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In the lifesciences, countless Black scientists, researchers and medical professionals have made groundbreaking discoveries that have significantly advanced our understanding of biology and medicine. They serve as inspiration for future generations of scientists from all backgrounds.
CF is a progressive genetic disease caused by defective CFTR proteins, which are crucial for regulating salt and water movement in cells. These mutations can vary in severity and impact on CFTR function, from complete protein dysfunction to defects in protein folding, trafficking or regulation.
The pandemic itself will also continue to spur innovations and changes such as greater shifts towards decentralized clinicaltrials and patient-centric approaches. In addition to vaccines, there are handfuls of drugs currently being evaluated in clinicaltrials for the treatment of COVID-19.
This is relevant for clinicaltrials as more regulators require evidence of efficacy in comparison to the standard of care, which is likely to be one of the blockbuster products. Several oncology products now have “blockbuster” status where sales exceed $1 billion annually. Herceptin (Trastuzumab) and Mabthera (Rituximab).
If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the lifesciences. Here is a look at some lifescience trends to expect in the coming year, including key themes and topics in the pharmaceutical, biotechnology and medical device industries to keep an eye out for in 2022.
While clinicaltrials are becoming more complex, oncology trials have generally had a greater level of complexity than trials in many other disease spaces. Despite this, the number of oncology trials worldwide has increased dramatically over the past two decades. Site Challenges in Oncology ClinicalTrials.
In an email to Pharmaceutical Technology , an NHSBT spokesperson said the centre will “support early phase clinicaltrials and preclinical work, providing a route to eventual commercial scale production”. In the past, researchers have often had to seek help from outside the UK, thus delaying clinicaltrials and patient access.
Celiac disease is an autoimmune disorder that is triggered by the consumption of gluten, a protein found in barley, wheat and rye. However, there are ongoing clinicaltrials for celiac disease to investigate potential new treatments. How does Beyond Celiac spread awareness about research and clinicaltrials for celiac disease?
Xtalks is taking a look at some of the major stories and innovations of the year in the lifesciences, many of which were driven by new, emerging and improved technologies. The year 2021 was all about continuing innovations in the lifesciences. LifeScience Innovations: Malaria and Influenza Vaccines.
Vepdegestrant, an investigational oral therapy, utilizes PROteolysis TArgeting Chimera (PROTAC) technology a mechanism that harnesses the bodys natural protein disposal system to selectively target and degrade the estrogen receptor.
Rishi Kundi, a clinical investigator at the University of Maryland Medical System, believes Symvess holds the potential to transform vascular trauma care by overcoming longstanding challenges like infection control and graft failure that have plagued traditional repair methods.
Menarini Group has announced positive topline data from the Phase III BROADWAY and TANDEM clinicaltrials, evaluating obicetrapib and the fixed-dose combination of obicetrapib with ezetimibe. Meanwhile, the TANDEM trial evaluated a fixed-dose combination of obicetrapib with 10 mg ezetimibe in a similar patient population.
Canadian clinical-stage biotech company Symvivo Corporation has developed an oral COVID-19 vaccine that entered clinicaltrials this week. The first healthy volunteer was dosed with the vaccine in Australia as part of the bacTRL-Spike COVID-19 Phase I clinicaltrial. COVID-19 ClinicalTrials.
The funding round saw participation from current investors of the company, Advent LifeSciences and Belinda Termeer. The company will utilise the Series A funds to progress its small molecule drug presently in development into first-in-human clinicaltrials for advanced metastatic breast cancer.
As Novavax awaits emergency use authorization (EUA) from the US Food and Drug Administration (FDA) for its protein-based COVID-19 vaccine NVX-CoV2373, the company shared new data this week on the vaccine’s efficacy against the South African B.1.351 1.351 variant.
Flow cytometric receptor occupancy assays are being increasingly used in preclinical and clinical studies. Both the areas of drug development and clinicaltrials are increasingly using in vitro assays to help determine the efficacy of an investigational therapeutic. What is Flow Cytometry? What is a Receptor Occupancy Assay?
By combining a monoclonal antibody against TROP2 a protein frequently overexpressed in cancers with a potent chemotherapy payload, it minimizes damage to healthy cells while attacking tumors effectively. The approval was based on the TROPION-Breast01 trial, which enrolled 732 patients with advanced disease.
Dual-function fusion proteins represent a cutting-edge approach in biopharmaceutical research, offering a promising avenue for the development of novel therapeutics in the treatment of complex diseases such as cancer and autoimmune disorders. SL-172154 entered clinicaltrials in mid-2020, following promising preclinical results.
Incytes recent announcement on the topline results from two Phase III clinicaltrials of povorcitinib in patients with hidradenitis suppurativa has stirred both hope and caution in the market. By inhibiting this protein, povorcitinib reduces the inflammatory signals that contribute to abscess and nodule formation.
It is also the first protein-based COVID-19 vaccine to be authorized in the country. Read the full articles here: Novavax Vaccine Becomes First FDA-Authorized Protein Vaccine for COVID-19. Subscribe to the Xtalks LifeScience Podcast to never miss a new episode.
Tarlatamab is an investigational drug currently undergoing clinicaltrials to assess its efficacy in patients with two distinct types of neuroendocrine cancer: SCLC and neuroendocrine prostate cancer (NEPC). Reese, executive vice president of Research and Development at Amgen, in the company’s press release.
Read more about the vaccine, including clinicaltrial results on its efficacy and safety. The treatment, an antisense oligonucleotide developed by Biogen, targets abnormal proteins arising from SOD1 mutations, which are associated with the development of ALS in some patients.
The FDA has allowed clinicaltrials assessing the injectable lenacapavir, an investigational long-acting HIV-1 capsid inhibitor in development by Gilead Inc., Lenacapavir is designed to act as an inhibitor of the HIV capsid protein, and it inhibits the virus at distinct stages (early and late) of its life cycle.
Otsuka Pharmaceutical has announced positive interim results from its Phase III clinicaltrial for sibeprenlimab. The primary endpoint of the trial was a reduction in 24-hour urine protein-to-creatinine ratio (uPCR) compared to placebo after nine months of treatment, which indicates improved kidney function.
Attributes of the drug, company and its clinicaltrials play a fundamental role in drug-specific PTSR and likelihood of approval. The drug candidate targets DNA-dependent protein kinase (DNA-PK). Merck KG, is a science and technology company. The company serves healthcare, performance materials, and lifesciences markets.
Attributes of the drug, company and its clinicaltrials play a fundamental role in drug-specific PTSR and likelihood of approval. The drug candidate targets DNA-dependent protein kinase (DNA-PK). Merck KG, is a science and technology company. The company serves healthcare, performance materials, and lifesciences markets.
Today’s pharmaceutical drug discovery process essentially relies on trial and error,” he expanded. “To To find potential drug candidates, disease-associated protein targets are tested against a small pool of less than ten million distinct chemotypes synthesised at great effort and expense over the past 100 years.
Mobasher Xtalks Clinical Edge is a magazine for clinical research professionals and all who want to be informed about the latest trends and happenings in clinicaltrials. The mechanism of action of our BTK CDAC allows for complete degradation of the BTK protein and has distinct advantages over current BTK inhibitors.
The companys pipeline includes MET-097i, currently in Phase IIb trials, and MET-233i, in early-stage clinicaltrials. The companys approach combines small molecule therapies and targeted protein degradation. Maze Therapeutics, Inc. IPO Date: January 31, 2025 Initial IPO Price: $16.00
As DMD is caused by a lack of functional dystrophin protein, which supports and strengthens muscle fibers, therapies designed to treat this rare genetic disorder have focused on exon skipping to avoid the area of the dystrophin gene that is mutated and produce a nearly full-length dystrophin protein.
The US Food and Drug Administration (FDA) recently granted approval for the Bruton tyrosine kinase inhibitor (BTKi) Brukinsa (zanubrutinib) based on the results from two Phase III clinicaltrials. Brukinsa is now approved to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
The world of neuroscience trials is rapidly evolving, presenting new challenges and opportunities for researchers and clinicians. The worldwide market for neurology clinicaltrials stood at a valuation of $5.24 There is currently a heightened emphasis on imaging and liquid biomarkers in neuroscience clinicaltrials.
Medicxi is a prominent international lifesciences investment firm that invested in over 60 portfolio companies such as Versanis Bio, Rivus Pharmaceuticals, Capella Bioscience and more. One of Medicxi’s portfolio companies called MiroBio is set to be acquired by Gilead Sciences for approximately $405 million in cash.
It occurs when a genetic error causes the overproduction of a protein called CSF1, which attracts inflammatory cells to the joint, often causing pain, swelling and reduced mobility. Looking ahead, Deciphera is exploring additional clinicaltrials and potential collaborations in the TGCT space.
Convidencia’s injectable version was granted World Health Organization (WHO) Emergency Use Listing (EUL), and its inhalant counterpart, currently in phase three clinicaltrials, has shown enormous promise. “It is programmed and coded for the production of the full-length spike protein of the coronavirus.
Reddy says one of LabCorp’s groundbreaking tests, OmniSeq INSIGHT, is an “all-in-one test that includes comprehensive genomics and immune profiling from a single tissue sample to identify all FDA-approved therapeutics, confirm immunotherapy eligibility for a patient, and identify appropriate nearby clinicaltrials.”. About the author.
Biogen and Ionis Pharmaceuticals have demonstrated that their experimental drug, BIIB080 (IONIS-MAPT), fulfilled the primary goal of safety and tolerability in the Phase Ib multiple ascending doses (MAD) clinicaltrials in mild Alzheimer’s disease patients. Details on the pipeline are still very thin.
The mission was first put forward during the UK’s G7 Presidency of June 2021, with support from representatives of the lifesciences industry. Messenger RNA vaccines contain nucleic acids that code for a specific protein, or target antigen, related to a virus or disease. The UK Government is behind this effort.
SMA is caused by mutations in the survival motor neuron 1 ( SMN1) gene, which along with the SMN2 gene, encode the SMN protein. It is administered as a one-time IV administration and expression of the SMN protein from the gene improves muscle movement and function, and ultimately survival. Zolgensma Gene Therapy: START Trial.
Abrysvo is an unadjuvanted vaccine and is composed of two preF proteins selected to optimize protection against RSV A and B strains. “A The genetic material of RSV (respiratory syncytial virus) encodes 11 proteins. The F-protein facilitates the fusion of the virus with the membrane of the host cell.
Number of on-going clinicaltrials: 14. We currently have 14 clinicaltrials in progress in the US, of which, four are joint clinicaltrials with Merck, Roche, and BMS,” says Dr Yang. “We Locations: 3 (US HQ, Korea office, Korea R&D center). Number of employees: 90+ (as of Oct. Creation: Jan.
The pre-clinical and clinical data they showcased at the meeting indicate that their cytokine immunotherapies are safe, potent and work for longer than other cytokine drugs for cancer. Based on these findings, Cytimmune will be moving these candidates into early clinicaltrial testing. What Are Nanomedicines?
Hympavzi reduces the activity of TFPI, a naturally occurring anticoagulation protein. Hympavzi is the first anti-tissue factor pathway inhibitor (anti-TFPI) approved in the US for hemophilia A or B, and it’s also the first hemophilia therapy to be administered via a pre-filled auto-injector pen.
The global trials are assessing the safety and efficacy of the vaccine in individuals aged 18 years or over from diverse racial and geographic groups who are healthy or have stable underlying medical conditions. Oxford-AstraZeneca COVID-19 Vaccine Induces Immune Responses in Early ClinicalTrials.
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