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 Trials In-Vivo Gene Editing 92

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

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

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 168

Gene Editing Gene Gene Therapy In-Vivo 168

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 95

In-Vivo Gene Genome Genomics 95

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 93

Gene Editing In-Vivo Gene Sequencing Gene 93

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

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

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

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

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

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

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 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

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

APRIL 15, 2024

Melissa Little, CEO of the Novo Nordisk Foundation Centre for Stem Cell Medicine “In the stem cell space, 2023 has seen significant increases in the delivery of pluripotent stem cell-derived cell types into first-in-human clinical trials. Gene therapy will move to ex vivo gene modification and the delivery of gene-modified cell types.

Gene Editing 59

Gene Editing Clinical Trials Clinical Trials In-Vivo 59

Drug Discovery World

DECEMBER 6, 2023

“We are impressed by the unique delivery and editing technologies that Eligo has developed, and we believe this will set the stage for Eligo to forge new paths in the application of in vivo gene-editing technologies.” The investment will fuel pre-IND and IND activities to achieve early human data readouts in a PhIb/IIa clinical trial.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 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.

In-Vivo 52

In-Vivo Gene Therapy Clinical Trials Clinical Trials 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.

In-Vivo 52

In-Vivo Gene Therapy Clinical Trials Clinical Trials 52

Roots Analysis

FEBRUARY 27, 2024

Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated. The global genome editing market is anticipated to grow at a CAGR of 12.6% during the forecast period 2023-2035. Get a complete list of the presentations, here.

Genome 40

Genome Genomics Gene Gene Editing 40

pharmaphorum

JANUARY 10, 2022

The German group is paying $40 million upfront to kick off the alliance, which is focusing on the development of up to five CRISPR drugs that can be delivered in vivo for liver-targeted diseases. – are around one-third the size of the more commonly used Cas9.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 52

Drug Discovery World

APRIL 10, 2024

iECURE has been given the go-ahead to begin US trials of its investigational gene editing-based therapy in newborn males with neonatal onset Ornithine Transcarbamylase (OTC) deficiency. The post FDA approves first in vivo gene insertion programme in infants appeared first on Drug Discovery World (DDW).

In-Vivo 80

In-Vivo FDA Approval Gene Gene Editing 80

Pharmaceutical Technology

MAY 24, 2023

is expected to enter Phase II/III registration trial as the first-line treatment for advanced Merkel cell carcinoma (MCC) early next year. to investigational new drug-enabling trials, expected in 2024, to treat aggressive diffuse large B-cell lymphomas (DLBCLs). Morphogenesis’ lead personalised cancer vaccine, IFx-Hu2.0,

Gene Therapy 130

Gene Therapy In-Vivo In-Vitro Immune Response 130

Drug Discovery World

JANUARY 31, 2024

Breye Therapeutics, a clinical-stage biopharmaceutical company developing novel oral therapies for retinal vascular diseases within ophthalmology, has started a Phase Ib/IIa clinical trial to investigate danegaptide, following oral administration, in patients suffering from diabetic macular oedema (DMO).

Clinical Trials 52

Clinical Trials Clinical Trials In-Vivo In-Vitro 52

Pharmaceutical Technology

OCTOBER 5, 2022

In 2010, two patients with end-stage refractory chronic lymphocytic leukaemia (CLL) were administered Novartis’ autologous anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy, Kymriah (tisagenlecleucel), as part of a Phase I trial.

In-Vitro 279

In-Vitro In-Vivo Sales Clinical Trials 279

Drug Discovery World

JANUARY 18, 2024

Poolbeg Pharma, a biopharmaceutical company focused on the development and commercialisation of medicines targeting diseases with a high unmet medical need, has announced promising in vivo results for POLB 001 in addressing cancer immunotherapy-induced cytokine release syndrome (CRS). million cases of cancer by 2030 1,2.

In-Vivo 64

In-Vivo Antibody Clinical Trials Clinical Trials 64

Pharmaceutical Technology

APRIL 28, 2023

Earlier in the year, the company had announced a move towards developing treatments for hemoglobinopathies like sickle cell disease and beta thalassemia and a focus on in vivo discovery. The US agency previously granted the Orphan Drug Designation to EDIT-301 for its study in beta thalassemia, in May 2022. g/dL and 45.4%

Gene Therapy 264

Gene Therapy Drugs Gene Gene Editing 264

Drug Discovery World

FEBRUARY 20, 2024

One example is that the model can circumvent the difficulties in demonstrating in vivo drug efficacy which arise because of mouse specific metabolism issues. Edinburgh-based PhaSER Biomedical (PhaSER) has received a Bill & Melinda Gates Foundation grant totalling $2.3m A key stage of compound progression. The post $2.3m

Drugs 52

Drugs In-Vivo Hormones Development 52

Drug Discovery World

MARCH 15, 2024

million to accelerate development of its lead drug candidates in the area of mitophagy, MTX325 and MTX652, through clinical trials. from our investors, we can now make the next vital steps, progressing with essential clinical trials.” Mission Therapeutics has raised £25.2 ($32.1) Thanks to this additional £25.2m

Clinical Trials 52

Clinical Trials Clinical Trials In-Vivo Trials 52

Drug Discovery World

APRIL 8, 2024

The American Association of Cancer Research (AACR) Annual Meeting launched on Sunday 7 April in San Diego with an Opening Plenary Session showcasing cutting-edge technological advances opening new frontiers in cancer science.

In-Vivo 52

In-Vivo In-Vitro Antibody Protein 52

Drug Discovery World

FEBRUARY 14, 2024

AlphaMedix is a targeted alpha therapy currently in Phase II clinical development, which consists of an SSTR-targeting peptide complex radiolabeled with lead-212 (212Pb) that serves as an in vivo generator of alpha particles. In the Phase I study, treatment was well-tolerated, with a response rate of 62.5%

In-Vivo 52

In-Vivo FDA Approval Clinical Trials Clinical Trials 52