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? As a first step, Circio is working on designing and validating circVec-AAV vectors for the treatment of genetic diseases, and testing these head-to-head against the mRNA-AAV equivalent in vivo.

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The Pharma Data

OCTOBER 21, 2020

and Shionogi Limited as shareholders, today announced the positive findings of a pooled analysis of COVID-19-related impacts across the investigational long-acting cabotegravir and rilpivirine clinical development programme. These findings were presented today at the 2020 Infectious Diseases Society of America (IDSA) IDWeek.

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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

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.

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Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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Covalent Modifiers

MAY 9, 2024

BIIB129 (25) demonstrated efficacy in disease-relevant preclinical in vivo models of B cell proliferation in the CNS, exhibits a favorable safety profile suitable for clinical development as an immunomodulating therapy for MS, and has a low projected total human daily dose.

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FDA Law Blog: Drug Discovery

NOVEMBER 2, 2023

Lewis, Senior Regulatory Device & Biologics Expert — On October 20, 2023, FDA announced the availability of the final guidance authored by CBER titled “Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies.” It finalized a draft guidance published in 2022.

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Drug Target Review

OCTOBER 16, 2024

It’s an exciting process—anticipating development risks, predicting outcomes, and aligning these with the greater vision of medical advancement. The true joy comes from navigating these complexities and asking the right questions early on, because these foundational steps are critical to the success of any clinical development process.

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The Pharma Data

AUGUST 24, 2020

The FDA has approved a request from American Gene Technologies to begin a clinical study into its HIV gene therapy. It will investigate the safety, measure key biomarkers and assess surrogate markers of efficacy in its AGT103-T treatment. Conor Kavanagh. Source link.

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The Pharma Data

JANUARY 27, 2021

HK), announced today that China Center for Drug Evaluation (CDE) has granted Breakthrough Therapy designation to Batoclimab (HBM 9161), a fully human anti-FcRn monoclonal antibody (mAb), for the treatment of adult patients with Myasthenia Gravis (MG). CAMBRIDGE, Mass. ROTTERDAM, Netherlands and SUZHOU, China , Jan. ” he added.

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The Pharma Data

AUGUST 31, 2021

today announced a label update for KEYTRUDA, Merck’s anti-PD-1 therapy, for its indication in first-line advanced urothelial carcinoma (bladder cancer) in the U.S. Continued approval was contingent upon verification and description of clinical benefit in confirmatory trials.

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The Pharma Data

JULY 28, 2021

– If Approved, Investigational TAK-994 May Provide a Future Treatment Option Targeting the Orexin Deficiency Underlying NT1. 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.

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The Pharma Data

NOVEMBER 29, 2020

Prior to joining Gannex, Dr. Palmer was Head of Liver Disease Clinical Development at Takeda Pharmaceuticals. She has also held senior leadership positions at Shire Pharmaceuticals and Kadmon Corporation, where she was in charge of the global development of NASH and other liver disease programs. . ” About Ascletis.

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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. In July, finerenone was approved under the name Kerendia® by the us (U.S.)

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The Pharma Data

APRIL 15, 2022

Louis is being advanced toward Phase I human clinical trials to treat COVID-19. Designed computationally and refined in the laboratory, the new protein therapies thwarted infection by interfering with the virus’ ability to enter cells. Food and Drug Administration (FDA). Food and Drug Administration (FDA). 2 omicron subvariant.

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The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics.

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The Pharma Data

MARCH 2, 2021

Boehringer Ingelheim and Gubra today announced a new research and licensing agreement focused on the identification and validation of targets and innovative peptide compounds for the treatment of obesity. iii Current treatment options have limited efficacy in terms of weight loss or are often associated with adverse events. “We

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The Pharma Data

JANUARY 17, 2021

PRIME is a program launched by EMA to optimize development plans and speed up evaluation of medicines that demonstrate major therapeutic advantage over existing treatments, or otherwise benefit patients without treatment options. These results were published in Journal of Clinical Oncology ( Ramos et al., BEDMINSTER, N.J.

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The Pharma Data

APRIL 27, 2022

We plan to discuss these data with health authorities, and we will continue to expand our tislelizumab clinical development program in pursuit of novel, synergistic combinations with the ultimate goal of extending survival for more patients.”. About 108,000 people of more than 140 nationalities work at Novartis around the world.

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The Pharma Data

MARCH 15, 2022

to advance the development of a subcutaneous delivery for Sarclisa ® with the goal of offering a unique patient-centric treatment experience. We are committed to build ing an industry-leading, sustainable pipeline with a steady stream of new therapies that have the potential to transform the practice of medicine ”. In the U.S.,

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FDA Law Blog: Drug Discovery

JUNE 12, 2024

Sasinowski — Hyman, Phelps & McNamara (HPM) would like to congratulate the 7 rare disease programs selected for the inaugural class of the FDA’s “Support for clinical Trials Advancing Rare disease Treatment” (START) pilot program.

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The Pharma Data

JUNE 2, 2022

Boehringer Ingelheim aims to use these antibodies to direct therapeutic effector mechanisms such as antibody-drug conjugates (ADCs) and T-cell engagers exclusively to tumor cells, and to that end develop a range of highly targeted cancer treatments. About Boehringer Ingelheim.

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DrugBank

JUNE 6, 2024

In the rapidly evolving landscape of oncology drug development, artificial intelligence (AI) has emerged as a revolutionary force, altering not just the pace but the very methodology of discovery and development.   What sets RADR ® apart is its robust data-driven approach.

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Drug Target Review

OCTOBER 10, 2024

What are the risks of over-reliance on healthy donor material in early-stage research, and how can decision-makers ensure a balanced integration of diseased material to avoid potential gaps when transitioning to clinical trials? Diseased cells are likely to be more fragile or less abundant, making it difficult to obtain enough for therapy.

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Agency IQ

FEBRUARY 12, 2024

BY RACHEL COE, MSC | FEB 7, 2024 10:21 PM CST The Bespoke Gene Therapy Consortium A collaborative effort, the Bespoke Gene Therapy Consortium (BGTC) was launched in October 2021 to “accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease.”

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The Pharma Data

MAY 15, 2023

FDA accepts application for Roche’s Vabysmo for the treatment of retinal vein occlusion (RVO) Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the U.S. Roche’s Chief Medical Officer and Head of Global Product Development. “If 1,2 Vabysmo’s safety profile was consistent with previous trials. mg) or aflibercept (2.0

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The Pharma Data

DECEMBER 22, 2020

Merck will assist with preclinical development and has the option to exclusively develop, manufacture and commercialize the candidate following Phase 1. A2 anticipates that the successful development of its allogeneic Tmod candidate therapy will enable subsequently developed allogeneic Tmod candidates.

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Sygnature Discovery

NOVEMBER 29, 2023

A new treatment for G4-targeted solid tumors including pancreatic cancer has been administered to patients for the first time in a Phase 1a clinical trial. This latest development follows the US Food and Drug Administration (FDA) giving clearance for QN-302 in July 2023 to proceed to this initial clinical trial stage.

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The Pharma Data

JANUARY 12, 2021

In December, life sciences giant Bayer launched a cell and gene therapy platform within its pharmaceutical division in order to become a leading company within a rapidly emerging and evolving field that offers the potential of life-saving therapies. Photo courtesy of Bayer. We have set the bar high.

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The Pharma Data

JUNE 25, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for lecanemab (BAN2401), an investigational anti-amyloid beta (A?) protofibril antibody for the treatment of Alzheimer’s disease (AD). 1 The proof-of-concept Study 201 explored the impact of treatment with lecanemab on reducing brain amyloid beta (A?)

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Creative Biolabs

MAY 25, 2022

For decades, cytotoxic-based chemotherapy has been the main treatment for all kinds of cancers. Fu, 2022) Clinical research progress of ADC After decades of efforts, more than 100 ADCs are currently under clinical development. These cytotoxic drugs include DNA base analogs, antimetabolic drugs, tubulin inhibitors, etc.

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The Pharma Data

MAY 2, 2022

The companies expect to complete submission of the NDA for treatment of MDD in the second half of 2022; associated filing for postpartum depression anticipated in the first half of 2023. Food and Drug Administration (FDA) for zuranolone in the treatment of major depressive disorder (MDD). Sage Therapeutics, Inc.

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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. In 2020, 78 percent of orphan drugs and biologics approved were targeted therapies.

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KIF1A

SEPTEMBER 30, 2023

Read the Article Both of these studies reflect a global effort to understand the genetic underpinnings of disorders so people can find the right treatments sooner. In addition to benefiting awarded sponsors, this pilot program will help create best practices of communication with the FDA during rare disease therapeutic development.

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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. About EMPEROR-Preserved.

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Drug Target Review

AUGUST 24, 2023

We will explore how GDF-15 impacts the tumour microenvironment and hinders the infiltration of T cells into the tumour, as well as the implications of neutralising GDF-15 to reverse its inhibitory effects and sensitise tumours to anti-PD-1 treatment. T-cell infiltration is a prerequisite for responses to checkpoint inhibitors.

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The Pharma Data

DECEMBER 13, 2020

NRG1 fusions are a rare genetic mutation that are increasingly recognized as a driver of multiple tumor malignancies, and an actionable target for HER3 targeted therapy. Up to 1% of all solid tumors harbor NRG1 fusions, therefore, it is important to identify this patient population and develop therapies that can treat them. [1].

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