Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

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Drug Discovery World

JUNE 14, 2023

The International Society for Cellular Therapy (ISCT) is a global society of clinicians, regulators, technologists, and industry partners with a shared mission to drive the translation of all cell and gene (CGT) therapies for the benefit of patients worldwide. DDW’s Megan Thomas heard from attendees about key trends in the sector.

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XTalks

FEBRUARY 10, 2023

According to a recent review published in Nature , as of April 2022 almost 1,800 active cell therapy clinical trials were listed in ClinicalTrials.gov, a 33 percent increase from 2021. In a recent webinar, Dr. Vassallo discussed the operational considerations for complex cell therapy clinical trials.

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pharmaphorum

JANUARY 25, 2023

Digital therapeutics company Woebot Health has announced enrolment of the first patient in a pivotal clinical trial to evaluate the safety and efficacy of WB001, an investigational therapeutic for postpartum depression (PPD), granted Breakthrough Device designation by the US Food and Drug Administration (FDA) in 2021.

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Delveinsight

FEBRUARY 11, 2021

However, they have only shrunk tumors in about half of patients who took them, along with chemotherapy, in the clinical trials. The first Fabry disease patient treated with Avrobio’s Plato gene therapy platform in a phase 2 trial has undergone the complete clearance of toxic kidney substrate.

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FDA Law Blog

JANUARY 2, 2022

Lenz, Principal Medical Device Regulation Expert — On December 23, 2021, CDRH released as a draft guidance, Assessing the Credibility of Computational Modeling and Simulation in Medical Device Submissions (Draft Guidance). By Adrienne R. Draft Guidance at 4. Draft Guidance at 8. Draft Guidance at 5.

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FDA Law Blog

JANUARY 2, 2022

Lenz, Principal Medical Device Regulation Expert — On December 23, 2021, CDRH released as a draft guidance, Assessing the Credibility of Computational Modeling and Simulation in Medical Device Submissions (Draft Guidance). By Adrienne R. Draft Guidance at 4. Draft Guidance at 8. Draft Guidance at 5.

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Drug Discovery World

JULY 5, 2023

It’s a brilliantly exciting time where we’re beginning to harvest the fruits of decades’ worth of breakthrough research undertaken by the world’s brightest scientists, and we’re seeing those advances in basic science translated to the clinic, where they’re making a difference in patients’ lives. Mark Garner, PhD.,

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Drug Discovery World

SEPTEMBER 28, 2022

AI-based diagnostic software and clinical trials technology proved to be two of the most attractive health investments, alongside mental health and wellness and medical imaging companies. . But has its application been as revolutionary as industry bods predicted? . The simple answer is no. The unavailability of good data .

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The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. SVP, Chief Medical Officer, Novartis Gene Therapies. “We

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Pharmaceutical Technology

SEPTEMBER 10, 2008

Before the inception of this institute, the facility was oriented towards pre-clinical toxicology and breeding of the laboratory animals. Before the inception of this institute, the facility was oriented towards pre-clinical toxicology and breeding of the laboratory animals. Dr Ivana Surovà: Biotest s.r.o.

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Roots Analysis

AUGUST 31, 2023

Type of Gene Regulation Based on the type of transcription factor ( activators / repressors ), gene regulation has been categorized into positive and negative gene regulation. Negative Gene Regulation: Repressor is a protein molecule that initiates negative regulation.

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Drug Discovery World

DECEMBER 15, 2022

Now, after nearly 35 years of research and more than a decade of preclinical progress, several different gene editing modalities are being tested in early phase clinical trials. Early phase clinical trials for gene editing therapies. Gene editing challenges and potential solutions. In one example, Choi et al.

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Drug Discovery World

NOVEMBER 23, 2023

Since then, the breadth and scope of the study has been expanded twice to investigate additional models for increasingly complex studies that aim to better inform decision-making at an earlier stage of drug discovery, gain deeper mechanistic insights to refine in vivo study assay design and reduce requirements for animal models.

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The Pharma Data

JANUARY 27, 2021

a clinical stage biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, today announces the enrollment of the first participant in Europe in its global Phase 3 clinical trial for HD. The first US subjects in the PROOF-HD trial were enrolled in October 2020.

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Drug Discovery World

FEBRUARY 15, 2023

However, over the last few decades, it has been widely accepted that interaction between the tumour and host immune response plays a critical role in regulating tumour biology and hence its progression. Over 250 clinical trials are currently in progress, and novel immuno-oncological treatments are already proving to be successful.

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The Pharma Data

NOVEMBER 10, 2020

Combined with real-world study (RWS) as well as medical theories and clinical cases, the monograph concludes, from the perspective of medical treatment, the experience of doctors in various regions of China in exploring the application of hydrogen-oxygen adjuvant therapy in mild, moderate, severe and critical patients at the baseline level.

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Delveinsight

JANUARY 18, 2021

The company was running Phase III trials of the therapy for AS; however, the therapy flunked in the trial failing to meet its primary endpoint. For instance, GTX-102 , an antisense therapy silencing the gene expression by binding to the RNA molecule behind the regulation of its expression, has also secured USFDA Fast Track Designation.

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Drug Discovery World

JULY 18, 2023

Current research is focused on designing CRISPR-enhanced CAR T cells that are resistant to immunosuppressive cytokines such as transforming growth factor-β (TGF-β) 2 , programmed cell death protein (PD-1) 3 , or other negative T cell regulators 4 (CTLA-4, LAG-3, and TIM-3), thus improving anti-tumour functions.

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Pharma Marketing Network

JANUARY 20, 2021

Assuming these findings are replicated in clinical trials, this vaccine, with its more-appealing route of administration and convenient (refrigerator) storage requirements could make an important contribution to containing the disease.

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The Pharma Data

JANUARY 11, 2021

The first co-published, peer-reviewed research paper between a commercial MPS provider and a regulator, demonstrates that data derived using CN Bio’s proprietary PhysioMimix system is appropriate for use in drug safety and metabolism applications, evidencing its enhanced performance versus standard techniques.

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FDA Law Blog

OCTOBER 11, 2022

The Act defines substantial evidence as that consisting of “adequate and well-controlled [clinical] investigations” upon which qualified scientific experts could conclude that a drug will have its purported effect. More importantly, replication is but one means to substantiate the results of a clinical investigation or scientific study.

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The Pharma Data

DECEMBER 23, 2020

Additionally, Avacta announced it submitted a Clinical Trial Application in the UK for a Phase 1, first-in-human, open label, dose-escalation and expansion study of its lead pre|CISION prodrug, AVA6000, in patients with locally advanced or metastatic selected solid tumors. Elsewhere around the world: Avacta Group – U.K.

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XTalks

OCTOBER 1, 2020

Dr. Lin received a medical degree from Shanghai Medical University and specialized in hematology after which she began working in the clinical research unit at the university, treating patients with acute leukemia. However, this has since changed, and now, transitioning between academia and industry has become more commonplace. Key Moments.

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Drug Discovery World

FEBRUARY 22, 2024

According to Medicine Discovery Catapult, these include: “Navigating the complexity of the CNS; understanding the disease pathology, and accessing predictable in vitro and in vivo cell models 2 ”. For example, it can take up to it three years to recruit enough participants for a dementia clinical trial. to £104.7m

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Drug Discovery World

MAY 10, 2023

This stage is more highly regulated and consists of both preclinical testing and clinical trials. Until recently, drug carcinogenicity assessment required two different in vivo studies, one of which was typically a two-year lifetime study in rats. billion and take over 10 years.

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Pharmaceutical Technology

APRIL 12, 2023

Current regulations for preclinical research In the UK, current regulation requires that all new medicines and vaccines be tested on animal models in preclinical studies, before being tested in humans in clinical trials. This was part of an investigation into an international primate smuggling ring.

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