Clinical Trials, Containment and In-Vivo - Clinical Research Informer

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

APRIL 24, 2023

Melanie Blank, clinical team leader for General Medicine Branch 1 at the US Food and Drug Administration’s (FDA) Division of Clinical Evaluation and Pharmacology/Toxicology (DCEPT), the agency is seeing one or two new applications coming in every week for new gene therapies for different diseases.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

Celltrion Group announces positive top-line efficacy and safety data from global Phase II/III clinical trial of COVID-19 treatment candidate CT-P59

The Pharma Data

JANUARY 12, 2021

INCHEON, South Korea–( BUSINESS WIRE )– Celltrion Group today announced top-line results from its randomised, double-blind, and placebo controlled global Phase II/III clinical trial of CT-P59, an anti-COVID-19 monoclonal antibody treatment candidate. days quicker compared to placebo. days; 95% C.I, days; 95% C.I,

Clinical Trials

Clinical Trials Clinical Trials Trials In-Vivo

Using PK Modeling and Simulation to Plan Studies for Pediatric Patient Populations

Camargo

APRIL 28, 2021

Conducting a clinical study in a pediatric patient population requires careful planning, as providing a benefit to a child without causing harm is a weighty responsibility. First Steps Toward Initiating Pediatric Clinical Studies. Data Collection from Preclinical and Adult Studies.

Clinical Trials

Clinical Trials Clinical Trials In-Vivo Drugs

The regulatory round-up: Eight key FDA decisions

Drug Discovery World

APRIL 4, 2023

The approval is for endometrial cancer that has progressed on or following a prior platinum-containing regimen in any setting and are not candidates for curative surgery or radiation. The Food and Drug Administration (FDA) in the US has made several key drug decisions over the last few weeks. Here’s a summary.

FDA Approval

FDA Approval In-Vivo Gene Editing Drugs

Are organ-chips the future of preclinical research?

Drug Discovery World

OCTOBER 27, 2023

Organ-chips combine cell culture with microfluidics to emulate the biological forces of different organ tissues and/or disease states, allowing pharmaceutical researchers to determine a drug candidates’ efficacy and toxicity ahead of clinical trials. Could organ-chips replace other research methods? Not yet, according to Ewart.

Research

Research Gene Therapy In-Vivo In-Vitro

In the News: October Regulatory and Development Updates

Camargo

NOVEMBER 11, 2020

Each month, Camargo’s “In the News” series highlights important changes and advancements in the regulatory and development space and explores how those changes could impact your program. Unpacking the (Black) Box: Antares Licenses Urology Product with Boxed Warning. of hyponatremia, or low blood sodium levels. In October, the U.S.

Development

Development Bioequivalency Generic Drugs Production

Untangling the Complexities of Cell and Gene Therapy Clinical Trials: A Supply Chain Perspective

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.

Clinical Trials

Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain

Overcoming diverging regulatory expectations to bring CGTs to market

Drug Discovery World

AUGUST 14, 2023

For example, the FDA’s Center for Biologics Evaluation and Research (CBER) 3 , which regulates biological products for human use, differentiates between in vivo gene therapy (new genes are inserted into the body to target cells) and ex vivo gene therapy (cells are modified outside the body and then administered back into the patient).

Gene Therapy

Gene Therapy Marketing Gene DNA

Lysogene Reports Positive Biomarker Data With LYS-SAF302

The Pharma Data

DECEMBER 27, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869).

Gene Therapy

Gene Therapy Clinical Trials Clinical Trials In-Vivo

OSE Immunotherapeutics Presents OSE-230 as a Novel Agonist Monoclonal Antibody Therapy Triggering Resolution of Chronic Inflammation

The Pharma Data

OCTOBER 29, 2020

The oral presentation entitled “ Agonist anti-ChemR23 mAb blunts tissue neutrophil accumulation and triggers chronic colitis inflammation resolution ” shows efficacy results for OSE-230 in chronic inflammatory preclinical models and ex vivo human models. 7 therapies.

Antibody

Antibody In-Vivo Development Clinical Trials

Fighting viruses is as easy as breathing

The Pharma Data

APRIL 8, 2022

Data from the research were recently included in the company’s Investigational New Drug (IND) application to the FDA to initiate a Phase 2 clinical trial for COVID-19. Data from the research were recently included in the company’s Investigational New Drug (IND) application to the FDA to initiate a Phase 2 clinical trial for COVID-19.

Immune Response

Immune Response In-Vivo Genetic Analysis Research

Gamida Cell Provides Regulatory Update on Biologics License Application for Omidubicel

The Pharma Data

DECEMBER 13, 2020

The company believes the clinical comparability requirement will be met if the time to neutrophil engraftment in patients from the company’s ongoing expanded access program (EAP) using omidubicel produced at Gamida Cell’s planned commercial manufacturing sites is consistent with the results achieved in the Phase 3 clinical trial.

Licensing

Licensing Licensing Clinical Trials Clinical Trials

Company Spotlight: Cue Biopharma Innovates in the Immunotherapy Space with its Immuno-STAT Platform

XTalks

AUGUST 17, 2020

Cue Biopharma is a Cambridge, Massachusetts-based clinical-stage biotechnology company that is focused on developing injectable immune biologics using its proprietary Immuno-STAT (Selective Targeting and Alteration of T cells) platform. Cell-Based Immunotherapy vs. Immune Biologics. cells, receptors) to treat disease.

Protein

Protein Engineer Immune Response In-Vivo

Kronos Bio Announces Publication of Preclinical Study Results for Investigational CDK9 Inhibitor KB-0742 in Cell Chemical Biology

The Pharma Data

OCTOBER 21, 2020

Company is on t rack to s ubmit an IND in the fourth quarter of 2020 and i nitiate a Phase 1 /2 clinical trial for advanced solid tumors in 2021. Research s howed KB-0742 i nhibited a ndrogen r eceptor -d ependent a ctivity and r educed t umor g rowth in c astration -r esistant p rostate c ancer. SAN MATEO, Calif., SAN MATEO, Calif.,

In-Vivo

In-Vivo In-Vitro Bioavailability Clinical Trials

AvantGen Announces Licensing of Its Anti-SARS-CoV-2 Antibodies to IGM Biosciences for COVID-19 Therapy Development

The Pharma Data

JANUARY 11, 2021

Our antibody clones in their original IgG format have shown potent neutralization activity in in vitro assays and, in the case of our lead clone, in an in vivo animal model. Headquartered in Mountain View, California, IGM Biosciences is a clinical-stage biotechnology company focused on creating and developing engineered IgM antibodies.

Antibody

Antibody Licensing Licensing Development

ImmunityBio’s hAd5 T-Cell COVID-19 Vaccine Candidate Shows Complete Protection of Airways in Non-Human Primates

The Pharma Data

DECEMBER 10, 2020

.–( BUSINESS WIRE )– ImmunityBio , a privately-held immunotherapy company, today announced its COVID-19 vaccine candidate protected nasal and lung airways of non-human primates against coronavirus (SARS-CoV-2) in a challenge study. This blocking of viral replication was observed in both the lung and nasal passages.

Vaccination

Vaccination Vaccine Immune Response Protein

Novartis announces lift of partial clinical trial hold and plans to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with SMA

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

Clinical Trials

Clinical Trials Clinical Trials Gene Therapy Trials

Delivering on the promise of gene editing

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.

Gene Editing

Gene Editing Gene In-Vivo Genome

Editas Medicine Announces the FDA has Cleared Initiation of the EDIT-301 Clinical TrialEDIT-301 is in development as a best-in-class, durable medicine for people living with sickle cell disease

The Pharma Data

JANUARY 10, 2021

Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. The Company is required to develop and submit to the FDA an improved potency assay prior to enrolling the efficacy phase of the RUBY trial.

Medicine

Medicine Development Clinical Trials Clinical Trials

Neuronascent Announces Publication of Pre-clinical Results of Alzheimer’s Disease Clinical Candidate, NNI-362 in SCR&T Journal

The Pharma Data

JANUARY 19, 2021

a privately-held neuron regeneration therapeutics company, today announced the publication of results of their novel pharmacotherapy: NNI-362 to allosterically stimulate neural regeneration in human cultures and in vivo models of aging and disease. CLARKSVILLE, Md., 20, 2021 (GLOBE NEWSWIRE) — Neuronascent Inc. , About NNI-362.

In-Vivo

In-Vivo Clinical Trials Clinical Trials In-Vitro

Cryomacs Freezing Bags – A Critical Component in the Successful Delivery of Biological Samples and Therapies

Roots Analysis

SEPTEMBER 18, 2023

While some biobanks ( especially non-clinical ones ) use mechanical freezers for storage, liquid nitrogen is one of the most reliable cryostorage mediums. Due to the infrastructure available for processing, freezing and storing of these container systems, blood bags are the preferred container for cell therapy biopreservation.

Engineer

Engineer Containment In-Vivo Production

Gene Switch: A Novel Platform for Switching Genes On and Off

Roots Analysis

AUGUST 31, 2023

Further, the transcription factors contain two molecular domains, the DNA binding domain and the activation domain. Moreover, some transcription factors contain additional domains, such as ligand binding domain in order to interact with chemical signals. Figure below presents an overview of the mechanism of action of gene switch.

Gene

Gene Gene Therapy Gene Expression Regulation

Re-assessing the risks of drug-induced arrhythmias during drug discovery

Drug Discovery World

AUGUST 15, 2023

However, development pipelines across multiple therapeutic areas are still not meeting the anticipated clinical outcomes that are needed for bringing new and cost-effective drugs to patients. Such techniques, if routinely deployed in drug discovery, may enable improved decision making in predicting serious adverse cardiovascular events.

Drugs

Drugs In-Vivo Development Research

Necessity Is the Mother of Innovation: Key Trends and Predictions from Biotech Showcase Digital After an Unprecedented Year That Drove Momentous Changes in Healthcare

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.

Vaccination

Vaccination Vaccine In-Vivo Genetics

Reflecting on PEGS Europe 2023

Drug Discovery World

NOVEMBER 30, 2023

DDW’s Megan Thomas attended PEGS Europe 2023 in Lisbon, Portugal, from Tuesday 14 November to Thursday 16 November 2023. The company’s goal is to accelerate functional characterisation and shortenoptimisation time in antibody discovery and t-cell workflows. GSK has developed high-throughput mammalian and E.

Antibody

Antibody Protein Engineer In-Vivo

TLC Announces Full Patient Enrollment in EXCELLENCE Trial of TLC599 for Osteoarthritis PainOn-schedule completion of enrollment despite COVID-19 pandemic; unique trial design evaluates safety and efficacy of single & repeat doses

The Pharma Data

DECEMBER 27, 2020

In these unprecedented times, we are especially grateful for the dedication of our research site staff and trial participants, whose commitment to EXCELLENCE facilitated the advancement of our lead program,” said George Yeh, President of TLC. SOUTH SAN FRANCISCO, Calif. and TAIPEI, Taiwan, Dec. About TLC599.

Trials

Trials Clinical Trials Clinical Trials Containment

Addressing the developability gap in pharma

Drug Discovery World

AUGUST 4, 2022

A fundamental aspect of biopharmaceutical drug development is the need for the data submitted in an Investigational New Drug (IND) to match the data containing in the Chemistry, Manufacturing and Control (CMC) section of a Biologics License Application (BLA). Even for regular mAbs there are challenges. Challenges.

Development

Development Protein Manufacturing Drugs

Sanofi continued its growth trajectory. Strong increase in Q1 2021 business EPS(1) at CER

The Pharma Data

APRIL 27, 2021

Strong increase in Q1 2021 business EPS ( 1) at CER. Q1 2021 sales increase of 2.4% at CER driven by growth drivers Dupixent ® and Vaccines. Specialty Care sales grew 15.3%, due to strong Dupixent ® performance (+45.6% to €1,047 million) and oncology launches. General Medicines core assets grew 4.4%, while GBU sales were down 3.8%.

Sales

Sales Vaccination Vaccine Medicine

Clinical Research Informer

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

Celltrion Group announces positive top-line efficacy and safety data from global Phase II/III clinical trial of COVID-19 treatment candidate CT-P59

Trending Sources

Using PK Modeling and Simulation to Plan Studies for Pediatric Patient Populations

The regulatory round-up: Eight key FDA decisions

Are organ-chips the future of preclinical research?

In the News: October Regulatory and Development Updates

Untangling the Complexities of Cell and Gene Therapy Clinical Trials: A Supply Chain Perspective

Overcoming diverging regulatory expectations to bring CGTs to market

Lysogene Reports Positive Biomarker Data With LYS-SAF302

OSE Immunotherapeutics Presents OSE-230 as a Novel Agonist Monoclonal Antibody Therapy Triggering Resolution of Chronic Inflammation

Fighting viruses is as easy as breathing

Gamida Cell Provides Regulatory Update on Biologics License Application for Omidubicel

Company Spotlight: Cue Biopharma Innovates in the Immunotherapy Space with its Immuno-STAT Platform

Kronos Bio Announces Publication of Preclinical Study Results for Investigational CDK9 Inhibitor KB-0742 in Cell Chemical Biology

AvantGen Announces Licensing of Its Anti-SARS-CoV-2 Antibodies to IGM Biosciences for COVID-19 Therapy Development

ImmunityBio’s hAd5 T-Cell COVID-19 Vaccine Candidate Shows Complete Protection of Airways in Non-Human Primates

Novartis announces lift of partial clinical trial hold and plans to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with SMA

Delivering on the promise of gene editing

Editas Medicine Announces the FDA has Cleared Initiation of the EDIT-301 Clinical TrialEDIT-301 is in development as a best-in-class, durable medicine for people living with sickle cell disease

Neuronascent Announces Publication of Pre-clinical Results of Alzheimer’s Disease Clinical Candidate, NNI-362 in SCR&T Journal

Cryomacs Freezing Bags – A Critical Component in the Successful Delivery of Biological Samples and Therapies

Gene Switch: A Novel Platform for Switching Genes On and Off

Re-assessing the risks of drug-induced arrhythmias during drug discovery

Necessity Is the Mother of Innovation: Key Trends and Predictions from Biotech Showcase Digital After an Unprecedented Year That Drove Momentous Changes in Healthcare

Reflecting on PEGS Europe 2023

TLC Announces Full Patient Enrollment in EXCELLENCE Trial of TLC599 for Osteoarthritis PainOn-schedule completion of enrollment despite COVID-19 pandemic; unique trial design evaluates safety and efficacy of single & repeat doses

Addressing the developability gap in pharma

Sanofi continued its growth trajectory. Strong increase in Q1 2021 business EPS(1) at CER

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