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.

Therapies 97

Therapies Clinical Trials Clinical Research Trials 97

Drug Target Review

DECEMBER 20, 2024

These innovations have started to shift industry perceptions, positioning AI as a transformative tool that could alter how drugs are developed, tested, and brought to market. By increasing the chances that more participants will receive the treatment being tested, recruitment becomes more efficient, speeding up the overall trial process.

Clinical Trials 128

Clinical Trials Trials Pharmaceuticals Drug Development 128

Drug Target Review

JANUARY 4, 2024

What distinguishes Alder Therapeutics’ approach to regenerative cell therapy development from traditional methods, and how does it aim to reduce risks in the preclinical phase? Traditional approaches to regenerative cell therapy development are defined by several challenges. For manufacturing, it’s no different.

Therapies 98

Therapies Small Molecule Production Disease 98

PPD

MARCH 11, 2025

Developing treatments for individuals living with rare diseases is critical, but orphan drug development is laden with unique obstacles that necessitate innovative, multifaceted approaches. This limited pool poses significant barriers to conducting statistically significant studies and validating the efficacy and safety of new treatments.

Drug Development 52

Drug Development Clinical Trials Disease Trials 52

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?

Therapies 64

Therapies Treatment Vaccine FDA Approval 64

PLOS: DNA Science

MARCH 21, 2024

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). million price tag for the one-time infusion, and for the older siblings who contributed to developing the gene treatment, but were too sick to receive it.

Therapies 98

Therapies Clinical Trials DNA Trials 98

ASPET

OCTOBER 20, 2023

However, Pramlintide an adjunct to insulin therapy, enhances glucagon suppression and thereby offers improved glycaemic control. Although clinical benefits of Pramlintide are well reported, there still exists a high patient resistance for the therapy as separate injections for Pramlintide and insulin are required to be administered.

Therapies 100

Therapies Pharmacokinetics Treatment Marketing 100

Conversations in Drug Development Trends

MAY 30, 2024

Since marketing authorization for the first breakthrough treatment in 1994, the steady increase in clinical trials reflects the community’s commitment to finding effective ALS treatments despite the numerous hurdles associated with clinical trial design, from proof-of-concept to pivotal trials. The MAA file in the E.U.

Treatment 81

Treatment Clinical Trials Small Molecule Therapies 81

Drug Target Review

SEPTEMBER 7, 2023

The first clinical candidate, BSB-1001, aims to transform leukemia treatment by targeting minor histocompatibility antigen-1. This two-part therapy aligns with allogeneic hematopoietic stem cell transplantation to enhance patient outcomes. TCX-102 complements this effort by focusing on TCRs against NPM1 gene mutations.

Treatment 98

Treatment Licensing Licensing Disease 98

Conversations in Drug Development Trends

OCTOBER 14, 2024

Fortunately, advances in clinical research are providing hope for better treatments and outcomes. Given the complexity and widespread impact of autoimmune and bone health conditions, developing new therapies is essential. One of the biggest hurdles in developing new therapies is recruiting diverse patients for clinical trials.

Research 52

Research Clinical Trials Therapies Immune Response 52

Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

Therapies 59

Therapies Treatment Engineering Molecular Biology 59

Drug Patent Watch

DECEMBER 9, 2024

From Lab to Market: The Long Road of Drug Development Once a promising compound is identified, it enters the long and costly process of drug development. Taxol: From Pacific Yew to Cancer Treatment Taxol (paclitaxel), a widely used cancer drug, was originally isolated from the bark of the Pacific yew tree.

Drugs 97

Drugs Drug Development Pharmaceuticals Production 97

Drug Target Review

AUGUST 6, 2024

Autoimmune diseases : Rheumatoid Arthritis : ADCs targeting specific immune cells or inflammatory mediators can provide more precise treatment options with potentially fewer side effects. Obesity : By targeting adipose tissue or specific metabolic pathways, ADCs could offer new treatments for obesity and related metabolic disorders.

Therapies 119

Therapies Pharmaceutical Companies Disease Treatment 119

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options. 3D rendering of Antibody Drug Conjugate Molecules.

Small Molecule 145

Small Molecule Drugs Clinical Pharmacology Trials 145

Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. Could you give us an overview of the Lineage platform?

Therapies 84

Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

ASPET

SEPTEMBER 7, 2023

Alglucosidase alfa, a recombinant human acid α-glucosidase, was the first approved treatment for Pompe disease, but its uptake into skeletal muscle via the cation-independent mannose-6-phosphate (M6P) receptor (CIMPR) is limited. These results further substantiate the crucial role of CIMPR binding in lysosomal targeting of ERTs.

Small Molecule 100

Small Molecule Therapies Disease Treatment 100

Fierce BioTech

MARCH 6, 2025

AI-powered digital phenotyping and real-world data (RWD) offer new ways to locate overlooked patient populations, identify gaps between clinical guidelines and real-world treatment patterns, and optimize market positioning and messaging.

Treatment 75

Treatment Therapies Marketing 75

ASPET

OCTOBER 12, 2023

The focus of the CET RAIDR effort is to bridge treatment gaps between threat identification and the implementation of licensed targeted MCMs, thereby strengthening warfighter resiliency. The CET RAIDR program conserves both time-to-market and funds by leveraging previous conventional development work as a launch point for repurposing efforts.

Therapies 100

Therapies Licensing Licensing FDA Approval 100

DrugBank

AUGUST 9, 2024

  The average cost of bringing a new drug to market is about $2.6 When a drug loses patent protection, generic or biosimilar versions can enter the market, leading to a rapid decline in sales for the original brand-name drug. Pharmaceutical Market Analysis The global pharmaceutical market revenue in 2024 is projected to be 1.15

Pharmaceuticals 98

Pharmaceuticals Research Pharmaceutical Companies Clinical Trials 98

Quanticate

NOVEMBER 21, 2024

An efficacy endpoint in oncology is a characteristic or variable that measures how beneficial a treatment is to a patient’s feeling, function, and survival in a clinical trial. They are essential for assessing whether new cancer therapies are safe and efficacious [1].

Clinical Trials 98

Clinical Trials Trials Therapies Treatment 98

PPD

DECEMBER 16, 2024

Addressing these underlying factors is essential; only by tackling the complexities of recruitment, timelines and regulatory compliance can sponsors achieve cost-effective and efficient trials, paving the way for long-term success in todays demanding market. Nearly 39% of sponsors cite these costs as primarily driven by complex protocols.

Drug Development 52

Drug Development Clinical Trials Compliance Trials 52

Drug Target Review

SEPTEMBER 6, 2024

What trends are driving the increased use of in vivo delivery methods in gene therapy? How is the market demand for lentiviral vectors evolving, and what factors are influencing this growth? There is a very significant increased market demand for lentiviral vectors. New therapeutic areas are emerging in a powerful way.

Therapies 59

Therapies Vaccine Marketing Disease 59

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.

Drug Development 98

Drug Development Metabolic Stability Clinical Trials Drugs 98

Antidote

JANUARY 3, 2023

Before becoming available to patients, every medication, therapy, and intervention must undergo a rigorous approval process: the clinical trial. Clinical trials are divided into phases that are aimed at testing the safety and efficacy of every potential new treatment before it is able to enter the market.

Clinical Trials 97

Clinical Trials Trials Therapies Treatment 97

DS in Pharmatics

JUNE 10, 2023

On April 12, 2023, BeNeLuxA1 announced that negotiations failed to secure reimbursement for Libmeldy, a gene therapy developed by Orchard Therapeutics for the treatment of metachromatic leukodystrophy, on grounds of price. In the UK, an initial application to NICE was rejected as well back in 2020. This is a huge […]

Marketing 66

Marketing Therapies Treatment 66

Conversations in Drug Development Trends

NOVEMBER 5, 2024

NC: I think the continued growth in cell and gene therapy research and the approvals we’re seeing for genetically-driven rare diseases. What emerging technologies or therapies are you most excited about for 2025, and how do you think they will impact rare disease treatments? DA: Several things!

Disease 52

Disease Clinical Trials Therapies Clinical Research 52

The Pharma Data

MAY 27, 2022

In the pivotal clinical study CARTITUDE-1, 98 percent of patients with relapsed or refractory multiple myeloma responded to a one-time treatment with ciltacabtagene autoleucel and 80 percent of patients who responded experienced a stringent complete response . In December 2017, Janssen Biotech, Inc. to develop and commercialise cilta-cel.[1].

Therapies 52

Therapies Treatment Pharmaceutical Companies Nurses 52

PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

Therapies 69

Therapies Immune Response DNA Virus 69

The Pharma Data

APRIL 27, 2022

NYSE: BHVN) today announced that the European Commission (EC) has granted marketing authorization for VYDURA® (rimegepant), a calcitonin gene-related peptide (CGRP) receptor antagonist for both the acute treatment of migraine with or without aura, and prophylaxis of episodic migraine in adults who have at least four migraine attacks per month.

Treatment 52

Treatment Marketing FDA Approval Production 52

The Pharma Data

JUNE 22, 2023

Kite Announces Completion of Marketing Authorization Transfer for Yescarta® CAR T-cell Therapy in Japan SANTA MONICA, Calif.–(BUSINESS in December 2022 about changes to their initial 2017 partnership whereby Daiichi Sankyo obtained the Marketing Authorization for Yescarta ®. to Gilead Sciences K.K.,

Therapies 40

Therapies Marketing Small Molecule Science 40

Drug Target Review

NOVEMBER 16, 2023

There is a substantial need for new therapies to treat drug-resistant infections without contributing to or perpetuating resistance; therefore, we are committed to developing highly effective treatment solutions that can be used against a broad range of bacterial infections. We are dedicated to expanding our pipeline beyond R327.

Pharmaceuticals 105

Pharmaceuticals Therapies Clinical Trials Trials 105

The Premier Consulting Blog

NOVEMBER 6, 2024

Inhaled combination products (ICP) have emerged as a significant advancement in the treatment of respiratory diseases such as asthma, chronic obstructive pulmonary disease (COPD), and other pulmonary conditions. What regulatory division governs ICPs? ICPs are subject to rigorous regulatory scrutiny due to their complexity.

Production 52

Production FDA Compliance Disease 52

Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

Drug Development 52

Drug Development Treatment FDA Approval Drugs 52

DrugBank

MARCH 7, 2024

The landscape of weight loss drugs has been rapidly evolving, and 2024 is poised to be another transformative year in this market. Pharmaceutical researchers, in particular, have a keen interest in understanding the unfolding dynamics of this market. Let’s delve into what lies ahead in the coming year.

Pharmaceutical Companies 98

Pharmaceutical Companies Clinical Trials Pharmaceuticals Therapies 98

DrugBank

MARCH 15, 2024

The weight loss industry has emerged as a major hub for innovative research, groundbreaking treatments, and economic opportunity. According to Grand View Research , the global weight loss and weight management market size was valued at $142.5 With sales soaring to $2.2 for this class of drugs.   billion and $41.6

Pharmaceutical Companies 97

Pharmaceutical Companies Pharmaceuticals Treatment Clinical Trials 97

The Pharma Data

NOVEMBER 17, 2021

Biogen moment blazoned that the European Commission (EC) has granted marketing authorization for VUMERITY ® (diroximel fumarate) to treat grown-ups with relapsing- remitting multiple sclerosis (MS). In EVOLVE-MS-2, the rate of overall treatment termination was lower in actors treated with VUMERITY compared to those treated with TECFIDERA (1.6

Treatment 52

Treatment Marketing Clinical Trials Biosimilars 52

Fierce BioTech

JANUARY 16, 2025

Yet the process of getting to market and enabling patient access is far from simple. Yet the process of getting to market and enabling patient access is far from simple. Register now to secure your spot.

Therapies 52

Therapies Treatment Disease Marketing 52