KIF1A

MARCH 16, 2024

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. Like hereditary spastic paraplegia, CMT is an umbrella disorder, associated with mutations in over 140 genes, but many diagnosed patients do not know their disease-causing mutation.

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The Pharma Data

SEPTEMBER 29, 2021

to enhance outcomes, new treatments which will target kidney-specific disease mechanisms are highly desired,” said Hiddo L. Christian Rommel, Member of the chief Committee of Bayer AG’s Pharmaceutical Division and Head of Research and Development. 1 in 3 adults is at risk for the disease. In the U.S.,

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Drug Target Review

JULY 11, 2023

In the quest to combat and eradicate diseases, scientists and researchers are increasingly turning to the immense potential of artificial intelligence (AI) as a tool. Her focus initially consisted of preclinical development strategies , clinical development optimisation, target discovery and validation.

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Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

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Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? But commitment to neuroscience drug development is critically important given the significant unmet medical needs and the ways in which patients, caregivers, and families suffer.

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Drug Target Review

DECEMBER 13, 2023

This is likely indicative of the heterogeneous nature of the disorder, and it is this complexity and diversity of clinical presentation and effects across multiple organ systems, that has made efforts to identify genetic risk factors using traditional genomic analysis approaches extremely challenging.

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Drug Target Review

JANUARY 15, 2024

An important limitation of antibodies against tumour antigens is that these agents direct responses to molecular targets present on the surface of the cell. Figure 1: HLA Class I presentation of peptides derived from intracellular antigens. As described below, it also presents challenges and opportunities for TCR-based therapeutics.

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The Pharma Data

JULY 29, 2021

Eisai and Biogen Present Preliminary Assessment of the Clinical Effects of Lecanemab Following 18 Months of Treatment in the Open-Label Extension of the Phase 2 Proof of Concept Study at 2021 Alzheimer’s Association International Conference (AAIC) . United States and virtually from July 26 to 30, 2021 (Presentation No.:

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KIF1A

SEPTEMBER 30, 2023

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. KIF1A-Related Research In the rare disease space, a common and straightforward question is: How rare is the disease? What’s a pre-print?

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The Pharma Data

FEBRUARY 24, 2022

Bayer announced today the initiation of the CONFIDENCE study, a Phase II, three-arm study that will investigate simultaneous initial combination therapy with finerenone and the SGLT2 inhibitor empagliflozin, compared with finerenone alone and empagliflozin alone respectively in patients with chronic kidney disease (CKD) and type 2 diabetes (T2D).

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The Pharma Data

OCTOBER 28, 2021

Bayer will present new renal and cardiovascular (CV) analyses from the comprehensive finerenone (Kerendia®) clinical trial program, including the Phase III FIGARO-DKD and FIDELIO-DKD studies, and the prespecified pooled analysis FIDELITY at the American Society of Nephrology (ASN)’s Kidney Week 2021 from 4-7 November.

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The Pharma Data

JANUARY 10, 2021

Chief Executive Officer of MorphoSys, will present at the 39 th Annual J.P. EST. Live audio of the presentation can be accessed from the Media and Investors section under Conferences on MorphoSys’ website, www.morphosys.com. The presentation as well as a replay of the webcast will also be available on MorphoSys’ website.

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The Pharma Data

MAY 26, 2023

Pfizer Presents Scientific Advancements from its Leading Oncology Portfolio at ASCO 2023 Annual Meeting will present data across its Oncology portfolio and growing pipeline, covering multiple tumor types and novel mechanisms of action at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago from June 2 through June 6.

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Drug Target Review

FEBRUARY 29, 2024

During my academic career, I had the opportunity to examine the underlying causes of neurodegenerative diseases like Parkinson’s disease and develop novel animal models to investigate potential disease-modifying therapeutics. The rigors of training as a physician-scientist present their share of obstacles to overcome.

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The Pharma Data

JULY 23, 2021

(Nasdaq: BIIB) today announced it will share multiple oral and poster presentations from its Alzheimer’s disease clinical development portfolio at the Alzheimer’s Association International Conference (AAIC), which will be held in Denver, Colorado and online July 26-30, 2021.

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Drug Target Review

JUNE 9, 2023

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

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Conversations in Drug Development Trends

APRIL 5, 2024

For others, this rare day is aptly the day we honor rare disease communities. Observed annually on the last day of February since 2008, Rare Disease Day has grown into a global movement for raising awareness, promoting research, and advocating for improved access to rare disease treatments and support services.

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The Premier Consulting Blog

OCTOBER 11, 2022

Clinical trials for ultra-rare diseases can be particularly challenging to mount due to small, geographically-dispersed patient populations. However, acceptance of that data depends on a sponsor’s understanding of the FDA criteria for historical control groups, study data standards, and clinical meaningfulness.

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Drug Target Review

JUNE 3, 2024

How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? By encapsulating specific self-antigens within our biodegradable nanoparticles, we enable the targeted delivery of the CNPs to antigen-presenting cells (APCs) primarily in the spleen and liver.

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The Pharma Data

NOVEMBER 14, 2020

The results were presented at The Liver Meeting Digital Experience (TLMdX), November 13–16, 2020 (Late Breaker #L02). These data offer new insights into potential therapeutic approaches to treating NASH, a disease which currently has limited treatment options.”. About NASH.

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Drug Target Review

DECEMBER 28, 2023

These newly formed mutated proteins are called neoantigens and they are only present on tumour cells and are not found in normal, healthy cells. Findings from a leading-edge study called TRACERx have demonstrated how mutations that occur very early in the process of cancer genesis remain present in all cells of a growing tumour.

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The Pharma Data

SEPTEMBER 16, 2020

New integrated analyses from our tumour agnostic Rozlytrek ® (entrectinib) clinical development programme. Blueprint Medicines will present new data from the registrational phase I/II ARROW trial, investigating Gavreto TM (pralsetinib) for the treatment of people with RET-mutant medullary thyroid cancer.

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The Pharma Data

JULY 28, 2021

The TAK-994 BTD was based, in part, on early phase and preliminary clinical data that indicates Takeda’s investigational oral orexin agonist may demonstrate substantially improved objective and subjective measurements of daytime wakefulness in NT1 patients. 1 Currently, TAK-994 is being studied in an ongoing Phase 2 (TAK-994-1501) study.

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The Pharma Data

APRIL 12, 2022

20% variant allele frequency reduction – At the time of analysis with > 2 year follow up the survival estimate was 100% in patients who had improvements in bone marrow fibrosis or variant allele frequency – Results were presented at the American Association for Cancer Research annual meeting.

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The Pharma Data

APRIL 26, 2022

NYSE: PFE) today reported positive Phase 2 pediatric data for their Lyme disease vaccine candidate, VLA15. Valneva and Pfizer plan to submit these data for publication and presentation at a future scientific congress. About VLA15 VLA15 is the only Lyme disease vaccine candidate currently in clinical development.

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The Pharma Data

OCTOBER 1, 2021

Deucravacitinib demonstrated efficacy no matter baseline characteristics, including weight , disease severity and former treatment with biologic or non-biologic therapies. These data (Presentation #2857) are being presented today during the Late Breaking News Session from 3:45 – 4:45 p.m.

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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. OspA is one of the most dominant surface proteins expressed by the bacteria when present in a tick.

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Vaccine Disease Protein Expression Trials 52

The Pharma Data

OCTOBER 28, 2020

SIGNAL Phase 2 trial data continue to support cognitive benefit and reduced brain atrophy with pepinemab treatment in Huntington’s disease. Data provide strong rationale for continued development in Huntington’s and other slowly progressive neurodegenerative diseases, including Alzheimer’s. ROCHESTER, N.Y., Vaccinex, Inc.

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The Pharma Data

NOVEMBER 4, 2020

5, 2020 /PRNewswire/ — Alligator Bioscience (Nasdaq Stockholm: ATORX) today announced that its CD40 targeting antibody mitazalimab will be presented at the World Immunotherapy Congress, November 2-6, 2020. The presentation will also summarize previously released clinical data from the Phase I study performed by Janssen Biotech Inc.

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Packaging Clinical Development Disease Drugs 40

The Pharma Data

NOVEMBER 5, 2021

The data were presented at the American Society of Nephrology (ASN) 2021 Annual Meeting. With presently no approved treatments, there’s a major unmet need for curatives that can delay progression to order failure, “ said John Tsai, Head of Global Drug Development and Chief Medical Officer at Novartis.

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The Pharma Data

OCTOBER 21, 2020

(Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, today announced that it will present a poster on SYNB8802, an investigational Synthetic Biotic medicine for the treatment of Enteric Hyperoxaluria (HOX), during the American Society of Nephrology (ASN) Kidney Week, Oct.

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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.?. The purpose of the?Ph1

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Trials Disease Clinical Trials Engineering 52

Drug Target Review

APRIL 9, 2024

How has nucleolar stress (NS) been linked to age-related diseases, and what approach did researchers use to investigate its toxicity? The nucleolus (and nucleolar stress) had been previously linked to aging and age-related diseases such as cancer or degeneration.

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Drug Target Review

JULY 20, 2023

Additionally, they compared their long COVID analysis with known genetic associations in various chronic diseases, particularly neurological and metabolic disorders. The combinatorial analysis revealed that 73 genes were significantly associated with long COVID development.

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The Pharma Data

MARCH 9, 2021

New data from 10 studies will be presented by Eli Lilly and Company (NYSE: LLY) at the upcoming virtual International Conference on Alzheimer’s & Parkinson Diseases 2021 (AD/PD 2021), March 9-14, 2021. vice president of Alzheimer’s disease development unit, Eli Lilly and Company.

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LifeSciVC

OCTOBER 10, 2024

At the congress, we were thrilled to share some of the clinical data emerging from our Phase 1 program , an ongoing study evaluating our potent and selective investigational CBL-B inhibitor, HST-1011, in patients with advanced solid tumors. The post ESMO Reflections: Glimmers of Hope with the Next Wave of I-O Therapies?

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The Pharma Data

JULY 12, 2021

Finerenone 10 mg or 20 mg is indicated to reduce the risk of sustained estimated glomerular filtration rate (eGFR) decline, end-stage kidney disease, cardiovascular death, non-fatal myocardial infarction, and hospitalization for heart failure in adult patients with chronic kidney disease (CKD) associated with type 2 diabetes (T2D).

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