Drug Discovery World

MAY 17, 2024

From a weight loss drug that prevents heart attacks and a gene therapy that restores hearing, to a vaccine that can treat viruses that don’t exist yet, our chosen news stories this week all represent potential breakthroughs in their respective fields. News round-up for 13-16 May by DDW Senior Digital Content Editor Diana Spencer.

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Pharmaceutical Technology

APRIL 28, 2023

The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement. The US agency previously granted the Orphan Drug Designation to EDIT-301 for its study in beta thalassemia, in May 2022.

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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

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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’. The recent US Federal Food and Drug Administration Agency (FDA) Modernization Act 2.0

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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).

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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. Gates Foundation grant to accelerate global drug discovery appeared first on Drug Discovery World (DDW). A key stage of compound progression. The post $2.3m

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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.

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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

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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).

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Drug Discovery World

MARCH 8, 2023

Dosing has begun in a Phase I clinical study evaluating BAT8008, an antibody-drug conjugate (ADC) that targets Trop2. Key objectives of the study are to determine the maximum tolerated dose (MTD), recommended Phase II dose (RP2D), and to evaluate the pharmacokinetics and preliminary efficacy in patients with advanced solid tumours.

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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.

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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.

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Worldwide Clinical Trials

NOVEMBER 27, 2023

Food and Drug Administration (FDA) announced its acceptance of the Biologics License Application (BLA) for exa-cel. 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.

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Drug Discovery World

MAY 9, 2023

Jantzen Sperry , PhD, Director of Scientific Operations at Certis Oncology looks at how the adoption of orthotopic PDX models can elevate the impact of cancer research and improve translation in oncology drug development. Fewer than 5% of oncology drugs that enter clinical trials in the US receive US FDA approval.

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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.

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Drug Discovery World

DECEMBER 7, 2023

This is the first example of an OOC provider’s data supporting clinical progression of a drug for metabolic, fibrotic liver disease. The post Organ-on-a-chip data supports clinical progression of liver disease drug appeared first on Drug Discovery World (DDW).

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pharmaphorum

FEBRUARY 13, 2022

German drugmaker Merck KGaA has reached an agreement to pilot an artificial intelligence platform developed by Quris that aims to identify potential safety problems with new therapeutic candidates as early as possible in the drug discovery process.

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Drug Discovery World

APRIL 5, 2023

Italian research institute and contract research organisation IRBM has become the only company from outside the United States to join cancer drug discovery network the Chemical Biology Consortium (CBC). The post Only non-US company joins cancer drug discovery consortium appeared first on Drug Discovery World (DDW).

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Drug Discovery World

APRIL 15, 2024

DDW’s Megan Thomas speaks to experts from the drug discovery industry about their predictions on the future of stem cells in drug discovery. Gene therapy will move to ex vivo gene modification and the delivery of gene-modified cell types. In addition, I believe we will see more exosome-based therapies enter the clinic in 2024.

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pharmaphorum

AUGUST 10, 2020

Gilead has filed its COVID-19 drug remdesivir with the FDA, to treat patients with severe disease, under the brand name Veklury. The drug is currently available to US patients under an Emergency Use Authorisation for treatment of hospitalised patients with severe COVID-19. Feature image courtesy of Rocky Mountain Laboratories/NIH.

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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.

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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.

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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. The post Positive Phase I data for oral fibrosis treatment appeared first on Drug Discovery World (DDW).

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Drug Discovery World

JUNE 30, 2023

Research tools, safety testing and regenerative medicines – these endless stem cell applications are powering precision, speed, and new modalities in drug development. Stem cells for use in drug discovery Stem cells are fast becoming an invaluable tool in the drug discovery process.

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Drug Discovery World

JANUARY 23, 2023

Researchers have discovered a clinical drug that sensitises glioblastoma (GBM) cells to radiotherapy and could replace the current standard of care. In addition, they discovered that cladribine, a clinical drug, activates DGKB and inhibits DGAT1. GBM is a WHO grade IV brain tumour with dismal prognosis.

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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

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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,

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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.

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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.

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Drug Discovery World

OCTOBER 20, 2022

Aqemia has raised €30 million in a Series A funding round to further support its mission to massively scale drug discovery through a first-in-class technological platform combining quantum-inspired physics and machine learning. . We are continuously recruiting to address our next technology and drug discovery challenges”, she added. .

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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.

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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).

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Drug Discovery World

MARCH 26, 2024

For example, progress made in developing gene mutant-specific inhibitors, antibody-drug conjugates (ADC), or cellular therapies. In vivo, METTL1 inhibitors induce tumour growth inhibition in both immune-deficient and immune-competent mouse strains. Results of that Phase I trial are expected to be presented later this year.

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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%

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Drug Discovery World

MAY 29, 2023

The company previously participated in the first human trial of a gene therapy drug candidate for Menkes disease, and recently partnered with Stand Up Therapeutics to produce and supply gene therapy products for the treatment of paralysis. “As

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Drug Discovery World

APRIL 8, 2024

Following this, Benjamin Cravatt, Professor and the Norton Gilula Chair in Biology and Chemistry at The Scripps Research Institute, discussed activity-based protein profiling (ABPP) and its use in discovering drug candidates for cancer-relevant proteins.

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Drug Discovery World

DECEMBER 14, 2023

A number of companies presented breakthrough data for their investigational breast cancer drugs at the San Antonio Breast Cancer Symposium. The results from this trial evaluating vepdegestrant in combination with palbociclib help advance our goals of benefitting patients with ER+/HER2- breast cancer.

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