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.

Disease 52

Disease Clinical Trials FDA Approval FDA 52

The Pharma Data

MARCH 22, 2021

Basel, 22 March 2021 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced the decision to discontinue dosing in the Phase III GENERATION HD1 study of tominersen in manifest Huntington’s disease (HD). In December 2017, Roche licensed the investigational molecule from Ionis Pharmaceuticals. About Huntington’s disease.

Disease 52

Disease Clinical Trials Pharmacokinetics Treatment 52

Drug Target Review

NOVEMBER 7, 2024

Introduction Therapeutic antibodies have proven to be indispensable medicines for addressing the most debilitating diseases. Agonist antibodies in immunological diseases Agonist antibodies targeting immune checkpoint pathways are increasingly recognised and investigated for their potential in immunological diseases.

Disease 52

Disease Immune Response Regulations Engineering 52

The Pharma Data

DECEMBER 15, 2020

A healthy immune system defends the body against disease and other conditions. Autoimmune disease impacts different parts of the body, weakening functionality. Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. It may be life-threatening.

Disease 52

Disease Clinical Trials Small Molecule Trials 52

Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. This approach can be highly targeted, with the potential to correct mutations that lead to specific diseases or deliver therapeutic genes to improve heart function.

Therapies 98

Therapies Disease RNA Engineering 98

Drug Target Review

AUGUST 16, 2023

Furthermore, it successfully distinguished early-stage cancer samples from those with common diseases, laying the foundation for a robust data platform for further in-depth analysis. This in-depth understanding of the disease could aid in selecting targeted therapies for individual patients, further enhancing treatment outcomes.

DNA 98

DNA Disease Therapies Treatment 98

Broad Institute

OCTOBER 11, 2023

He also read everything he could about her disease, including emerging evidence that suggested that the immune system could recognize and kill Merkel cell carcinoma. His mother had a presentation of the disease that suggested her immune system was already on the job. This is what we need for patients,” Manguso said.

Research 137

Research Immune Response Clinical Trials Therapies 137

The Pharma Data

MAY 26, 2023

More ways, to save more lives, for less money: World Health Assembly adopts more Best Buys to tackle noncommunicable diseases The World Health Organization has expanded the list of ‘NCD best buys’. The report is part of the NCDs Global Action Plan 2023-2030 and is an update from 2017 and is core to the Implementation Roadmap on NCDs.

Disease 52

Disease Government Treatment Production 52

The Pharma Data

MARCH 8, 2021

Valneva SE (“Valneva”), a specialty vaccine company focused on prevention of diseases with major unmet needs, and Pfizer Inc. We are excited to be part of the Lyme disease vaccine development program with Valneva. Food and Drug Administration (FDA) in July 2017 3. About Lyme Disease. burgdorferi naïve volunteers.

Vaccine 52

Vaccine Disease Protein Expression Trials 52

Broad Institute

SEPTEMBER 15, 2023

We apply STACI to analyze the spatio-temporal progression of Alzheimer’s disease and identify the associated nuclear morphometric and coupled gene expression features.

Disease 40

Disease 40

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.

Drug Development 52

Drug Development Disease Therapies Engineering 52

New Drug Approvals

OCTOBER 12, 2024

Alzheon licensed ALZ-801 from NeuroChem and is developing it for Alzheimer’s disease. ALZ-801 is an advanced and markedly improved candidate for the treatment of alzheimer’s disease. Clinical Pharmacokinetics and Safety of ALZ-801, a Novel Prodrug of Tramiprosate in Development for the Treatment of Alzheimer’s Disease.

Pharmacokinetics 62

Pharmacokinetics Small Molecule Disease Licensing 62

The Pharma Data

MARCH 16, 2022

(Nasdaq: BIIB) announced that The Journal of Prevention of Alzheimer’s Disease (JPAD) today published a peer-reviewed manuscript detailing data from the pivotal Phase 3 EMERGE and ENGAGE trials for ADUHELM ® (aducanumab-avwa) 100 mg/mL injection for intravenous use in early Alzheimer’s disease.

Disease 52

Disease Clinical Trials Trials Treatment 52

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. 2017 Jan;155(1):234–47. 2017 Jul 6;21(1):14–7. Using stem cell-derived neurons in drug screening for neurological diseases. 2017 Feb;16(2):115–30. Toxicol Sci.

Drugs 111

Drugs FDA Disease Animal Testing 111

New Drug Approvals

NOVEMBER 13, 2023

PATENT WO2023214314 VODOBATINIB FOR REDUCING PROGRESSION OF PARKINSON’S DISEASE (wipo.int) Vodobatinib (N’-(2-chloro-6-methylbenzoyl)-4-methyl-3-[2-(3-quinolyl) ethynyl]-benzohydrazide), a c-Abl inhibitor is represented by Formula I (referred hereinafter interchangeably as vodobatinib or compound of Formula International Publication Nos.

Treatment 62

Treatment Disease Therapies Production 62

The Pharma Data

JULY 21, 2021

Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company focused on the development and commercialization of prophylactic vaccines for infectious diseases with significant unmet medical need, and Pfizer Inc. Food and Drug Administration (FDA) in July 2017 3. About Lyme Disease. Juan Carlos Jaramillo M.D.,

Vaccine 52

Vaccine Disease Trials Protein Expression 52

The Pharma Data

APRIL 7, 2022

These coverage restrictions, including the distinction between accelerated approval and traditional approval, have never been applied to FDA-approved medicines for other disease areas. ADUHELM is indicated for the treatment of Alzheimer’s disease. It may also limit coverage for any future approved treatment in the class. About Biogen.

Treatment 52

Treatment Therapies Disease FDA Approval 52

Drug Target Review

AUGUST 9, 2023

Published on bit.bio’s website, this article authored by Dr Tony Oosterveen dives into the close relationship between FTD and ALS with a focus on mechanisms underlying the neuropathology of both diseases. ALS is the most common motor neuron disease and is estimated to affect more than 80,000 people worldwide.

RNA 98

RNA Regulations Disease DNA 98

The Pharma Data

MARCH 16, 2022

These are meaningful findings, which further our understanding of amyloid and downstream biomarkers, such as p-tau 181, in Alzheimer’s disease and can help inform how long patients may benefit from treatment to reduce amyloid beta plaque,” said Samantha Budd Haeberlein, Ph.D., SVP, Head of Neurodegeneration Development at Biogen.

Disease 52

Disease Clinical Trials Treatment Trials 52

Drug Target Review

APRIL 19, 2024

And so, I have been the managerial co-founder and CEO of Sibylla since 2017. The scientific founders have developed a pioneering method for drug discovery that applies to the research and design of new drugs effective against diseases that are incurable to date. It still blows my mind to think about it.

Molecular Biology 111

Molecular Biology Research Pharmaceuticals Small Molecule 111

Drug Target Review

OCTOBER 17, 2023

Metabolic changes and tumourigenicity Cancer is a multi-faceted disease which has previously been seen as a disorder in cell proliferation, the increase in cell numbers resulting from cell division. 2017 July [cited 2023 October 9]; 3(2):45-51. Waltham, MA: Academic Press, Imprint of Elsevier; 2014 [cited 2023 October 11] p.761-5.

Regulations 98

Regulations Therapies Treatment Disease 98

The Pharma Data

SEPTEMBER 29, 2021

In 2017, an alarming 1.2 to enhance outcomes, new treatments which will target kidney-specific disease mechanisms are highly desired,” said Hiddo L. to enhance outcomes, new treatments which will target kidney-specific disease mechanisms are highly desired,” said Hiddo L. 1 in 3 adults is at risk for the disease.

Clinical Development 52

Clinical Development Disease Clinical Trials Trials 52

Drug Target Review

SEPTEMBER 4, 2023

There have been several studies that have reported the ability of infectious disease agents to be drug resistant. Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. They could be spread by physical contact, airborne, waterborne, foodborne, or vector-borne.

Drugs 111

Drugs RNA DNA Disease 111

KIF1A

JUNE 17, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. #ScienceSaturday posts share exciting scientific developments and educational resources with the KAND community. What’s a pre-print?

Laboratories 98

Laboratories Therapies Disease Research 98

Drug Target Review

APRIL 25, 2024

A 2017 study, conducted by University of Southern California and National Institute of Health scientists, identified seven smoking-associated hypomethylated CpGs, with five of these loci having been previously reported as hypomethylated in smoker’s blood. Characterising them can give us a better understanding of disease relevance and risk.”

DNA 64

DNA Disease Science Research 64

Drug Target Review

OCTOBER 26, 2023

1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases. The Lancet 390 , 849-860 (2017). Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. Wang D, Tai PW, Gao G.

Engineering 105

Engineering Virus Therapies Immune Response 105

Conversations in Drug Development Trends

AUGUST 10, 2023

As one of the more recent fields to emerge in the clinical space, psychedelics have gained significant traction as a potential treatment for various neuropsychiatric diseases. Psychedelic Research at Worldwide Since 2017, Worldwide Clinical Trials have supported a myriad of sponsors leading the way in psychedelic research programs.

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Science Clinical Trials Trials Clinical Research 98

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. How are disease targets selected for challenge trials?

Vaccine 111

Vaccine Trials Disease Treatment 111

Drug Target Review

JANUARY 15, 2024

Cancer is a disease driven by variable genetic mutations. Dr Becker cofounded Onchilles Pharma in 2017 to translate breakthrough research in neutrophil biology into an entirely new treatment paradigm with the potential for pan-cancer killing activity.

Treatment 105

Treatment Immune Response Therapies Clinical Trials 105

The Pharma Data

NOVEMBER 2, 2020

adults by using blood pressure (BP) thresholds from the Seventh Report of the Joint National Committee on Prevention, Detection, Evaluation, and Treatment of High Blood Pressure (JNC7) and the 2017 American College of Cardiology-American Heart Association (ACC-AHA) BP guidelines. adults) using the 2017 ACC-AHA guideline criteria.

Pharmaceuticals 52

Pharmaceuticals Treatment Disease Research 52

DrugBank

JUNE 27, 2023

Out of the millions of genetic variants present in human populations, GWAS are designed to uncover those associated with specific traits or diseases. 9 Polygenic diseases are much more common, and thousands of genetic variants with very small effects could impact their phenotype.

Disease 87

Disease Treatment Drugs Clinical Trials 87

The Pharma Data

OCTOBER 20, 2020

20, 2020 — In 2017 to 2019, 65.3 Centers for Disease Control and Prevention National Center for Health Statistics. percent of women aged 15 to 49 years in the United States were currently using contraception in 2017 to 2019. TUESDAY, Oct. Kimberly Daniels, Ph.D., and Joyce C. Abma, Ph.D., The researchers found that 65.3

Disease 52

Disease Research 52

The Pharma Data

APRIL 25, 2021

Nirsevimab demonstrate d protect ion against respiratory syncytial virus disease in healthy infants in Phase 3 trial. It is designed for use in a broad infant population, including all infants experiencing their first RSV season and infants with congenital heart disease or chronic lung disease entering their first and second RSV season.

Virus 40

Virus Trials Disease Hospitals 40

Agency IQ

MAY 17, 2024

The Creating Hope Reauthorization Act would reauthorize the FDA’s Rare Pediatric Disease Priority Review Voucher program. The markup hearing comes three months after the E&C Committee last held a hearing on 18 bills related to rare diseases, including all four of the above-mentioned bills. DORIS MATSUI (D-Calif.)

FDA 40

FDA Disease FDA Approval Production 40

The Pharma Data

JANUARY 11, 2021

The agency previously granted the gene therapy Orphan Drug and Rare Pediatric Disease designations in 2017. The trial plans to enroll 99 boys aged 4 to 7 years across 55 sites in 15 countries. PF-06939926 received Fast-Track designation from the FDA in October.

Therapies 52

Therapies Trials FDA Treatment 52

The Pharma Data

AUGUST 9, 2020

Pivotal phase III stud y of etrolizumab in Crohn’s disease is ongoing. Roche is studying additional investigational medicines in inflammatory bowel diseases. We are fully analysing these data to learn more about how we might address the needs of people with this devastating disease.

Clinical Trials 52

Clinical Trials Disease Treatment Trials 52

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. CAR-NK cell trials only commenced in 2017 and most are still recruiting patients. NK cells can be readily extracted from umbilical cord blood or peripheral blood of adult donors.

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Therapies Cell Biology Clinical Trials Trials 118