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 246

Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

XTalks

APRIL 14, 2022

Conventional in vitro and in vivo studies are used in the drug development pipeline. Take this quiz to test your knowledge about in silico trials! Register for this free, upcoming webinar to learn more about the benefits of implementing in silico clinical trials with mechanistic models for drug development. Want to learn more?

Trials 98

Trials In-Vivo In-Vitro Clinical Trials 98

Pharmaceutical Technology

MAY 19, 2023

Myeloid Therapeutics has raised $73m to support the continued clinical development of its lead cell therapy programme, MT-101, in Phase I/II trials for T cell lymphoma. MT-302 is a TROP2-targeting in vivo chimeric antigen receptor (CAR) that has been designed to express in the myeloid compartment.

In-Vivo 246

In-Vivo Gene Therapy Gene RNA 246

pharmaphorum

FEBRUARY 1, 2024

A single dose of a gene-editing drug being developed by Intellia Therapeutics almost eliminated attacks in patients with hereditary angioedema (HAE) in a clinical trial, pointing to the potential of CRISPR-based drugs delivered in vivo to treat diseases.

Gene Editing 95

Gene Editing In-Vivo Drugs Clinical Trials 95

BioSpace

OCTOBER 18, 2023

The trial, which is the first late-stage study of an in vivo CRISPR treatment in the U.S., will start by the end of 2023. Intellia's NTLA-2001 is a treatment candidate for transthyretin amyloidosis cardiomyopathy.

Gene Editing 112

Gene Editing In-Vivo Gene Trials 112

pharmaphorum

NOVEMBER 18, 2022

Gene-testing specialist Editas Medicine has halted development of its lead clinical programme for congenital eye disorders after it generated lacklustre results in a phase 1/2 trial. The post After disappointing trial, Editas puts lead CRISPR drug up for sale appeared first on. 30% of all LCA cases.

Sales 92

Sales In-Vivo Trials Gene Editing 92

Pharmaceutical Technology

MAY 16, 2023

The approval also includes clearance for a Phase I clinical trial protocol of NXP900. NXP900 plus Osimertinibplus enzalutamide in vivo showed a possible ability to reverse acquired resistance in metastatic castration-resistant prostate cancer.

In-Vivo 130

In-Vivo In-Vitro Clinical Trials Clinical Trials 130

Drug Discovery World

JUNE 8, 2023

Grown in vitro, these cells can provide patient specific human brain models from a large cohort of AD patients – to create a ‘clinical trial in a dish’. has paved the way for the use of cell-based assays to investigate drug safety and efficacy, including this innovative ‘clinical trial in a dish’ approach.

Clinical Trials 97

Clinical Trials Clinical Trials Trials In-Vitro 97

BioSpace

NOVEMBER 18, 2021

Avrobio’s ex vivo lentiviral gene therapy platform led to durable responses measured in years, and significant reductions in disease-related biomarkers that far exceed today’s standard of care.

In-Vivo 98

In-Vivo Gene Therapy Gene Trials 98

Drug Discovery World

JUNE 5, 2023

Aviceda Therapeutics has dosed the first patient in its Phase II SIGLEC trial investigating AVD-104 as a treatment for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). AVD-104 is a novel glycan-coated nanoparticle, supported by a strong pre-clinical in vivo efficacy and safety profile. “I

Trials 52

Trials In-Vivo Drugs 52

Worldwide Clinical Trials

NOVEMBER 27, 2023

In trials, Casgevy was shown to help prevent episodes of debilitating pain, known as vaso-occlusive crises, that often plague patients affected by sickle cell disease. In June 2023, the U.S. Food and Drug Administration (FDA) announced its acceptance of the Biologics License Application (BLA) for exa-cel.

Gene Editing 189

Gene Editing Gene Gene Therapy In-Vivo 189

Drug Discovery World

JUNE 12, 2023

The US Food and Drug Administration (FDA) has approved Minoryx Therapeutics’ Phase III clinical trial (CALYX) of leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (cALD). Patient recruitment is expected to start by the end of Q2 2023 with results anticipated by late 2025.

FDA Approval 52

FDA Approval Trials Radiology In-Vivo 52

Pharmaceutical Technology

MARCH 29, 2023

The trial, which is investigating healthy adults with a minimum body mass index of 30 kg/m2, will see VK2735 administered as an oral tablet once daily for 28 days. The Phase I study is an extension of a previously completed Phase I trial that evaluated the subcutaneous administration of VK2735.

Drugs 264

Drugs In-Vivo Marketing Trials 264

Drug Discovery World

FEBRUARY 22, 2024

With no in-house laboratory space, the company works closely with multiple international contract research organisations (CROs), including Axxam, which carry out all of the Libra R&D, from compound screening assays, to in vivo pharmacokinetic/pharmacodynamic and ADME work, as well as in vitro and in vivo pre-clinical safety assessments.

Clinical Trials 52

Clinical Trials Clinical Trials Trials In-Vivo 52

pharmaphorum

JULY 13, 2022

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.

Gene Editing 97

Gene Editing Trials Drugs Gene 97

Pharmaceutical Technology

NOVEMBER 22, 2022

The robust expertise of IASO in monitoring possibly best-in-class CARs leveraging its fully-human antibody discovery platform (IMARS), quickly conducting clinical trials and its in-house GMP facility for producing plasmid, virus vector and CAR-T cells intends to make its new therapies available to larger population across the world.

Development 276

Development In-Vivo Engineer Clinical Trials 276

BioSpace

OCTOBER 18, 2021

On Monday, LogicBio Therapeutics unveiled clinical trial results demonstrating the first-ever in vivo, nuclease-free genome editing in little humans.

In-Vivo 93

In-Vivo Gene Genome Genomics 93

Pharmaceutical Technology

APRIL 4, 2023

Formerly known as CTX001, exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy. The BLAs are supported by data obtained from the ongoing Phase III CLIMB-111 and CLIMB-121 trials, along with an ongoing long-term follow-up CLIMB-131 trial. It is being assessed for the treatment of SCD or TDT patients.

Gene Therapy 246

Gene Therapy Gene Editing In-Vivo Gene 246

XTalks

APRIL 24, 2023

Rare disease clinical trials are complex due to the additional scientific, medical, operational and regulatory requirements of newly emerging advanced therapies, such as gene therapy,” says Dr. “Thankfully, we can anticipate that many more of these new treatments are expected in the future.”

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

APRIL 13, 2023

Significant collaborative R&D programmes that are likely to be affected include Takeda’s partnership with Codexis for AAV-based gene therapy for Fabry disease and Takeda’s $3.6bn dollar deal with Poseida for the development of nonviral in vivo gene therapy programmes for the hereditary bleeding disorder haemophilia A.

Gene Therapy 243

Gene Therapy Marketing Gene In-Vivo 243

Drug Discovery World

MARCH 8, 2023

The post Company launches ADC Phase I trial in solid tumours appeared first on Drug Discovery World (DDW). Trop2 is not expressed or has low expression levels in normal human tissues while overexpressed in a variety of solid tumours, especially epithelial tumours.

Trials 52

Trials In-Vivo In-Vitro Antibody 52

Pharmaceutical Technology

AUGUST 18, 2022

The latest approval is based on findings from the Phase III clinical trials, HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up LTF-303 study. The latest approval is based on findings from the Phase III clinical trials, HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up LTF-303 study.

Gene Therapy 246

Gene Therapy Gene Genotype Genetics 246

Pharmaceutical Technology

FEBRUARY 1, 2023

Ellipses Pharma stated that the dual FLT3 inhibition and Aurora kinase demonstrated to overcome the acquired resistance to selectively inhibit FLT3 in vitro and in vivo. The FDA IND clearance allows the company to expand its ongoing first-in-human Phase I/II clinical trial of EP0042.

FDA Approval 130

FDA Approval In-Vivo In-Vitro Trials 130

pharmaphorum

JUNE 23, 2022

Novartis has shouldered its way into the in vivo gene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). Other companies – notably Vertex Pharma/CRISPR Therapeutics and bluebird bio – are taking an ex vivo approach to SCD and thalassaemia and are much closer to market.

Gene Editing 80

Gene Editing In-Vivo Gene Sequencing Gene 80

Drug Discovery World

SEPTEMBER 29, 2023

Immunotherapy has taken centre stage this week, with several announcements relating to regulatory approval, investment and research results for antibody and CAR-T therapies, perhaps most significantly, encouraging early-stage data for the first in vivo CAR-T therapy. The top stories: €27.3M

In-Vivo 52

In-Vivo Drugs Antibody Clinical Trials 52

Drug Discovery World

APRIL 2, 2024

A new Case Study is now available to download for free: ‘In Vivo Evaluation Of Bispecific-Cell Engager Efficacy & Safety ‘ Discover a clinically predictive and reproducible platform developed by The Jackson Laboratory for simultaneous efficacy and safety assessment.

Drugs 59

Drugs In-Vivo Development Trials 59

Pharmaceutical Technology

SEPTEMBER 19, 2022

The latest development is based on findings from the single-arm, open-label Phase II/III ALD-102 (Starbeam) and Phase III ALD-104 clinical trials. A one-time gene therapy, Skysona leverages ex-vivo transduction with the Lenti-D lentiviral vector for adding the ABCD1 gene’s functional copies into the hematopoietic stem cells of the patient.

FDA Approval 246

FDA Approval In-Vivo Gene Therapy Clinical Trials 246

Drug Discovery World

MARCH 10, 2023

Perhaps most interestingly this week, after previous caution over in vivo gene therapy trials, the FDA has given the go ahead to Intellia Therapeutics to study NTLA-2002 in hereditary angioedema (HAE). The post This week in drug discovery (6-10 March) appeared first on Drug Discovery World (DDW).

In-Vivo 52

In-Vivo Clinical Trials Clinical Trials Drugs 52

Pharmaceutical Technology

JUNE 21, 2023

It is designed to enhance the function of regulatory T cells (Tregs) in vivo to treat the systemic and neuro-inflammation underlying autoimmune and neurodegenerative diseases. This had a potent neuroprotective effect on nigral dopaminergic neurons in affected mice.

In-Vivo 130

In-Vivo Immune Response Clinical Trials Clinical Trials 130

Pharmaceutical Technology

JULY 14, 2022

AVR-RD-05 makes use of the own hematopoietic stem cells (HSCs) of a patient that are transduced ex vivo with a lentiviral vector encoding the human IDS enzyme. Next year, Avrobio is expected to initiate a collaborator-sponsored Phase I/II clinical trial for Hunter syndrome in partnership with the University of Manchester, UK.

Gene Therapy 130

Gene Therapy Gene Drugs In-Vivo 130

Pharmaceutical Technology

SEPTEMBER 14, 2022

The company is currently progressing its lead drug candidate, RYZ101, into clinical trials for various solid tumour indications. A Phase Ib clinical trial of the drug is underway, enrolling subjects with neuroendocrine tumours, and Phase III trials are anticipated to commence next year.

In-Vivo 130

In-Vivo Clinical Trials Clinical Trials Engineer 130

WCG Clinical

MAY 28, 2024

The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies. Recent headlines have highlighted the potential for Chimeric Antigen Receptor (CAR)-based therapies to provide clinical benefits to persons affected by lupus. In these cases B cell depletion is a feature, not a bug.

In-Vivo 52

In-Vivo Clinical Trials Clinical Trials FDA Approval 52

BioTech 365

SEPTEMBER 29, 2020

First patient was administered today The trial aims at validating the safety of continuous administration of Ivermectin in oral form Simultaneously, several long-acting injectable formulations based on MedinCell’s BEPO® technology are being tested in vivo A 1-month active injectable prophylactic … Continue reading →

Clinical Trials 40

Clinical Trials Clinical Trials In-Vivo Trials 40

Drug Discovery World

MAY 17, 2024

The top stories: Gene therapy cures profound genetic deafness within 24 weeks The investigational gene therapy DB-OTO improved hearing to normal levels in a child (dosed at 11 months of age) within 24 weeks, according to trial data presented at the American Society of Gene and Cell Therapy (ASGCT) annual conference.

Gene Therapy 52

Gene Therapy Gene Drugs In-Vivo 52

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. The rapidly expanding landscape of cell therapy trials has put an operational strain on many clinical sites due to limited resources and infrastructure. “In

Clinical Trials 52

Clinical Trials Clinical Trials Trials Gene Therapy 52

Drug Discovery World

MARCH 13, 2023

Perhaps most interestingly this week, after previous caution over in vivo gene therapy trials, the FDA has given the go ahead to Intellia Therapeutics to study NTLA-2002 in hereditary angioedema (HAE). You can listen below, or find The Drug Discovery World Podcast on Spotify , Google Play and Apple Podcasts.

Gene Therapy 52

Gene Therapy Drugs In-Vivo Clinical Trials 52

Drug Discovery World

MAY 26, 2023

AM1476, a highly selective small molecule, delivered as an orally administered tablet, was found to be safe and well-tolerated at all relevant doses in a trial of approximately 100 healthy subjects. Skin fibrosis is a leading feature of SSc associated with significant disability.

In-Vivo 52

In-Vivo Trials Marketing Drugs 52