Clinical Trials, Containment, Genetics and In-Vivo

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

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. The webinar included operational strategies for these trials from Laura Omoboni, MS, Executive Director of Clinical Trial Management. Rare diseases can often be progressive, chronic and fatal.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

The regulatory round-up: Eight key FDA decisions

Drug Discovery World

APRIL 4, 2023

Read more: FDA Fast Track Designation for retinitis pigmentosa treatment Drug candidate for TUBB4a leukodystrophy, SynaptixBio The FDA has granted Rare Paediatric Disease (RPD) Designation to SynaptixBio’s drug candidate for TUBB4a leukodystrophy – a genetic and debilitating condition which mainly affects babies and young children.

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

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

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

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

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

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

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

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. Early phase clinical trials for gene editing therapies.

Gene Editing

Gene Editing Gene In-Vivo Genome

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.

Gene

Gene Gene Therapy Gene Expression Regulation

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

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

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

In other areas of the industry (such as cell and genetic medicine development) progress continued at an impressively brisk pace despite the pandemic. Regarding OWS’s support for vaccine development, Dr Slaoui noted that 5 of the 6 vaccines selected (from a total of 94 programs) are currently in phase 3 development or approved.

Vaccination

Vaccination Vaccine In-Vivo Genetics

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

Clinical Research Informer

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

The regulatory round-up: Eight key FDA decisions

Trending Sources

Are organ-chips the future of preclinical research?

Overcoming diverging regulatory expectations to bring CGTs to market

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

Lysogene Reports Positive Biomarker Data With LYS-SAF302

Fighting viruses is as easy as breathing

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

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

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

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

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

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

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

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