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.

Engineering 110

Engineering DNA Virus Immune Response 110

Drug Target Review

JANUARY 8, 2024

Contrary to popular belief, ageing is not caused by just random wear and tear of our bodies over time but is instead caused by a discrete set of biological mechanisms that we now better understand and can target with therapies. a clinical-stage private biopharma company developing therapies for neurological and psychiatric diseases.

Science 145

Science Disease FDA Approval Clinical Trials 145

Drug Target Review

MAY 22, 2024

How does Circio’s circVec technology aim to enhance the potency and reduce the cost of current gold-standard gene therapy? There are three major reasons for why Circio has selected AATD as its lead gene therapy program. Firstly, there have been previous AAV gene therapy clinical studies in this indication that have shown promise.

Therapies 59

Therapies RNA Protein Expression DNA 59

The Pharma Data

JUNE 2, 2023

Gilead and Kite Oncology Demonstrate Growing Hematology Pipeline and Strength of Leading Cell Therapy Portfolio at EHA FOSTER CITY, Calif. & & SANTA MONICA, Calif.–(BUSINESS –(BUSINESS WIRE)– Gilead Sciences, Inc.

Therapies 52

Therapies Clinical Development Treatment Trials 52

Drug Target Review

DECEMBER 13, 2024

These types differ in their etiology, natural history, and present distinct challenges in disease management. 2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates.

Therapies 52

Therapies Treatment Clinical Trials Drugs 52

Drug Target Review

FEBRUARY 29, 2024

Through further research and work as a principal investigator on clinical studies for several central nervous system indications, I laid the groundwork for a career in gene therapy drug development. AskBio) I continue to work to bring innovative gene therapies to patients in need.

Therapies 111

Therapies Disease Clinical Trials Science 111

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).

Clinical Development 52

Clinical Development Therapies Disease Treatment 52

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.

Biosimilars 52

Biosimilars Clinical Development Science Therapies 52

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.

Clinical Trials 52

Clinical Trials Clinical Development Clinical Supply Trials 52

The Pharma Data

JANUARY 21, 2021

.–( BUSINESS WIRE )– Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, today announced an oral presentation on updated efficacy, safety, and dosing management of poziotinib from Cohorts 1 and 2 of the ZENITH20 clinical trial.

Pharmaceuticals 52

Pharmaceuticals Clinical Trials Licensing Licensing 52

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.:

Clinical Trials 52

Clinical Trials Treatment Clinical Development Disease 52

The Pharma Data

JULY 28, 2021

Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) to TAK-994, 1 its Phase 2 investigational oral orexin agonist, which is designed to selectively target orexin 2 receptors. 2 Data from the completed Phase 2 study will be presented at a future scientific conference after the study’s conclusion.

Clinical Development 52

Clinical Development Therapies Laboratories Treatment 52

KIF1A

SEPTEMBER 30, 2023

Read the Guide FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies One frustrating aspect of traditional drug development, especially for rare disease communities, is the tempo of regulatory decisions on potential drugs. What’s a pre-print?

Disease 111

Disease FDA Clinical Trials Therapies 111

Conversations in Drug Development Trends

JANUARY 24, 2024

Much of what we discuss I covered in my presentation, “Leveraging Genetics to Support Rare Disease Clinical Trials,” at last year’s World Orphan Drug Congress (WODC) EU. One diagnostic example that I discussed in my presentation is autism. Enrolling adequate populations will be required to advance these programs.

Trials 75

Trials Disease Clinical Trials Clinical Research 75

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.

Clinical Trials 52

Clinical Trials Trials Disease Hospitals 52

Drug Target Review

JUNE 6, 2023

We are in an era of immuno-oncology (IO) revolution with many approved therapies now available to treat a broad range of cancers. In the context of a tumour microenvironment, Tregs are often present in high numbers, preventing an effective immune response to the tumour. Efficacy at tolerated doses so far has been disappointing.

Engineering 98

Engineering Immune Response Biochemical Pharmacology Clinical Trials 98

The Pharma Data

SEPTEMBER 29, 2021

Patients are going to be randomized to receive either finerenone 10mg or 20mg or placebo on top of individually tolerated maximum labeled doses of a renin-angiotensin system (RAS)-blocking therapy like an angiotensin-converting enzyme (ACE) inhibitor or an angiotensin II receptor blocker (ARB).

Clinical Development 52

Clinical Development Disease Clinical Trials Trials 52

The Pharma Data

NOVEMBER 9, 2021

We ’re agitated to partake data from our robust clinical development programs at AASLD’s The Liver Meeting ®, including the rearmost data demonstrating the positive impacts of bulevirtide for people living with HDV.”. For more than 20 years, Gilead has sought to address some of the biggest challenges in liver disease.

Disease 52

Disease Clinical Trials Treatment Pharmacokinetics 52

Broad Institute

OCTOBER 11, 2023

His mother had a presentation of the disease that suggested her immune system was already on the job. But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way.

Research 137

Research Immune Response Clinical Trials Therapies 137

The Pharma Data

OCTOBER 26, 2020

Nasdaq: ARDS), a biopharmaceutical company focused on the discovery and development of novel anti-infective therapies to treat life-threatening infections, today announced the Company will present at the ROTH Capital Partners 2020 MedTech Innovation Forum on Wednesday, October 28, 2020. SAN JOSE, Calif. , AR-301 (VAP).

Pharmaceuticals 52

Pharmaceuticals Virus Hospitals Clinical Trials 52

The Pharma Data

JUNE 2, 2022

New multi-program collaboration to develop allogeneic TCR-T/CAR-T programs brings together Immatics’ allogeneic gamma delta T cell therapy platform ACTallo ® with Bristol Myers Squibb’s technologies and oncology drug development expertise. Executive Vice President, Research & Early Development, Bristol Myers Squibb. “We

Therapies 52

Therapies Production Engineering Clinical Development 52

Drug Target Review

APRIL 3, 2024

I am currently working as Chief Scientific Officer at CatalYm, where we translate the latest scientific findings into clinical applications. Our company has identified a new role for Growth Differentiation Factor 15 (GDF-15) as a potent local immunosuppressor mediating cancer resistance to therapy.

Science 105

Science Clinical Development Treatment DNA 105

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.

Trials 52

Trials Treatment Disease Pharmaceuticals 52

LifeSciVC

JULY 11, 2023

Redig, SVP and Head of Clinical Development at HotSpot Therapeutics, as part of the From The Trenches feature of LifeSciVC The American Cancer Society for Oncology (ASCO) annual meeting is among the highlights of the clinical oncology calendar. By Amanda J.

Therapies 66

Therapies Trials Clinical Development Science 66

The Pharma Data

NOVEMBER 14, 2020

The results were presented at The Liver Meeting Digital Experience (TLMdX), November 13–16, 2020 (Late Breaker #L02). The companies are also presenting preclinical data supporting the development of combination approaches in NASH. We are now carefully evaluating next steps together based on a thorough assessment of data.”.

Trials 52

Trials Science Treatment Disease 52

The Pharma Data

JANUARY 27, 2021

. A total of 15 patients have been recruited to date in the Phase I dose escalation part of the study, all of whom were late stage and have failed conventional treatments, including several lines of rituximab-containing therapies. ET ) to discuss the results and next steps in clinical development of BI-1206.

Pharmaceuticals 52

Pharmaceuticals Clinical Trials Licensing Licensing 52

The Pharma Data

APRIL 11, 2023

Bayer is advancing its oncology R&D efforts in three scientific areas that have the potential to address unmet needs in cancer patients: next-generation Immuno-Oncology, Targeted Radionuclide Therapies and Precision Molecular Oncology. Data from all three areas of scientific focus will be showcased during this year’s meeting.

Research 40

Research Clinical Trials Small Molecule Trials 40

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.

Medical Schools 52

Medical Schools Trials Disease Clinical Development 52

The Pharma Data

JANUARY 14, 2021

(NASDAQ: FPRX) today announced clinical results from the global, randomized, double-blind placebo-controlled Phase 2 FIGHT trial evaluating first-in-class targeted therapy bemarituzumab in advanced gastric or gastroesophageal junction (GEJ) cancer. The ASCO GI presentation slides are available on the company’s website.

Trials 52

Trials Treatment Therapies Clinical Trials 52

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.

Bioinformatics 145

Bioinformatics Disease Regulations Immune Response 145

The Pharma Data

SEPTEMBER 11, 2021

– Designation is based on results from the EMPEROR-Preserved phase III trial, which established Jardiance as the first therapy to show statistically significant improvement in heart failure outcomes in adults with heart failure with preserved ejection fraction. vice president, Product Development, Lilly.

Therapies 52

Therapies FDA Hospitals FDA Approval 52

SugarCone Biotech

JUNE 10, 2024

[link] Immunity is complex and can be dangerous when exploited clinically, as demonstrated by the lethal administration of TNF, or anti-CD40L antibody (Biogen) or CAR-T cells expressing the CD16 Fc-receptor (Unum), among many other examples. This observation may be important given the critical role of DC in tumor antigen presentation.

Immune Response 88

Immune Response Production Regulations Clinical Trials 88

Conversations in Drug Development Trends

DECEMBER 20, 2023

In November 2023, at Outsourcing Clinical Trials Dach in Zurich, our Executive Director, Oncology Strategy Lead, Matt Cooper , presented “Delivering Oncology Studies – Challenges and Considerations.”

Clinical Trials 83

Clinical Trials Trials Pharmacokinetics FDA 83

The Pharma Data

SEPTEMBER 16, 2020

executive vice president of Research and Development at Amgen, along with members of Amgen’s clinical development team and clinical investigators, will discuss Phase 1 data being presented on the Company’s investigational KRAS G12C inhibitor sotorasib (AMG 510). Reese , M.D., On Monday, Sept.

Clinical Development 52

Clinical Development Therapies Disease Research 52

Drug Target Review

MAY 7, 2024

Small patient populations often result in difficulty in determining a significant effect of a therapy, given the low confidence power associated with the statistics. However, some biomarkers may only be present in a minority of patients, requiring a large patient population to identify such markers.

Disease 59

Disease Clinical Trials Clinical Pharmacology Trials 59

The Pharma Data

OCTOBER 25, 2020

Real-world effectiveness data on Oxbryta ® (voxelotor) in the treatment of sickle cell disease featured as oral presentation. Two abstracts have been accepted for presentation, including real-world effectiveness data of Oxbryta ® (voxelotor) tablets in the treatment of sickle cell disease (SCD). SOUTH SAN FRANCISCO, Calif., .

Treatment 40

Treatment Disease FDA FDA Approval 40

Drug Target Review

APRIL 9, 2024

Our approach to investigate the toxicity driven by nucleolar stress was to induce it with certain arginine-rich peptides, present in patients of ALS, which were known to accumulate at nucleoli and cause this type of stress. Among other discoveries, the group developed inhibitors of the ATR kinase and showed their potential for cancer therapy.

RNA 64

RNA Cell Biology Clinical Development DNA 64