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Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
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.
This significance and focus are ever more apparent when the starting cellular material is imperative to drug product success. Within cell therapyclinical trials, apheresis collections contribute to the specific constituents of the given therapy.
As a result, drug developers make better decisions more quickly (removing 50% of study timeline whitespace) to bring new therapies to market faster. As risks are identified, customized action plans are created and provided to clinical teams to keep studies on track.
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?
Less clear is whether we can productively and safely inhibit TGF- activity at all given the toxicity issues associated with TGF- inhibition. We recently viewed quite positive updates from Scholar Rock who have a TGF--1 antagonist in the clinic. So, you cannot link antagonist targeting these two pathways together, at least in the TME.
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.
Additionally, for illustrative reasons this is geared towards a single target / product focus vs. broader platform diligence, though many of these mental models will apply for selecting targets and indications for a platform. with gene editing or gene therapy, enzyme replacement therapy), agonism (e.g., in liver, in CNS)?
Cell and gene therapies for treating multiple myeloma, such as CAR-T, have resulted in breakthrough health outcomes for patients. In the last decade, approval of cell and gene therapy (CGT) products has increased significantly.
A clinicaldevelopment plan — a comprehensive strategy for developing an investigational product through regulatory submission — is a critical component of drug development and helps ensure that new therapies are safe, effective, and of high quality.
The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.
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.
Real-world data (RWD) and real-world evidence (RWE) have emerged as transformational tools in addressing these hurdles, advancing rare disease drug development. Developing effective ECs requires more than just matching clinical trial inclusion and exclusion criteria within the RWD source.
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. Of those participants who transitioned back to injectables, the median duration of oral therapy was 51 days.
The highly dynamic biotech industry has a core need to remain as flexible and agile as possible across the spectrum of clinicaldevelopment activities. One primary way these companies create a nimble and adaptive environment is by outsourcing some portion of clinicaldevelopment functions.
Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.
The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.
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.
Brevig, Senior Regulatory Device and Biologics Expert — On December 7, 2022, FDA’s Center for Biologics Evaluation and Research (CBER) and the Office of Tissues and Advanced Therapies (OTAT) held a town hall to answer questions related to cell therapy and tissue-engineered products chemistry, manufacturing, and controls (CMC).
Lewis, Senior Regulatory Device & Biologics Expert — On October 20, 2023, FDA announced the availability of the final guidance authored by CBER titled “Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies.” A considerable roadblock in the development of RMT products is a lack of regulatory predictability.
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).
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.
Baynes, head of Global ClinicalDevelopment (GCD) and Chief Medical Officer, Merck Research Laboratories (MRL), will be retiring from Merck in July. He also led the expansion of Merck’s research and development in China and Japan. “It Eliav Barr, senior vice president, Global ClinicalDevelopment.
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). In the U.S.,
A large majority of drug developers utilize FSP or hybrid FSP/FSO models, half are “heavy users” Heavy users: Those that use FSP or hybrid FSP/FSO models for most of their clinicaldevelopment outsourcing. Heavy users leverage these models for nearly three-quarters of their outsourcing.
Merck will assist with preclinical development and has the option to exclusively develop, manufacture and commercialize the candidate following Phase 1. Under the agreement A2 will receive an upfront payment, and will be eligible for opt-in and milestone payments, plus royalties on sales of any approved product.
This reduction in cost does not equate to a compromise in quality or efficacy, as biosimilars undergo rigorous analytical and functional characterization to demonstrate biosimilarity to the reference product. biosimilars are often priced slightly lower than their reference products, limiting their potential for cost savings.
15, 2020 /PRNewswire/ — Thermo Fisher Scientific announced the launch of its Gibco CTS Rotea Counterflow Centrifugation System, a modular, closed cell therapy processing system that enables scalable, cost-effective cell therapydevelopment and manufacturing.
CARLSBAD, Calif. ,
– Second Product Candidate Expected to Enter Clinic in First Half of 2021. Preclinical Data Underscore Treatment Potential for PBFT02 in Frontotemporal Dementia with Granulin (GRN) Mutations, a Devastating, Progressive Disorder Impacting Adults with No Approved Disease-Modifying Therapy Options. PHILADELPHIA, Jan.
Working alongside KSQ will facilitate smart drug discovery and development of what we hope will be transformative new therapies for patients with intractable forms of cancer.”. KSQ is also eligible to receive tiered royalties on net sales of each approved product. sales for that product. sales for that product.
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
Boehringer Ingelheim and Veeva Systems (NYSE: VEEV) today announce Boehringer Ingelheim’s selection of Veeva Development Cloud to drive end-to-end processes and seamless information sharing enterprise wide for greater speed in the delivery of innovative therapies. About Veeva Systems. About Boehringer Ingelheim.
1,2 Many, however, do have dysfunctional proteins at their core, and for these diseases, a new option is being explored: autophagy-boosting therapies. Gene therapy, of course, also holds great promise. Most of these conditions are genetic in origin and the majority have no effective treatment.
For clinical research professionals, this journey often involves reconciling differing feedback from regulatory agencies, each with unique standards, priorities, and expectations. Despite these efforts, variations between regulatory agencies remain, often resulting in divergent feedback that can complicate clinicaldevelopment strategies.
How does GigaGen’s single-cell discovery and development platform differ from traditional methods of producing polyclonal antibody therapies? Current methods for producing pAb therapies rely on regular plasma donations for their development.
As part of its broad development program, BeiGene expects to work with the NHSA for potential NRDL inclusion in future expanded indications for these medicines.
As part of its broad development program, BeiGene expects to work with the NHSA for potential NRDL inclusion in future expanded indications for these medicines. .
These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. Join the club.
Boehringer Ingelheim and the Agency for Science, Technology and Research (A*STAR) today announced a global licensing agreement under which Boehringer Ingelheim will obtain exclusive worldwide rights to research, develop and commercialize products based on a panel of innovative, tumor-specific antibodies from A*STAR.
This approach has produced a broad range of approved and investigational products. Some examples of new products incorporating TCR and TCR-like technologies are below. Engineering soluble T-cell receptors for therapy. Cancer Therapy With TCR‑Engineered T Cells. References Robinson R, McMurran C, McCully M, Cole D.
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. His mother had a presentation of the disease that suggested her immune system was already on the job.
Draft guidance on potency assays for CGT products garners extensive stakeholder input Late last year, the FDA published a draft update to its 2011 guidance on potency assays for cell and gene therapyproducts, unveiling a major shift in approach to the issue. How does “potency” apply to cell and gene therapy (CGT) products?
Differences in regulatory sentiments and industry sponsors’ subsequent clinicaldevelopment strategies historically restricted access based on geography. was withdrawn, rendering it unapproved for patient use, based on negative review comments by the Committee for Medicinal Products for Human Use. The MAA file in the E.U.
– Designation is based on results from the EMPEROR-Preserved phase III trial, which established Jardiance as the first therapy to show statistically significant improvement in heart failure outcomes in adults with heart failure with preserved ejection fraction. vice president, ProductDevelopment, Lilly.
Based on the Phase 2 study findings to date, marstacimab may have the potential to offer improved bleed control via subcutaneous injection and potentially eliminate the need for prophylactic factor replacement, providing an enhanced treatment option compared to factor replacement therapy.”. About Pfizer Rare Disease. Pfizer Inc.:
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