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As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Safety biomarkers account for adverse effects of a therapy under study. For use as endpoints, biomarkers must be correlated to a valid clinical outcome.
Background on the Grants Program Launched as part of the Orphan Drug Act of 1983, this program aims to encourage research and development of drugs, biologics, medical devices, and medical foods for rare diseases, defined as conditions that affect fewer than 200,000 people in the U.S. Relative to other areas of medicine (e.g.,
In the rapidly advancing field of cell therapies, Dr Jason Bock has emerged as a leader, known for his innovative approach to optimising the development process. With over 25 years of experience in therapeutics, Bock has played a pivotal role in shaping the future of cell therapies, particularly through his work at CTMC.
Where TGF- is present there is no or limited IFN- secretion by T cells and that means no PD-L1 expression within the tumor microenvironment, aka the TME. The recent integrin inhibition program that was being developed by Pliant has not yet demonstrated a positive/risk benefit profile at least in pulmonary fibrosis.
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. Could you describe the platform of macrophage biology and cell engineering used by Resolution Therapeutics in developing their cell therapies?
Cell and gene therapy (CGT) studies are rapidly gaining momentum in the Asia-Pacific region, fueled by growing patient demand and a thriving ecosystem of innovation. In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches.
There are a lot of drug development challenges that are, in some cases, unique to neuroscience, including neurological diseases, psychiatric disorders, and rare diseases affecting the nervous system. Part of the challenge is that patients present with very different kinds of clinical phenotypes, meaning the populations are heterogeneous.
These types differ in their etiology, natural history, and present distinct challenges in disease management. 2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates.
Differences in regulatory sentiments and industry sponsors’ subsequent clinicaldevelopment strategies historically restricted access based on geography. A condition of approval is the completion of a Phase 3 study designed to evaluate pheno-conversion in SOD1 mutation carriers (ATLAS study) to confirm clinical benefit.
At the congress, we were thrilled to share some of the clinical data emerging from our Phase 1 program , an ongoing study evaluating our potent and selective investigational CBL-B inhibitor, HST-1011, in patients with advanced solid tumors. The post ESMO Reflections: Glimmers of Hope with the Next Wave of I-O Therapies?
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.
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.
In most cancers, the tumour evolves by acquiring mutations that confer growth advantages or resistance to therapies. These newly formed mutated proteins are called neoantigens and they are only present on tumour cells and are not found in normal, healthy cells. This allows the cancer to evolve and develop resistance to the therapy.
How does Circio’s circVec technology aim to enhance the potency and reduce the cost of current gold-standard gene therapy? There are three major reasons for why Circio has selected AATD as its lead gene therapy program. Firstly, there have been previous AAV gene therapyclinical studies in this indication that have shown promise.
Gilead and Kite Oncology Demonstrate Growing Hematology Pipeline and Strength of Leading Cell Therapy Portfolio at EHA FOSTER CITY, Calif. & & SANTA MONICA, Calif.–(BUSINESS –(BUSINESS WIRE)– Gilead Sciences, Inc.
Real-world data (RWD) and real-world evidence (RWE) have emerged as transformational tools in addressing these hurdles, advancing rare disease drug development. MDT helps bridge the gap in preference heterogeneity to ensure that rare disease therapies are developed with the diverse needs of patients in mind.
Pfizer Presents Scientific Advancements from its Leading Oncology Portfolio at ASCO 2023 Annual Meeting will present data across its Oncology portfolio and growing pipeline, covering multiple tumor types and novel mechanisms of action at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago from June 2 through June 6.
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).
Chief Executive Officer of MorphoSys, will present at the 39 th Annual J.P. EST. Live audio of the presentation can be accessed from the Media and Investors section under Conferences on MorphoSys’ website, www.morphosys.com. The presentation as well as a replay of the webcast will also be available on MorphoSys’ website.
An important limitation of antibodies against tumour antigens is that these agents direct responses to molecular targets present on the surface of the cell. Figure 1: HLA Class I presentation of peptides derived from intracellular antigens. As described below, it also presents challenges and opportunities for TCR-based therapeutics.
Contrary to popular belief, ageing is not caused by just random wear and tear of our bodies over time but is instead caused by a discrete set of biological mechanisms that we now better understand and can target with therapies. a clinical-stage private biopharma company developingtherapies for neurological and psychiatric diseases.
. “Amgen is expanding the reach, impact and potential of our innovative therapies to personalize care for patients with historically difficult-to-treat cancers like lung cancer,” said David M. executive vice president of Research and Development at Amgen. Reese, M.D., In the U.S.,
.–( BUSINESS WIRE )– Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, today announced an oral presentation on updated efficacy, safety, and dosing management of poziotinib from Cohorts 1 and 2 of the ZENITH20 clinical trial.
Eisai and Biogen Present Preliminary Assessment of the Clinical Effects of Lecanemab Following 18 Months of Treatment in the Open-Label Extension of the Phase 2 Proof of Concept Study at 2021 Alzheimer’s Association International Conference (AAIC) . United States and virtually from July 26 to 30, 2021 (Presentation No.:
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. AskBio) I continue to work to bring innovative gene therapies to patients in need.
Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced the presentation of data from the company’s HIV clinicaldevelopment program at the 11th International AIDS Society Conference on HIV Science (IAS 2021) from July 18-21. Late Breaking Oral Presentation OALC01LB03. Abstract 2361.
Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) to TAK-994, 1 its Phase 2 investigational oral orexin agonist, which is designed to selectively target orexin 2 receptors. 2 Data from the completed Phase 2 study will be presented at a future scientific conference after the study’s conclusion.
Read the Guide FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies One frustrating aspect of traditional drug development, especially for rare disease communities, is the tempo of regulatory decisions on potential drugs. What’s a pre-print?
Bayer will present new renal and cardiovascular (CV) analyses from the comprehensive finerenone (Kerendia®) clinical trial program, including the Phase III FIGARO-DKD and FIDELIO-DKD studies, and the prespecified pooled analysis FIDELITY at the American Society of Nephrology (ASN)’s Kidney Week 2021 from 4-7 November.
Roche’s Chief Medical Officer and Head of Global Product Development. From the success of our first-in-class B-cell MS therapy OCREVUS, we are poised to continue advancing the science in MS with our new investigational BTK inhibitor fenebrutinib, and in NMOSD with the recent FDA approval of ENSPRYNG.”. Abstract Title.
We are in an era of immuno-oncology (IO) revolution with many approved therapies now available to treat a broad range of cancers. In the context of a tumour microenvironment, Tregs are often present in high numbers, preventing an effective immune response to the tumour. Efficacy at tolerated doses so far has been disappointing.
Patients are going to be randomized to receive either finerenone 10mg or 20mg or placebo on top of individually tolerated maximum labeled doses of a renin-angiotensin system (RAS)-blocking therapy like an angiotensin-converting enzyme (ACE) inhibitor or an angiotensin II receptor blocker (ARB).
Much of what we discuss I covered in my presentation, “Leveraging Genetics to Support Rare Disease Clinical Trials,” at last year’s World Orphan Drug Congress (WODC) EU. One diagnostic example that I discussed in my presentation is autism. Enrolling adequate populations will be required to advance these programs.
We ’re agitated to partake data from our robust clinicaldevelopment programs at AASLD’s The Liver Meeting ®, including the rearmost data demonstrating the positive impacts of bulevirtide for people living with HDV.”. For more than 20 years, Gilead has sought to address some of the biggest challenges in liver disease.
Nasdaq: ARDS), a biopharmaceutical company focused on the discovery and development of novel anti-infective therapies to treat life-threatening infections, today announced the Company will present at the ROTH Capital Partners 2020 MedTech Innovation Forum on Wednesday, October 28, 2020. SAN JOSE, Calif. , AR-301 (VAP).
His mother had a presentation of the disease that suggested her immune system was already on the job. But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way.
New multi-program collaboration to develop allogeneic TCR-T/CAR-T programs brings together Immatics’ allogeneic gamma delta T cell therapy platform ACTallo ® with Bristol Myers Squibb’s technologies and oncology drug development expertise. Executive Vice President, Research & Early Development, Bristol Myers Squibb. “We
New integrated analyses from our tumour agnostic Rozlytrek ® (entrectinib) clinicaldevelopment programme. Blueprint Medicines will present new data from the registrational phase I/II ARROW trial, investigating Gavreto TM (pralsetinib) for the treatment of people with RET-mutant medullary thyroid cancer.
Redig, SVP and Head of ClinicalDevelopment at HotSpot Therapeutics, as part of the From The Trenches feature of LifeSciVC The American Cancer Society for Oncology (ASCO) annual meeting is among the highlights of the clinical oncology calendar. By Amanda J.
The results were presented at The Liver Meeting Digital Experience (TLMdX), November 13–16, 2020 (Late Breaker #L02). The companies are also presenting preclinical data supporting the development of combination approaches in NASH. We are now carefully evaluating next steps together based on a thorough assessment of data.”.
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A total of 15 patients have been recruited to date in the Phase I dose escalation part of the study, all of whom were late stage and have failed conventional treatments, including several lines of rituximab-containing therapies.
ET ) to discuss the results and next steps in clinicaldevelopment of BI-1206.
Bayer is advancing its oncology R&D efforts in three scientific areas that have the potential to address unmet needs in cancer patients: next-generation Immuno-Oncology, Targeted Radionuclide Therapies and Precision Molecular Oncology. Data from all three areas of scientific focus will be showcased during this year’s meeting.
Phase 3 Data from KEYNOTE-564 to be Presented at Upcoming Medical Meeting and Submitted to Regulatory Authorities. Results will be presented at an upcoming medical meeting and will be submitted to regulatory authorities. KENILWORTH, N.J.–(BUSINESS In the U.S. About KEYTRUDA ® (pembrolizumab) Injection, 100 mg.
20% variant allele frequency reduction – At the time of analysis with > 2 year follow up the survival estimate was 100% in patients who had improvements in bone marrow fibrosis or variant allele frequency – Results were presented at the American Association for Cancer Research annual meeting.
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