Drug Target Review

NOVEMBER 1, 2023

2 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. 1-5 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. Mediators of Inflammation. 2017;2017:1–15.

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The Pharma Data

MAY 31, 2022

the completion of enrollment of its Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease (PD). Ph1 clinical trial is to evaluate the safety, tolerability, and?preliminary?efficacy?of of BRT-DA01 in patients with Parkinson’s disease.?. preliminary?efficacy?of

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Codon

JANUARY 21, 2024

Today, refined versions of these human challenge studies have become standard practice in testing vaccines for vector-borne diseases (e.g., yellow fever, malaria, and dengue), evaluating new drugs or treatments, and studying pathogenesis, the process by which a disease develops. Why are challenge trials becoming more popular?

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Drug Target Review

OCTOBER 25, 2024

How does precision neuroscience differ from traditional approaches in the treatment of neurodegenerative diseases, and what advantages does it offer? Traditionally, Alzheimer’s disease is diagnosed by clinical examination, medical history and cognitive testing. We believe we are at a breakthrough moment in Alzheimer’s disease research.

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The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

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PPD

NOVEMBER 7, 2023

Food and Drug Administration (FDA) draft guidance, “Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials.” The issue of including diverse populations that represent disease prevalence and country and regional demographics is not new to industry.

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Clinical Trials Trials Clinical Research Disease 59

PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

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Advarra

AUGUST 16, 2022

A rare, or orphan, disease by definition affects a small percentage of the population — fewer than 200,000 people in the U.S. But the numbers add up, and taken together, rare diseases impact an estimated 30 million Americans. Since then, the FDA has significantly changed its approach to rare and orphan diseases.

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Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Up until 2023 around 600 NK cell clinical trials have been reported and some 200 are clinically active and still recruiting patients.

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The Pharma Data

APRIL 7, 2021

Sutimlimab, a first-in-class investigational C1s inhibitor, has the potential to be the first approved treatment for hemolysis in adults with cold agglutinin disease, a serious and chronic autoimmune hemolytic anemia. Sutimlimab has the potential to address a major unmet medical need for people with cold agglutinin disease.”.

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Conversations in Drug Development Trends

FEBRUARY 22, 2023

Rare Disease Day on February 28th is a chance for rare disease communities to come together as one and join in conversation with stakeholders searching for treatments, raising money to support research, and changing the way healthcare and governments interact with patients. In my case, my daughter Iris has GM1 gangliosidosis.

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The Pharma Data

AUGUST 22, 2021

The trial accrued 25 cases of symptomatic COVID-19 at the primary analysis.There were no cases of severe COVID-19 or COVID-19-related deaths in those treated with AZD7442. The trial included 5,197 participants in a 2:1 randomisation AZD7442 to placebo. The trial was conducted in 87 sites in the US, UK, Spain, France and Belgium.

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The Pharma Data

APRIL 3, 2021

The CoVIg-19 Plasma Alliance today announced that the Phase 3 Inpatient Treatment with Anti-Coronavirus Immunoglobulin (ITAC) clinical trial sponsored and funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), did not meet its endpoints. About the ITAC Trial.

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The Pharma Data

NOVEMBER 12, 2020

The DETOUR1 trial is the first-in-man trial of the FDA-designated Breakthrough Device, The Detour System, evaluating the safety and effectiveness of the novel procedure and device system for treating long, complex lesions in the superficial femoral artery (SFA). An illustration of a completed Detour Procedure (Graphic: Business Wire).

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New Drug Approvals

SEPTEMBER 29, 2024

gingivalis has been called a risk factor for Alzheimer’s disease ( Kanagasingam et al., gingivalis DNA was detected in postmortem cortices from people with AD and healthy controls, and in CSF of AD patients ( Jan 2019 news on Dominy et al., gingivalis is associated with cardiovascular disease.

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The Pharma Data

JANUARY 5, 2021

RAD011 is a pivotal-trial ready synthetic cannabidiol oral solution with potential utilization in multiple endocrine and metabolic orphan diseases. Acquisition and associated PWS trial costs to be funded from existing operations. Disease Highlights. Davis, executive chairman of Benuvia.

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The Pharma Data

DECEMBER 5, 2020

.–(BUSINESS WIRE) December 5, 2020 — Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, today announced new clinical data from its Phase 2 trial evaluating SY-1425, its first-in-class selective retinoic acid receptor alpha (RAR?)

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The Pharma Data

JANUARY 19, 2021

The Phase 1 clinical trial is planned to be conducted in Canada and targeted to recruit up to 48 and 24 healthy volunteers for the single-ascending dose (SAD) and multiple- ascending dose (MAD) cohorts, respectively. About ALS-4.

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Small Molecule Clinical Trials Trials Pharmacokinetics 52

The Pharma Data

NOVEMBER 12, 2020

12 November 2020 — The CALAVI Phase II trials for Calquence (acalabrutinib) in patients hospitalised with respiratory symptoms of COVID-19 did not meet the primary efficacy endpoint. No new safety signal for Calquence was observed in the trials. No new safety signal for Calquence was observed in the trials.

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The Pharma Data

JULY 22, 2021

NOTE: ADUHELM TM (aducanumab-avwa)injection 100 mg/mL solutionis indicated for the treatment of Alzheimer’s disease. Treatment with ADUHELM should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials. Biogen Inc.

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FDA Law Blog: Drug Discovery

OCTOBER 2, 2023

Just as the original 1962 efficacy standard makes scientific judgment central to a determination of substantial evidence, the 1997 amendment makes scientific judgment central to a determination of what constitutes sufficient confirmatory evidence to substantiate a single adequate and well-controlled trial.

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The Pharma Data

JANUARY 17, 2021

n-Lorem Foundation has taken on a challenge that many nations consider too great: treating patients with ultra-rare diseases (which affect 30 or fewer people) for free, for life. n-Lorem selects patients based on their genetic mutation and the organ affected rather than their specific diseases. “We We’re the middleman,” he said.

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Disease Clinical Trials Small Molecule Treatment 52

Drug Target Review

APRIL 26, 2023

Gene-editing methods allow researchers to modify DNA, resulting in alterations in physical characteristics such as eye colour and susceptibility to diseases. 1 RNA-delivery particles have the potential to revolutionise the treatment of lung diseases such as cystic fibrosis.

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Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Now, FDA’s CBER is setting its sights on making ambitious strides toward tackling rare diseases.

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Broad Institute

JANUARY 30, 2024

Bhattacharyya wears many coats today, splitting his time between the Broad and the infectious disease ward at MGH, where a diagnostic method he developed at Broad is now being tested to see if it helps patients quickly receive the best antibiotics for their infections. million deaths attributed directly to resistant infections.

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The Pharma Data

DECEMBER 29, 2020

As in earlier outpatient trial, immune status when patients entered the trial was a strong predictor of viral load and clinical outcomes. The primary clinical objective of this initial analysis was to determine if there was sufficient efficacy in these patients to warrant continuing the trial (i.e., futility analysis).

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Drug Target Review

SEPTEMBER 26, 2023

Human derived induced pluripotent stem cells (hiPSCs) have revolutionised research and are increasingly used for toxicology screening and disease modelling. Dr Rockley previously completed an MSc in Cancer Pharmacology and have experience in clinical research from an oncology clinical trial coordinator role. 2019 Mar 1;55:93–100.

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Drug Target Review

SEPTEMBER 21, 2023

Some report that they believe that if they don’t take the medication, they may develop onchocerciasis-related severe skin diseases and may go blind (river blindness) and would prefer a treatment that does not repeat every year. Need for adulticidal drugs More than 99 percent of countries worldwide has been affected by this disease.

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The Pharma Data

OCTOBER 1, 2021

Results from the Phase 3 POETYK PSO-1 and POETYK PSO-2 trials show that response rates for deucravacitinib continued to extend through Week 24 and were maintained through Week 52 in patients with moderate to severe plaque psoriasis. CESTidentified through 52 weeks of knowledge. million people within the us.

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The Pharma Data

AUGUST 9, 2021

Positive high-level results from the head-to-head DESTINY-Breast03 Phase III trial showed that Enhertu (trastuzumab deruxtecan), the AstraZeneca and Daiichi Sankyo Company, Limited (Daiichi Sankyo) HER2-directed antibody drug conjugate (ADC), demonstrated superiority over trastuzumab emtansine (T-DM1).

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The Pharma Data

JANUARY 31, 2021

Clover plans to initiate a global Phase 2/3 efficacy trial of its protein-based S-Trimer COVID-19 vaccine candidate adjuvanted with Dynavax’s CpG 1018 plus alum in the first half of 2021 with an interim analysis for vaccine efficacy potentially in the middle of 2021. EMERYVILLE, Calif. and CHENGDU, China , Feb.

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The Pharma Data

JANUARY 17, 2021

The treatment could be administered in primary care centers to people who test positive for COVID-19, avoiding hospitalization due to the progression of the disease and complementing the vaccine in the early phase after vaccination. BARCELONA, Spain , Jan. 18, 2021 /PRNewswire/ — Grifols (MCE: GRF, MCE: GRF.P,

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The Pharma Data

JANUARY 13, 2021

Fast Track designation is well-timed, as we anticipate starting our Phase 2 clinical trial in hospitalized COVID-19 patients this month, and should help bring Brilacidin to patients faster in these dire times.”. in July 2019, who have recently initiated a Phase 1 study with their formulation.

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The Pharma Data

JANUARY 11, 2021

In clinical trials, it could detect COVID-19 with 90%+ accuracy in about two minutes. . By the end of January, Johnson & Johnson is expected to deliver preliminary trial results for its one-dose COVID-19 vaccine candidate. . DalCor Pharmaceuticals initiated the Phase II dal-COVID trial of dalcetrapib for COVID-19.

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The Pharma Data

MARCH 15, 2022

The sBLA submitted to the FDA by AstraZeneca included data from the OSTRO Phase III trial, which met both co-primary endpoints with a safety profile consistent with the known profile of the medicine. The Company remains committed to bringing Fasenra to patients with CRSwNP and a second Phase III trial, ORCHID , in this indication is ongoing.

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The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Launched in 2019, the company raised $700 million in June 2020 in its initial financing. Harr was also a director for Loxo Oncology.

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The Pharma Data

JANUARY 3, 2021

InspirMed is a newly established subsidiary of TLC specializing in the development of inhalable liposome formulation programs, such as TLC19 for the treatment of Coronavirus disease 2019 (COVID-19) as well as other programs for severe acute and chronic pulmonary diseases. Cautionary Note on Forward-Looking Statements.

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