Drug Channels

APRIL 4, 2022

Rare Disease Innovation & Partnering Summit. Hybrid Event May 17-19, 2022 | Boston, MA www.informaconnect.com/rare. Don’t miss the Rare Disease Innovation & Partnering Summit, coming up May 17-19, 2022 in Boston, MA. Visit www.informaconnect.com/rare for further details and to register. d/b/a Drug Channels Institute.

Disease 77

Disease Therapies FDA Trials 77

Drug Target Review

JANUARY 2, 2024

Through a series of experiments, they successfully developed a personalised stem cell therapy using a data-driven, single-cell technique based on swift subcellular proteomic imaging. We are studying the placement of organelles within cells and how they communicate to help better treat disease,” said Coskun.

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RNA Immune Response Therapies Disease 128

Drug Target Review

AUGUST 6, 2024

A number of areas represent significant opportunities for Antibody Drug Conjugates (ADCs) beyond oncology, leveraging their ability to deliver therapeutic agents specifically to diseased cells or tissues while minimising off-target effects.

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Therapies Pharmaceutical Companies Disease Treatment 117

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?

Disease 59

Disease Engineering Regulations Clinical Research 59

Vial

DECEMBER 8, 2023

With technological innovation and scientific research propelling the development of new therapies, anticipation for the future is high. As we look forward to 2024, several groundbreaking therapies are poised to radically change the landscape of ophthalmology, providing hope and improved treatment options for patients with ocular conditions.

Therapies 52

Therapies Clinical Trials Trials Treatment 52

Drug Target Review

SEPTEMBER 9, 2024

functions to reset the immune system and potentially provide remission for allergic diseases? In allergic diseases, APCs identify and present unknown antigens to the immune system. This can lead to inadequate long-term effectiveness with low rates of disease remission. They also require frequent administration.

Disease 64

Disease Immune Response Clinical Trials Treatment 64

The Pharma Data

JUNE 30, 2021

With a median follow-up of two years, the study met the primary endpoint of event-free survival (EFS; hazard ratio 0.398, p <0.0001). Event-free survival is defined as time from randomization to disease progression, commencement of new lymphoma therapy, or death from any cause. This press release features multimedia.

Therapies 52

Therapies Treatment Virus Vaccine 52

Drug Target Review

DECEMBER 5, 2024

Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.

Therapies 52

Therapies Virus DNA Compliance 52

Drug Target Review

OCTOBER 9, 2023

The potential of stem cell therapies is rooted in the pluripotency of these early embryonic stem cells. Dr Laura Kerosuo explains that their discoveries cast fresh illumination on the sequence of events in early embryonic development. It can be applied to various inquiries, including the comparison of diseased and healthy tissue.

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Therapies Doctors Disease Research 110

ASPET

NOVEMBER 30, 2023

In this study, we evaluated the long-term efficacy of novel synthetic neurosteroids in preventing the development of chronic epilepsy and hyperexcitable ictal events in a rat OP model of SE. It significantly reduced the occurrence of epileptic biomarkers of HFOs and interictal spikes, indicating potential disease-modifying activity.

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Disease Therapies Treatment 100

LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Join the club.

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Small Molecule Trials Therapies Disease 137

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

Alta Sciences

JANUARY 11, 2024

It is estimated that there are over 6,000 monogenic diseases, affecting over 350 million people worldwide; for these diseases, cell and gene therapy may provide hope for a cure. However, there are significant challenges associated with the successful development of these complex, leading-edge therapies.

Therapies 52

Therapies Immune Response Disease Virus 52

KIF1A

JULY 15, 2023

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. When the brain’s electrical activity changes enough to cause noticeable changes in behavior, we call that event a seizure. From Gschwind et al. 2023, Neuron.

Disease 98

Disease Treatment Research Therapies 98

PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. Muscle makes up about 40 percent of body weight. million DNA bases.

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FDA Approval Treatment FDA DNA 98

The Pharma Data

OCTOBER 14, 2021

The results, which demonstrate that patients treated with Biogen’s portfolio of MS therapies mount an effective antibody response to COVID-19 vaccination, are being presented at the 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) virtual meeting, October 13-15, 2021.

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Vaccine Therapies Disease Immune Response 52

The Pharma Data

AUGUST 30, 2021

Positive high-level results from the FoCus Phase III trial in Wilson disease showed ALXN1840 met the primary endpoint with a statistically significant improvement in daily mean copper mobilisation from tissues, demonstrating superiority compared with standard-of-care (SoC) treatments. Wilson disease. 1, 2, 3.

Disease 52

Disease Trials Therapies Treatment 52

Conversations in Drug Development Trends

APRIL 5, 2024

For others, this rare day is aptly the day we honor rare disease communities. Observed annually on the last day of February since 2008, Rare Disease Day has grown into a global movement for raising awareness, promoting research, and advocating for improved access to rare disease treatments and support services.

Disease 52

Disease Clinical Trials Trials Treatment 52

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. Patients and caregivers also assess the benefits offered by different therapies, weighing the progression-free survival with their off-target effects.

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Small Molecule Drugs Clinical Pharmacology Trials 145

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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Therapies FDA Trials Clinical Trials 52

The Pharma Data

JUNE 1, 2022

The United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for the treatment of people with hemophilia A, a rare and life-threatening bleeding disorder, based on data from the pivotal XTEND-1 P hase 3 stud y.

Therapies 52

Therapies FDA Science Treatment 52

The Pharma Data

SEPTEMBER 11, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for empagliflozin as an investigational treatment for adults with heart failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced. 2,3 A leading cause of hospitalization in the U.S. o Completion: 2020.

Therapies 52

Therapies Hospitals Clinical Trials Trials 52

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.

Drug Development 52

Drug Development Disease Clinical Trials Drugs 52

FDA Law Blog: Drug Discovery

DECEMBER 6, 2024

Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. The draft guidance also directs sponsors to consider the biological relevance of a particular animal or disease model for pharmacology studies.

Therapies 59

Therapies FDA Production Disease 59

The Premier Consulting Blog

OCTOBER 11, 2022

Clinical trials for ultra-rare diseases can be particularly challenging to mount due to small, geographically-dispersed patient populations. In this article, we review two case studies involving the successful use of RWD or RWE in advancing the clinical development of treatments for rare diseases. Reference: [1] Hansen, et al.

Disease 52

Disease FDA FDA Approval Treatment 52

Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

Small Molecule 105

Small Molecule Therapies Regulations Treatment 105

Drug Target Review

JANUARY 15, 2024

Gaining access inside cells Fortunately, evolution has provided a mechanism, found in all higher vertebrates, for the immune system to monitor intracellular events. Engineering soluble T-cell receptors for therapy. Cancer Therapy With TCR‑Engineered T Cells. The FEBS Journal 288:6159–6173 (2021). Schafer P, Kelly L, Hoyos V.

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Engineering DNA Virus Immune Response 110

Conversations in Drug Development Trends

FEBRUARY 28, 2023

95% of rare diseases don’t have a treatment approved and won’t anytime soon. Uplifting Athletes As a 501(c)(3) nonprofit, UA harnesses the power of sport and partnerships with patient advocacy groups to fund the researchers developing datasets for rare diseases that have never been studied or understood before.

Clinical Trials 52

Clinical Trials Disease Trials Research 52

PPD

AUGUST 5, 2024

Pulmonary arterial hypertension (PAH) is a progressive and fatal lung disease that is caused or influenced by multiple factors. As your CRO partner, we deliver equally dedicated and experienced teams that understand this disease and the range of assessments used for collecting endpoint data in PAH clinical trials.

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Clinical Trials Trials Clinical Research Disease 98

The Pharma Data

MAY 27, 2022

Novartis today announced the US Food and Drug Administration (FDA) has granted accelerated approval for Kymriah ® (tisagenlecleucel) for the treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy.

FDA Approval 52

FDA Approval Therapies FDA Treatment 52

The Pharma Data

SEPTEMBER 27, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for empagliflozin as an investigational treatment for adults with coronary failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced. 2,3 a number one explanation for hospitalization within the U.S.

Therapies 52

Therapies Clinical Trials Hospitals Trials 52

The Pharma Data

JULY 12, 2021

Finerenone 10 mg or 20 mg is indicated to reduce the risk of sustained estimated glomerular filtration rate (eGFR) decline, end-stage kidney disease, cardiovascular death, non-fatal myocardial infarction, and hospitalization for heart failure in adult patients with chronic kidney disease (CKD) associated with type 2 diabetes (T2D).

FDA Approval 52

FDA Approval Disease FDA Treatment 52

Drug Channels

JULY 11, 2022

Delivered as a Hybrid Event. This important event convenes key stakeholders including manufacturers, specialty pharmacies, hub providers and more for unrivaled collaboration to leverage innovation and optimize access. To find out how you can promote an event on Drug Channels, please contact Paula Fein (paula@drugchannels.net).

Specialty Pharmacies 83

Specialty Pharmacies Pharmacy Biosimilars Compliance 83

DrugBank

AUGUST 9, 2024

Humira , AbbVie's monoclonal antibody for autoimmune diseases, generated over $20 billion in annual revenue at its peak before losing patent exclusivity in 2023.  This exponential growth is primarily fueled by the escalating global burden of chronic diseases. trillion dollars, and it is forecasted to exceed 1.4

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Pharmaceuticals Research Pharmaceutical Companies Clinical Trials 77

LifeSciVC

APRIL 24, 2024

Target validation : Do we believe the target(s) plays a central role in disease biology and that modulation will modify disease? There is nothing more exciting than digging into a new target and trying to develop a thesis on whether modulation may be impactful in disease. Is the functionality of mutations known?

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Production Small Molecule Regulations Trials 111

Elrig

MAY 18, 2023

The most important drug discovery event of the year? 18-19 October 2023 The most important drug discovery event of the year? It’s with great excitement that we announce the return of ELRIG’s FREE TO ATTEND Drug Discovery 2023 (DD 2023); Europe’s leading event for the drug discovery community! Save the date! Save the date!

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Drugs Science Research Therapies 40

The Pharma Data

JUNE 26, 2021

Other data highlighted in the publication and ASCO presentation: Median time to first symptomatic skeletal event or death was 11.5 Other data highlighted in the publication and ASCO presentation: Median time to first symptomatic skeletal event or death was 11.5 months in the 177 Lu-PSMA-617 plus SOC arm versus 6.8

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Therapies Treatment International FDA 52