Drug Discovery World

APRIL 19, 2024

Askbio’s AB-1005 (also known as AAV2-GDNF), an investigational gene therapy for the treatment of Parkinson’s disease, was well tolerated with no serious adverse events in a Phase Ib clinical trial. mL) were well tolerated, with no SAEs associated with the investigational gene therapy or contrast agent.

Therapies 130

Therapies Disease Clinical Trials Trials 130

Drug Discovery World

MAY 15, 2024

Dr Catherine Beech , CEO at Exonate and Dr Pete Adamson , CSO at Breye Therapeutics discuss the need for better therapies to treat diabetic retinal disease and current progress in the field. Despite affecting such a large subset of diabetic patients, treatment options are limited to repeated, invasive intravitreal injections to the eye.

Disease 162

Disease Treatment Therapies Pharmaceuticals 162

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).

Treatment 130

Treatment Clinical Trials Pharmacokinetics Trials 130

Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

FDA Approval 130

FDA Approval Therapies FDA Disease 130

Drug Discovery World

JANUARY 30, 2023

Register to attend Cell and gene therapy: sustaining growth and maximising opportunities, a free-to-attend DDW webinar, supported by Benchling and BPS Bioscience, on February 17, 2023 at 8am PST / 11am EST / 4pm GMT / 5pm CET. Cell and gene therapies (CGT) are transforming the way diseases are treated.

Disease 130

Disease Treatment Therapies Drug Development 130

Drug Discovery World

MAY 24, 2023

Moderna has reported encouraging interim data from its Phase I/II trial of mRNA-3927, an mRNA therapy for rare metabolic disorder propionic acidemia (PA). The data was presented at the 2023 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting.

Therapies 130

Therapies Clinical Trials Pharmacokinetics Trials 130

Drug Discovery World

FEBRUARY 15, 2024

DDW Editor Reece Armstrong speaks to Tracy M Woody , President and Chief Executive Officer of Cessation Therapeutics, about the company’s goal to develop an antibody therapy to prevent fentanyl overdoses. TW : Narcan (or naloxone) is a very important treatment for fentanyl overdose. Yet opioid deaths are high in the US.

Therapies 147

Therapies FDA Approval FDA Government 147

Drug Discovery World

JUNE 15, 2023

A Phase I clinical trial testing a CAR-T cell therapy treatment in children with acute myeloid leukaemia (AML) has been paused due to a patient’s death. The PLAT-08 clinical trial is a Phase I study of 2seventy bio’s cell therapy SC-DARIC33 in patients with AML.

Clinical Trials 130

Clinical Trials Therapies Trials FDA 130

Drug Discovery World

DECEMBER 19, 2022

Researchers have found that a topical gene therapy gel helps heal wounds in patients with life-threatening blistering skin disease epidermolysis bullosa (EB). . It’s an intensely painful condition, and medical treatment for it has been limited largely to palliative care. . One trial participant was Vincenzo Mascoli, aged 22.

Therapies 130

Therapies Clinical Trials Trials Virus 130

Drug Discovery World

SEPTEMBER 26, 2022

UMC Utrecht and its partners have received a research grant to unravel how the intestinal microbiota can be exploited to boost cancer immunotherapy and limit therapy side-effects. . These adverse events often present in the intestinal tract as severe inflammation. USA) and MicroViable Therapeutics (Spain). .

Therapies 130

Therapies Research Treatment Disease 130

Drug Discovery World

NOVEMBER 14, 2023

Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments. Verve-101 is an investigational, in vivo base editing medicine designed to be a single-course treatment that inactivates the PCSK9 gene in the liver to durably lower blood LDL-C. “Of

Therapies 130

Therapies Trials Clinical Trials Disease 130

Drug Discovery World

SEPTEMBER 6, 2022

In recent years the advanced therapy industry has been the most significant disruptor in medicine. Cell and gene therapies are not only transforming the way diseases are treated but are also aiming to cure entirely new patient populations. Record breaking investments in 2021 fuel an industry on fire . billion raised.

Therapies 130

Therapies Clinical Trials Production Disease 130

Drug Discovery World

APRIL 3, 2024

million from the Spanish Research Agency (AEI), under the call for grants for Public-Private Collaboration Projects, to accelerate the development of an innovative treatment aimed at controlling disabling and lethal haemorrhage.

Therapies 130

Therapies Laboratories Pharmacokinetics Treatment 130

Drug Discovery World

NOVEMBER 24, 2023

AstraZeneca has announced that the National Institute for Health and Care Excellence (NICE) has recommended Imfinzi (durvalumab) in combination with gemcitabine and cisplatin (chemotherapy) for NHS use in England and Wales for the first-line treatment of adults with locally advanced, unresectable, or metastatic biliary tract cancer (BTC).

Therapies 130

Therapies Licensing Licensing Treatment 130

Drug Discovery World

NOVEMBER 30, 2023

The reports were received from clinical trials and postmarketing adverse event (AE) data sources. The FDA has cautioned that patients and clinical trial participants receiving treatment with these products should be monitored life-long for new malignancies.

FDA 162

FDA Clinical Trials Therapies Production 162

Chemical Biology and Drug Design

JULY 26, 2023

Abstract Noncoding RNAs (ncRNAs) are engaged in key cell biological and pathological events, and their expression alteration is connected to cancer progression both directly and indirectly. However, there are several limitations, including delivery, uptake, and short half-life, in the application of ncRNAs in cancer treatment.

Engineering 100

Engineering Therapies Cell Biology Drugs 100

Drug Discovery World

JUNE 12, 2024

Immune-checkpoint inhibitor (ICI) therapy has transformed treatment outcomes for RCC. However, ICIs can also trigger immune-related adverse events and may not benefit all patients. If successful, the data we generate could help us learn more about the biology of renal cell carcinoma and its treatments.”

Protein Expression 130

Protein Expression Medical Schools Treatment Therapies 130

Drug Discovery World

MAY 10, 2024

291 patients were randomised to treatment either with evenamide or placebo as add-on to their current antipsychotic therapy. There was no difference in the incidence of CNS, psychiatric, gastrointestinal or other adverse events between evenamide and placebo. points, compared to 7.6

International 182

International Pharmaceuticals Treatment Trials 182

Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. million price point per dose.

Therapies 130

Therapies Clinical Trials Vaccine Disease 130

Chemical Biology and Drug Design

FEBRUARY 13, 2024

Some researchers proposed that the symptoms involved in AD (loss of memory, cognitive impairment, and amnesia) are the main event linked to the cholinergic neurotransmitter (acetylcholine). This review focuses on the last 10 year's literature search on huprine and its analogues for the treatment of AD.

Disease 100

Disease Treatment Therapies Research 100

Drug Discovery World

AUGUST 2, 2023

Renaissance Pharma has announced its first development programme focused on Hu14.18, a humanised anti-GD2 monoclonal antibody (mAb), licensed from St Jude Children’s Research Hospital for the treatment of newly diagnosed high-risk neuroblastoma. and three-year event-free (EFS) of 73.7%. and three-year event-free (EFS) of 73.7%.

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Licensing Licensing Hospitals Therapies 246

Vial

DECEMBER 8, 2023

With technological innovation and scientific research propelling the development of new therapies, anticipation for the future is high. As we look forward to 2024, several groundbreaking therapies are poised to radically change the landscape of ophthalmology, providing hope and improved treatment options for patients with ocular conditions.

Therapies 52

Therapies Clinical Trials Trials Treatment 52

Drug Discovery World

JUNE 28, 2023

Takeda has revealed favourable interim results from its Phase III crossover trial evaluating the safety and efficacy of TAK-755 (recombinant ADAMTS13) replacement therapy for the prophylactic treatment of congenital thrombotic thrombocytopenic purpura (cTTP). Acute TTP events have a mortality rate of >90%, if left untreated.

Trials 130

Trials Clinical Trials Therapies Drugs 130

PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. A gene-based treatment would have to alter many cells to exert a noticeable effect. Researchers have been working on developing gene therapy for DMD for decades. million DNA bases. Hyperbole or Hope?

FDA Approval 75

FDA Approval Treatment FDA DNA 75

Drug Discovery World

JANUARY 27, 2023

Key features of the Winter issue include an overview of the drug discovery landscape in 2023, sector successes and challenges in the Scandinavian market, and how a drug sourced from the aerospace industry could offer treatments for multiple disease targets.

Therapies 246

Therapies Laboratories Treatment Disease 246

Drug Discovery World

MAY 8, 2024

No medication has been approved by the FDA for vaping cessation in the United States,” said lead author Nancy Rigotti, Director of Massachusetts General Hospital’s Tobacco Research and Treatment Center and a Professor of Medicine at Harvard Medical School. For cytisinicline treatment compared to placebo, 31.8% versus 15.1%

Clinical Trials 182

Clinical Trials Compliance Trials Drugs 182

Drug Discovery World

JUNE 26, 2024

The drug was tested for the treatment of moderate-to-severe obstructive sleep apnoea (OSA) in adults with obesity, with and without positive airway pressure (PAP) therapy. In the primary endpoint, tirzepatide reduced moderate-to-severe OSA severity by up to 62.8% (about 30 fewer events per hour).

Drugs 147

Drugs Clinical Trials Disease Treatment 147

Drug Discovery World

APRIL 19, 2024

The headline news this week all concerns clinical trials for new drugs that could have a significant impact on future patient treatment, including trials testing therapeutics for ALS, cystic fibrosis, gonorrhoea, TB and Parkinson’s. News round-up for 15-19 April by DDW Digital Content Editor Diana Spencer.

Clinical Trials 130

Clinical Trials Vaccine Trials Drugs 130

Drug Discovery World

DECEMBER 12, 2023

Exonate, an mRNA therapy company focused on treatments for diabetic complications, has announced that its lead ophthalmology asset, EXN407, has achieved its prespecified endpoints in a Phase Ib/IIa study. During the trial, EXN407 met all safety and pharmacokinetic parameters and displayed encouraging signals of biological activity.

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Small Molecule Trials Pharmacokinetics Treatment 130

FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Therapies 119

Therapies FDA FDA Approval Treatment 119

Drug Discovery World

APRIL 21, 2023

The event showcased the very latest innovations in cancer therapies, from personalised mRNA vaccines to bispecific antibodies. The post This week in drug discovery (16-21 April) appeared first on Drug Discovery World (DDW).

Clinical Trials 130

Clinical Trials Vaccine Drugs Therapies 130

Drug Discovery World

JUNE 21, 2024

Allogene Therapeutics and Foresight Diagnostics have announced the initiation of a Phase II trial evaluating the use of cemacabtagene ansegedleucel (cema-cel) as part of the first line treatment regimen for newly diagnosed large B-cell lymphoma (LBCL) patients who are likely to relapse.

Trials 147

Trials Compliance Clinical Trials Doctors 147

Drug Discovery World

APRIL 25, 2023

University of California researchers have dosed the second participant in their clinical trial to identify a potential cure for HIV utilising CAR-T cell therapy. The trial is the first-in-human clinical study investigating the duo CAR-T cell therapy for the treatment of HIV. “We

Research 130

Research Clinical Trials Therapies Clinical Development 130

Drug Discovery World

MARCH 22, 2024

The US Food and Drug Administration (FDA) has approved Tryvio (aprocitentan) for the treatment of hypertension in combination with other antihypertensive drugs, to lower blood pressure in adult patients who are not adequately controlled on other drugs. Now we are bringing significant medical progress for patients with systemic hypertension.

FDA Approval 130

FDA Approval FDA Therapies Treatment 130

Drug Discovery World

JANUARY 27, 2023

Cell and gene therapies have been making the news this week, as we launch our very own guide to global innovation in CGT with the Winter issue of DDW, as well as an exciting new virtual event on the same topic. News round-up for 23-27 January by DDW Digital Content Editor Diana Spencer.

RNA 130

RNA Therapies Drugs Virus 130

Drug Discovery World

JANUARY 10, 2024

Emphasising that 34,000 patients worldwide have received commercially available CAR-T cell immunotherapies, the commentary notes that the observed rate of 20 reported cases of T cell malignancies is notably lower than that reported for more conventional alternative treatments.

FDA 147

FDA Therapies International Treatment 147

Drug Discovery World

JANUARY 31, 2024

By Megan Thomas, DDW Multimedia Editor The last time the DDW team were in Boston for a SLAS event two years ago, the world was very much in the early recovery period of Covid-19. For many, it was their first in-person event – a welcome return.

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Government Vaccine Disease Science 130