Conversations in Drug Development Trends

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment.

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Drug Target Review

SEPTEMBER 22, 2023

Organoid technologies are becoming an invaluable solution for preclinical research, with the ability to augment the development of personalised medicine, drug discovery and gene therapies. Organoids for cell therapy and drug discovery. Human organoids in basic research and clinical applications. Elsevier; 2020.

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PPD

OCTOBER 17, 2024

Cell and gene therapy (CGT) studies are rapidly gaining momentum in the Asia-Pacific region, fueled by growing patient demand and a thriving ecosystem of innovation. In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches.

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Conversations in Drug Development Trends

JULY 9, 2024

The discussions at the conference shed light on innovative approaches to enhance long-term outcomes for patients, focusing on combination therapies that not only aid in weight loss but also help preserve muscle mass, an essential component of healthy body composition.

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Drug Target Review

OCTOBER 25, 2024

How does precision neuroscience differ from traditional approaches in the treatment of neurodegenerative diseases, and what advantages does it offer? Traditionally, Alzheimer’s disease is diagnosed by clinical examination, medical history and cognitive testing.

Disease 59

Disease Clinical Trials Therapies Science 59

Vial

DECEMBER 4, 2023

Clinical research is an integral component of the healthcare ecosystem. It is pivotal in the development of cutting-edge treatments, therapies, and interventions for a wide array of diseases. Patient Payments in Clinical Research: Perception Over the Years In the U.S.,

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Vial

MAY 20, 2024

Distinguishing the Roles of Preclinical vs. Clinical CROs in Clinical Research Randomized clinical trials are the gold-standard of evidence-based medicine, representing the culmination of the long, arduous drug development pipeline. billion in 2023 and is expected to grow at a CAGR of 7.0% between 2024 and 2030.

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Advarra

MARCH 10, 2023

Having a one in four chance of developing sickle cell disease (SCD) is uniquely, and overwhelmingly, familiar to the African American population. Considered a rare disease, research in finding better therapies – and possibly even a cure – for SCD may be competing for funding with conditions impacting broader populations.

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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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Advarra

AUGUST 8, 2023

Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. We also discuss how a centralized biosafety review process can benefit this type of research.

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Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. The post Risk Assessment for use of Engineered Genetic Materials in Clinical Research appeared first on Advarra.

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Vial

DECEMBER 8, 2023

With technological innovation and scientific research propelling the development of new therapies, anticipation for the future is high. The goal of this therapy is to replace a disease-causing gene with a healthy one, disable a disease-causing gene, or introduce a new gene to aid in fighting a disease.

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Drug Target Review

APRIL 11, 2024

Targeting pathways involved in SMC phenotypic modulation holds promise for developing novel therapeutic approaches for cardiovascular diseases. Can you explain the methodology used to investigate the genetic variants regulating extracellular matrix (ECM) secretion in SMCs, and predict proteins associated with vascular disease?

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Disease Engineering Regulations Clinical Research 59

Drug Target Review

FEBRUARY 21, 2024

Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?

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The Premier Consulting Blog

APRIL 18, 2024

Development of cell and gene therapies is growing rapidly, given the major advances in genomic technologies and increasing scientific understanding of genetic regulation and immunology. This blog will outline three characteristics of a successful nonclinical program to support entry into clinical trials for a cell or gene therapy product.

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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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The Premier Consulting Blog

JUNE 18, 2023

One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases. However, with rare diseases there may be no available treatments that could serve as SoC or active control in a clinical trial and assigning patients to placebo may be unethical.

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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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Olympian Clinical Research

MAY 6, 2024

As medical understanding grew, so did the sophistication of the therapies. These targeted therapies revolutionized treatment, offering relief to severe cases by specifically addressing the underlying immune responses causing the disease.

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Treatment Small Molecule Clinical Research Immune Response 52

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.

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Conversations in Drug Development Trends

OCTOBER 14, 2024

Autoimmune diseases and bone health conditions are not just medical challenges – they’re personal battles that millions of people fight every day. Fortunately, advances in clinical research are providing hope for better treatments and outcomes.

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Research Clinical Trials Therapies Immune Response 59

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

OCTOBER 14, 2024

Autoimmune diseases and bone health conditions are not just medical challenges – they’re personal battles that millions of people fight every day. Fortunately, advances in clinical research are providing hope for better treatments and outcomes.

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Research Clinical Trials Therapies Immune Response 52

PPD

AUGUST 5, 2024

Pulmonary arterial hypertension (PAH) is a progressive and fatal lung disease that is caused or influenced by multiple factors. The clinical sites working on these trials are highly experienced and have a deep understanding of the complexities of successfully running PAH clinical trials. None offers a cure for PAH.

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Olympian Clinical Research

FEBRUARY 22, 2024

However, advancements in treatments and ongoing clinical research offer new hope. Understanding Hidradenitis Suppurativa HS is more than just a skin condition; it’s a systemic disease that can lead to severe physical discomfort and emotional distress.

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Treatment Clinical Research Clinical Trials Therapies 52

PPD

NOVEMBER 4, 2024

The PPD clinical research business of Thermo Fisher Scientific has conducted a comprehensive survey involving 150 drug developers from around the world. This year’s findings reveal a nuanced landscape where rising clinical trial costs, complex protocol designs and patient recruitment issues have emerged as significant challenges.

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Advarra

MARCH 20, 2023

The Food and Drug Administration (FDA) recently released new guidance regarding cellular and gene therapy products, one of which may significantly impact early-phase clinical trials of such products. This article explores the implications of this guidance, including new approaches for studies focused on cellular or gene therapies.

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FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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PPD

MAY 18, 2023

For neurodegenerative disease research, the care partner, or caregiver, is equally important as the person with dementia. Even with stronger awareness of studies, drug developers must contend with the inherent lack of trust between the dementia community and clinical trials. Recognize and relieve the caregiver burden.

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Clinical Trials Trials Clinical Research Doctors 86

PPD

JUNE 7, 2023

Adults aren’t the only patients affected by the increasing prevalence of liver disease. The number of pediatric patients diagnosed with nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) is on the rise, yet there are currently no approved therapies to treat NAFLD and NASH in adult or pediatric populations.

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Conversations in Drug Development Trends

OCTOBER 24, 2023

In the US, a rare disease is one with 200,000 or fewer patients. Rare diseases exist in every therapeutic area and are often the more complex indications of each therapeutic area. Although no two rare disease patient communities are identical, there are common themes to the evolutionary arc of PAO maturation.

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Clinical Trials Disease Therapies Drug Development 52

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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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. AskBio) I continue to work to bring innovative gene therapies to patients in need.

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Therapies Disease Clinical Trials Science 111

PPD

MARCH 13, 2024

In combination, they provide confidence in dose escalation or protocol adaptation decisions, which ultimately benefit patients who often have critical and life-limiting diseases. Within cell therapy clinical trials, apheresis collections contribute to the specific constituents of the given therapy.

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Olympian Clinical Research

NOVEMBER 9, 2022

Although this disease is estimated to affect less than 50,000 Americans, it may be life-threatening, especially for people who are already in poor health. Radiation therapy. Neurological conditions, such as Parkinson’s disease or epilepsy. Immunosuppressive therapy may also be used to treat bullous pemphigoid.

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Treatment Clinical Research Immune Response Therapies 52

Olympian Clinical Research

OCTOBER 30, 2020

The disease is more common than many may realize, with approximately 2.6 At Olympian Clinical Research we’ve gotten a chance to work with countless Schizophrenia patients over the years through our clinical trials, and we know just how untrue many of the common assumptions about Schizophrenia really are. .

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PPD

OCTOBER 24, 2024

Coordinated efforts are needed to combat this disease and improve the clinical status of persons with HBV infection. Currently there is no cure for hepatitis B, however there are several drugs in clinical development to improve the lives and clinical outcomes for persons with chronic HBV infection, including functional cure strategies.

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