shared their expertise in these diseases and validated the predicted targets and compounds through in vitro and in vivo studies. We will continue to expand our collaboration to address unmet needs in treating intractable diseases.”. About Standigm. Standigm is an AI-driven drug discovery company.
This new collaboration builds on the positive experience from previous partnerships with Gubra and combines Gubra’s proprietary streaMLine platform and expertise in obesity and peptide chemistry with Boehringer Ingelheim’s expertise in lead optimization and clinicaldevelopment. population will have obesity. About Boehringer Ingelheim.
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