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.

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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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SCIENMAG: Medicine & Health

FEBRUARY 26, 2024

The 7th World Conference on Targeting Phage Therapy 2024, two-day event dedicated to advancing the field of phage research and therapy will be hosted at Corinthia Palace Malta on June 20-21.

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Drug Discovery World

NOVEMBER 24, 2022

DDW’s Diana Spencer asks what will be needed for Europe to compete in the growing global market for advanced therapies. . In terms of ATMPs, other countries are ramping up support for these innovative therapies. ATMP clinical trial activity is twice as high in the US and almost three times as high in China than in Europe.

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Drug Discovery World

MARCH 15, 2024

Biotechnology company Allyx Therapeutics has reported positive Phase Ib clinical data for its lead compound, ALX-001, a first-in-class, synapse-targeted, disease-modifying oral therapy in development for neurodegenerative diseases. All doses were well-tolerated and there were no serious adverse events.

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Drug Discovery World

AUGUST 29, 2023

A stem cell derived investigational therapy for treating Parkinson’s disease has shown promise in early clinical trials. BlueRock Therapeutics, a subsidiary of Bayer, has announced details of the positive data from a Phase I clinical trial for bemdaneprocel (BRT-DA01).

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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 Casgevy, a cell-based gene therapy, is approved for the treatment of SCD in patients 12 years of age and older with recurrent vaso-occlusive crises (VOCs).

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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). . One trial participant was Vincenzo Mascoli, aged 22. This treatment made a huge difference in quality of life for Vincenzo and other trial participants.” .

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Drug Discovery World

SEPTEMBER 8, 2023

The first quadruplex-edited, allogeneic CAR-T cell therapy candidate has entered clinical trials in the US. The primary objectives of the Phase I portion of the trial are the assessment of safety, tolerability, and identification of the recommended Phase II dose and lymphodepletion regimen.

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Drug Discovery World

NOVEMBER 1, 2023

Axicabtagene ciloleucel, Kite Two indirect comparative analyses of CAR T-cell therapy axicabtagene ciloleucel vs. bispecific antibodies epcoritamab and glofitamab, for relapsed/refractory diffuse large B-Cell lymphoma, showed improved progression-free survival (PFS). Median PFS was 13.8 months and median duration of response (DoR) was 15.5

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Drug Discovery World

NOVEMBER 14, 2023

A Phase Ib trial has shown low-density lipoprotein cholesterol (LDL-C) reductions up to 55% and blood PCSK9 protein reductions up to 84% after a single infusion of Verve Therapeutics’ Verve-101. The post Single-course in vivo base editing therapy proven to lower cholesterol appeared first on Drug Discovery World (DDW).

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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. ViaCyte, Beam, and Verve are all due to begin gene editing trials this year.

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Drug Discovery World

APRIL 16, 2023

Patients with treatment-naïve resectable non-small cell lung cancer (NSCLC) who received neoadjuvant durvalumab (Imfinzi) plus chemotherapy and adjuvant durvalumab monotherapy had improved event-free survival (EFS) and pathological complete response (pCR) compared with those who received neoadjuvant chemotherapy alone. Overall, 77.6%

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Drug Discovery World

APRIL 10, 2023

RESP-X, Infex Therapeutics’ anti-virulence therapy to treat Pseudomonas aeruginosa (Pa) infections in non-cystic fibrosis bronchiectasis (NCFB) patients, has shown a promising interim safety and tolerability profile in its ongoing first-in-human Phase I clinical trial.

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Drug Discovery World

JULY 19, 2023

The primary endpoint of iADRS measures cognition and activities of daily living such as managing finances, driving, engaging in hobbies, and conversing about current events. This is the first Phase III trial of any investigational medicine for Alzheimer’s disease to deliver 35% slowing of clinical and functional decline.”

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Drug Discovery World

SEPTEMBER 12, 2022

If anything, this period has been a reminder that people are the primary reason we do what we do, and the Patient Access, Healthcare & People track at Advanced Therapies Europe 2022 allowed DDW’s Multimedia Editor Megan Thomas to share several reflections on the topic. . Never before was the whole world a potential patient.

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Drug Discovery World

APRIL 3, 2024

Obtaining these results will allow us to reach a key milestone of the project: approval from the health authorities to test CM-352 in a Phase I clinical trial. The post Validating a revolutionary therapy against lethal haemorrhage appeared first on Drug Discovery World (DDW). Our initial focus will be on the US FDA.

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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.

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Drug Discovery World

JULY 12, 2023

There is an urgent need for new therapies that safely and effectively eliminate congestion and improve clinical outcomes, and we believe DSR has the potential to be a disease-modifying heart failure therapy for these patients.” MOJAVE study design The non-randomised cohort consists of three eligible patients who will be treated with DSR 2.0,

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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. The post This week in drug discovery (15-19 April) appeared first on Drug Discovery World (DDW).

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Drug Discovery World

NOVEMBER 24, 2023

7,8 Results from the primary endpoint of the TOPAZ-1 Phase III trial showed that durvalumab, in combination with standard of care gemcitabine plus cisplatin (chemotherapy) demonstrated a clinically meaningful and durable overall survival (OS) benefit as a treatment for patients with advanced BTC.6 0.97, p=0.021). 9 An additional 6.5

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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.

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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

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Drug Discovery World

MAY 8, 2024

The ORCA-V1 trial was led by Achieve Life Sciences, and co-led by an investigator from Massachusetts General Hospital. In their previous clinical trial, the research team found that cytisinicline helped people to quit smoking traditional cigarettes. Cytisinicline was well tolerated and no serious adverse events were reported.

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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. billion, compared to $19.9 Cancer is king .

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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.

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Drug Discovery World

AUGUST 2, 2023

A novel Phase II trial incorporating Hu14.18 into induction therapy and additionally, within post-consolidation therapy, demonstrated outstanding patient outcomes with overall survival (OS) of 86.0% and three-year event-free (EFS) of 73.7%. achieved a partial response or better at the end of induction.

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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. OncoHost and Dana-Farber Cancer Institute have launched a new study to identify biomarkers for renal cell carcinoma (RCC).

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Drug Discovery World

JUNE 26, 2024

on the apnoea-hypopnea index (AHI) compared to placebo in the SURMOUNT-OSA Phase III clinical trials. 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. Tirzepatide demonstrated a mean reduction of up to 62.8%

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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

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 In 2021 there were an estimated 1.5

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PPD

MAY 18, 2023

Yet, four out of five care partners of people with neurodegenerative disorders have never been informed of clinical trial opportunities , according to a survey of more than 250 caregivers conducted by PPD, the clinical research business of Thermo Fisher Scientific. There’s little time to lose.

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Advarra

JUNE 13, 2024

Recombinant DNA technologies and genetically modified biological agents are being adapted for a wide scope of therapeutic applications, and their use is becoming increasingly common in clinical trials. For example, a site conducting only protocols involving mRNA may be able to leverage a generic SOP covering any mRNA protocol at that site.

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Advarra

JUNE 20, 2023

Innovation Organizations conducting oncology clinical trials face challenges distinct from the rest of the research community. cell and gene therapies), with other therapeutic areas then pushing it further. Barriers to Enrollment Recent findings indicate significant barriers to enrolling patients remain in clinical trials.

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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.

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Drug Discovery World

APRIL 21, 2023

The event showcased the very latest innovations in cancer therapies, from personalised mRNA vaccines to bispecific antibodies. This is according to results of a Phase I/II clinical trial presented at the AACR Annual Meeting 2023 in Florida. overall response rate.

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Advarra

FEBRUARY 24, 2023

How do you know your experimental therapy is working? But what does “working” really mean when efficacy means something subjective, like quality of life, reduced psychiatric events, or less symptoms? In most cases, the results you are really looking for and trying to prove in a trial can be complex.

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