ASPET

JUNE 27, 2024

Estrogen receptor (ER)-negative breast cancers are known to be aggressive and unresponsive to anti-estrogen therapy, and triple negative breast cancers are associated with poor prognosis and metastasis. Thus, new targeted therapies are needed.

Therapies 100

Therapies Protein Expression 100

Drug Discovery Today

FEBRUARY 4, 2022

• Face-to-face event, taking place in London, UK, 26-27th April, will feature two dedicated tracks: ‘Scientific’, focused on seven core therapy areas and ‘Life Sciences Business’ Programme features keynote from George Freeman MP, with an update on the UK Government Life Sciences VisionRegistration for BioTrinity 2022 now available online

Science 113

Science Government Therapies 113

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

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. Our recent work proposes the use of an intracellular toolkit to map organelle bio-geography in stem cells that could lead to more precise therapies.”

RNA 128

RNA Immune Response Therapies Disease 128

ASPET

AUGUST 2, 2024

Based on previous studies, we hypothesized that vascular senescence increases the risk of bleeding during warfarin therapy. The vascular senescence group tended to experience more bleeding or thromboembolic/ischemic events during one year of follow-up, while there was no statistical difference.

Therapies 100

Therapies Hospitals International 100

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

AUGUST 6, 2024

Cardiovascular diseases : Atherosclerosis : ADCs could be engineered to target and deliver drugs to atherosclerotic plaques, potentially stabilising them and preventing cardiovascular events. Liver Fibrosis : Targeted therapies using ADCs could help reduce liver fibrosis and prevent the progression to cirrhosis.

Therapies 117

Therapies Pharmaceutical Companies Disease Treatment 117

Chemical Biology and Drug Design

NOVEMBER 4, 2024

Some researchers hypothesized that the primary event associated with the cholinergic neurotransmitter (acetylcholine) is memory loss and cognitive impairment. Due to the disease's complicated pathogenesis, long-term therapy with a single target candidate is futile. As a result, multitargeted and multifunctional therapies have emerged.

Disease 100

Disease Therapies Research 100

Alta Sciences

JUNE 11, 2024

My Attendance at the 2024 Boston Society Cell and Gene Therapy Conference pmjackson Tue, 06/11/2024 - 20:49 , via Wikimedia Commons" data-entity-type="file" data-entity-uuid="c7a7fa8b-b2fe-4d84-a75e-d1ba3a4e2caf" src="[link] width="742" height="249" loading="lazy" /> Ecm85, CC BY-SA 3.0

Therapies 52

Therapies Molecular Biology Immune Response Science 52

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.

Therapies 52

Therapies FDA Trials Clinical Trials 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. what is the altascientist?

Therapies 52

Therapies Immune Response Disease Virus 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

Science Daily: Pharmacology News

JULY 31, 2023

Simply remembering events can trigger brain rhythms, even more so than when people are experiencing the actual event, says a new study. The findings could lay foundations for cognitive impairment therapy and help improve memory.

Therapies 76

Therapies 76

Chemical Biology and Drug Design

FEBRUARY 13, 2024

Some researchers proposed that the symptoms involved in AD (loss of memory, cognitive impairment, and amnesia) are the main event linked to the cholinergic neurotransmitter (acetylcholine). Of the multiple hypotheses for AD, the cholinergic and amyloid-β (Aβ) hypothesis are the main targeting hypothesis for AD.

Disease 100

Disease Treatment Therapies Research 100

ASPET

MARCH 20, 2024

5 in part due to overexpression of microRNA-9-3p/5p impacting post-transcriptional events. Our laboratory previously demonstrated reduced levels of TOP2β/180 (and the paralog TOP2α/170) in an acquired etoposide-resistant K562 clonal cell line, K/VP.5

DNA 100

DNA Laboratories Therapies Regulations 100

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!

Drugs 40

Drugs Science Research Therapies 40

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. This study was published in Nature Communications.

Therapies 110

Therapies Doctors Disease Research 110

Chemical Biology and Drug Design

MAY 11, 2023

Neuronal degeneration may arise due to a variety of pathological events, like inflammatory responses, neurotransmitter dysregulation, oxidative damage, mitochondrial malfunction, apoptosis, and genetic factors. Crocin, a naturally occurring carotenoid molecule, was found to have neuroprotective potential in the therapy of this disorder.

Disease 100

Disease Research Clinical Trials Treatment 100

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. Similarly, the neurosteroid analog VX significantly attenuated SRS, discharges, HFOs, and ictal events.

Disease 100

Disease Therapies Treatment 100

Drug Channels

NOVEMBER 4, 2022

He suggests that lessons learned from the COVID-19 pandemic can inform both innovation in AMR therapies and our understanding of AMR. This virtual event is presented by OptimizeRx in collaboration with Melina Therapeutics. Read on for Matt’s insights.

Therapies 86

Therapies Marketing Drugs 86

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

Alta Sciences

OCTOBER 29, 2024

PBSS - San Francisco Bay—Current State of Bioanalysis for Biologics and Cell/Gene Therapy Products iianiro Tue, 10/29/2024 - 20:55 Image foster_city_sunset.jpg Synopsis Foster City, CA URL [link] Event End Date Tue, 11/05/2024 - 12:00 Event Start Date Fri, 11/15/2024 - 12:00 Weight 1

Production 40

Production Therapies 40

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

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 describes various methods to assess either cell distribution for cell therapy products or vector biodistribution for gene therapy products.

Therapies 59

Therapies FDA Production Disease 59

Alta Sciences

DECEMBER 29, 2023

Webinar—Nonclinical AAV Gene Therapy Development–Key Considerations on Study Design, Laboratory Endpoints, and Regulatory Guidance lperez Fri, 12/29/2023 - 18:03 Image Website_Webinar_BioA Gene Therapy.jpg Tags Preclinical Research URL [link] Event End Date Thu, 02/29/2024 - 12:00 Event Start Date Thu, 02/29/2024 - 12:00 Weight 1

Laboratories 40

Laboratories Therapies Research 40

Alta Sciences

JULY 18, 2023

Webinar—Nonclinical Safety Assessment for Gene Therapy Products—Key Considerations lperez Tue, 07/18/2023 - 18:35 Image Website_Webinar_AAV-SoMe_v2.jpg jpg Tags Preclinical Research URL [link] Event End Date Thu, 10/19/2023 - 12:00 Event Start Date Thu, 10/19/2023 - 12:00 Weight 1

Production 40

Production Therapies Research 40

Alta Sciences

OCTOBER 29, 2024

PBSS - Boston—Preclinical Development and IND Filing for new modalities, ADCs, SiRNA/ASO, Gene and Cell Therapies iianiro Tue, 10/29/2024 - 18:14 Image cambridge-massachusetts.png Synopsis Boston, MA URL [link] Event End Date Wed, 11/06/2024 - 12:00 Event Start Date Wed, 11/06/2024 - 12:00 Weight 1

Therapies 40

Therapies 40

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

Drug Channels

APRIL 4, 2022

Hybrid Event May 17-19, 2022 | Boston, MA www.informaconnect.com/rare. Come together with experts from across the rare disease community, in-person or virtually, to unite in areas of unmet medical need and create life-transforming therapies and breakthroughs. Rare Disease Innovation & Partnering Summit.

Disease 77

Disease Therapies FDA Trials 77

Precision for Medicine

OCTOBER 17, 2023

Precision for Medicine is proud to be attending the 3rd Gene and Cell Therapies: CMC, CGT & Vector Manufacturing Summit in Vienna, Austria. The post 3rd Gene and Cell Therapies: CMC, CGT & Vector Manufacturing Summit(June 20-21) appeared first on Precision For Medicine. Schedule a meeting here.

Therapies 52

Therapies 52

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. A permanent gene edit to produce a difficult-to-titrate supply of GLP1 in the liver?

Small Molecule 137

Small Molecule Trials Therapies Disease 137

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.

Small Molecule 145

Small Molecule Drugs Clinical Pharmacology Trials 145

PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. But even if gene therapy could reach sufficient muscle cells to improve quality of life, another hurdle looms: the dystrophin gene that’s deleted in DMD is huge, the largest in a human genome at 2.2

FDA Approval 98

FDA Approval Treatment FDA DNA 98

KIF1A

JULY 15, 2023

When the brain’s electrical activity changes enough to cause noticeable changes in behavior, we call that event a seizure. By identifying short and subtle changes in movements, we may be able to characterize epileptic events with more precision. From Gschwind et al. 2023, Neuron.

Disease 98

Disease Treatment Research Therapies 98

Eye on FDA

AUGUST 4, 2021

Below is a chart that looks at 3 numbers – the number of AdComms; the number of new molecular entities approved each year, and the number of drugs that had breakthrough therapy designation.

FDA 122

FDA Clinical Pharmacology FDA Approval Therapies 122

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

Drug Target Review

MAY 6, 2024

The clinical efficacy of BRAF inhibitors, like most other targeted cancer therapies, is inevitably limited by resistance development. We found that, indeed, cancer’s earliest defence mechanisms are triggered almost immediately after exposure to a targeted cancer therapy. What if our science can directly impact patients’ lives?”

Therapies 105

Therapies Science Small Molecule Clinical Trials 105