Fierce BioTech

JULY 25, 2023

How combining datasets from multiple sources can inform and improve commercial strategies Hundreds of rare disease treatments have entered the market over the past decade—thanks largely to a combination of government incentives, strong urging from patient advocacy groups and advances in cell and gene therapies.

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DrugBank

JUNE 13, 2024

These innovative technologies are changing how medications reach their targets within the body, leading to more effective treatments with fewer side effects.   Take cancer treatment, for example. Implantable insulin pumps equipped with continuous glucose monitoring (CGM) sensors can update old diabetes treatment options.

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PPD

OCTOBER 12, 2023

Cell and gene therapies (CGTs) are one of the fastest growing areas in human therapeutics. These treatments use a vector to introduce the desired nucleic acid code to replace or modify protein expression or use cells to alter/restore a specific cell type.

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Fierce BioTech

AUGUST 7, 2024

A Focus on ADC and Radioisotope Therapy pesurya Wed, 08/07/2024 - 11:14 Wed, 10/16/2024 - 14:00 Resource Type Webinar Promotion Start Fri, 08/16/2024 - 11:17 Promotion End Sun, 02/16/2025 - 11:00 Duration 60 Minutes Medical imaging is critical in clinical trials, serving as a key component in assessing treatment efficacy and safety.

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FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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Conversations in Drug Development Trends

JULY 9, 2024

The recent surge in GLP-1 treatments has ignited a profound shift in the pharmaceutical landscape, with more studies being announced than ever before. However, the treatment journey does not end with weight reduction, as maintaining weight loss overall is equally crucial.

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The Pharma Data

MARCH 7, 2022

Astellas”) today announced topline results from the Phase 3 SKYLIGHT 4™ clinical trial investigating the long-term safety of fezolinetant, an investigational oral, nonhormonal compound being studied for the treatment of moderate to severe vasomotor symptoms associated with menopause (VMS) which will support future regulatory filing submissions.

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Drug Target Review

NOVEMBER 23, 2023

This means the neuropeptide network probably uses different strategies to transmit and process information than a more hierarchical wired network does. elegans be applied to the development of targeted therapies for neuropsychiatric conditions like eating disorders, OCD, and PTSD? elegans ?

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Drug Target Review

JULY 12, 2023

Molecular-level biochemical assays like transcriptomics, genomics and proteomics have emerged as valuable tools for identifying potential targets in cancer treatment through deep cyclic inhibition (DCI). Importantly, transcriptomics information shows that certain aspects of a disease change the gene expression profile.

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thought leadership

JUNE 10, 2024

Cell and Gene Therapies (CAGTs) represent a transformative step in medicine, offering unprecedented potential to treat and potentially cure diseases once deemed untreatable. However, the path to accessing these advanced treatments can be daunting.

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Drug Target Review

OCTOBER 25, 2023

Their findings may result in new treatments for reproductive conditions. He stated that this could inform new treatments for reproductive conditions like recurrent pregnancy loss and preeclampsia. Our findings could potentially lead to a cell therapy approach to treat those types of conditions.”

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DrugBank

JULY 8, 2024

Revolutionizing Treatment Through the Skin with Transdermal Drug Delivery Systems The human skin, often perceived as a passive barrier to the external environment, holds potential as a route for drug administration. These fluctuations can lead to unwanted side effects or compromise the effectiveness of the treatment.

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DrugBank

OCTOBER 17, 2024

By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.

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The Pharma Data

APRIL 7, 2022

This unprecedented CMS decision effectively denies all Medicare beneficiaries access to ADUHELM ® (aducanumab-avwa), the first and only FDA-approved therapy in a new class of Alzheimer’s drugs. It may also limit coverage for any future approved treatment in the class. ADUHELM is indicated for the treatment of Alzheimer’s disease.

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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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PPD

JANUARY 5, 2023

In an industry survey led by PPD, Thermo Fisher Scientific’s clinical research business, rare diseases emerged as a top therapeutic area in drug developers’ pipelines, with 39% of global biotech and biopharma leaders pursuing therapies in the broad rare disease space.

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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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Alta Sciences

MARCH 1, 2024

Norbert Makori and Pierre Jolicoeur: The Benefits of Combining Preclinical and Bioanalytical for Your Gene Therapy Studies pmjackson Fri, 03/01/2024 - 22:20 Recent advances in gene therapy have allowed for diseases to be approached differently. q: How can Altasciences support sponsors’ Gene Therapy Programs?

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Covalent Modifiers

APRIL 18, 2024

This breakthrough led to the development and approval of sotorasib (AMG510) and adagrasib (MRTX849), revolutionizing the treatment of KRASG12C-dependent lung cancer. Insights from successful KRASG12C targeting informed the design of molecules addressing other mutations, often in a covalent manner.

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FDA Law Blog: Drug Discovery

MARCH 13, 2023

Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?

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Broad Institute

SEPTEMBER 23, 2024

New approach may yield modified messenger RNAs for treating a wide range of conditions By Ari Navetta September 23, 2024 Breadcrumb Home New approach may yield modified messenger RNAs for treating a wide range of conditions The LEGO framework could help scientists create mRNA therapies that last longer and require smaller doses.

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Olympian Clinical Research

NOVEMBER 9, 2022

Therefore, if you or a loved one is dealing with this rare condition, it’s essential to educate yourself about the condition and its potential treatments. The more you know, the better equipped you’ll be to make informed decisions about your care. Radiation therapy. Bullous Pemphigoid Risk Factors and Causes.

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ASPET

JULY 15, 2024

XELOX therapy, which comprises capecitabine and oxaliplatin, is the standard first-line chemotherapeutic regimen for colorectal cancer. This study aimed to develop a translational PK-TD model using rat data to inform dosing strategies and TD implications in humans.

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PLOS: DNA Science

SEPTEMBER 5, 2024

Diagnosis began to shift from a body-part basis to a molecular one, which I wrote about in “Mutation and location important in cancer treatment” for The Lancet in 2015. These findings can then be used to predict response to treatments as well as survival time.

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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. & Other data include efficacy and safety information, healthcare resource use in patients with AML and MDS, and proof-of-concept of magrolimab on calreticulin in myelofibrosis CD34-positive cells.

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The Pharma Data

MARCH 9, 2022

Investigators at Cedars-Sinai have identified a potential new therapy for COVID-19: a biologic substance created by reengineered human skin cells. Although still in the early stages, the findings open the possibility of having a new therapy for COVID-19 patients.

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The Pharma Data

JULY 8, 2021

ADUHELM is 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. Please see the full Prescribing Information. INDICATION.

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The Pharma Data

DECEMBER 27, 2020

28, 2020 /PRNewswire/ — C-RAD announces today that C-RAD has been selected as the partner to implement surface tracking technology for three proton cancer treatment centers in the US. C-RAD offers a specific version of its Catalyst System for use in proton and particle therapy – Catalyst PT.

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Alta Sciences

SEPTEMBER 13, 2023

Four Pain Models Altasciences Uses to Assess Treatments During Clinical Trials pmjackson Wed, 09/13/2023 - 17:01 September is pain awareness month, and the importance of pain management and continued research into effective analgesics is integral to helping patients suffering from various pain conditions.

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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). For more information, please visit: www.harbourbiomed.com. CAMBRIDGE, Mass.

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The Pharma Data

MARCH 25, 2021

SG is a first-in-class therapy targeting Trop-2, a protein frequently expressed in multiple types of epithelial tumors, such as TNBC, where high expression is associated with poor survival and relapse. Metastatic triple-negative breast cancer is an aggressive and life-threatening cancer.

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The Pharma Data

NOVEMBER 25, 2020

AHA: Ablation Better Than Meds for Initial A-Fib Treatment. 25, 2020 — Cryoballoon ablation is superior to drug therapy as an initial treatment for the prevention of atrial arrhythmia recurrence in patients with paroxysmal atrial fibrillation , according to a study published online Nov. percent in the drug-therapy group.

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The Pharma Data

FEBRUARY 8, 2021

Janssen Announces Treatment with ERLEADA ® (apalutamide) Significantly Improved Overall Survival in Patients with Metastatic Castration-Sensitive Prostate Cancer. In addition, health-related quality of life (HRQoL), per total Functional Assessment of Cancer Therapy–Prostate (FACT-P), continued to be maintained with ERLEADA ®.

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The Pharma Data

MAY 27, 2022

The FDA’s decision is informed by the results of Pfizer’s nonclinical studies and a Phase 2a clinical study of ervogastat/clesacostat, which showed that treatment with ervogastat/clesacostat reduced liver fat with a favorable safety and tolerability profile. Pfizer Inc. NYSE: PFE) today announced the U.S.

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On Medicine

MARCH 8, 2023

The first challenge is analyzing human chemosignals to identify the chemicals carrying the information on the emotional state of the sender. The final outcome of the project is to propose a new human chemosignal-based treatment for social anxiety. To this end, the project is focusing on the emotions of happiness and fear.

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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 ”. John Reed , MD , Ph.

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Common Sense for Drug Policy Blog

JANUARY 4, 2024

Cost Benefit Analysis of Opioid Treatment, Syringe Service Programs, and Test & Treat "Although model projections can only provide estimates of health benefits and costs, such analyses can provide intuition around critical mechanisms and assumptions to inform decision making.

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