Broad Institute

NOVEMBER 25, 2024

Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases.

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Advarra

NOVEMBER 14, 2024

The DSMB’s primary responsibilities include : Monitoring patient safety: Ensuring participants are not exposed to undue risk and adverse events are promptly identified and addressed. For example, consider a global Phase III, double-blind study, in a novel new therapy.

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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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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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DrugBank

JANUARY 29, 2025

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. Each of these mechanisms represents a distinct strategy to address the underlying genetic perturbations contributing to disease pathogenesis.

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New Drug Approvals

APRIL 11, 2025

1] [2] Adverse effects The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). [2] Fitusiran 1711.0g/mol, 1] It is an antithrombin-directed small interfering ribonucleic acid. [1]

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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.

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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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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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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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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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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.

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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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Drug Target Review

MARCH 17, 2025

However, a deeper understanding of brain function particularly the role of synaptic plasticity is now opening the door to innovative therapies. Event-driven pharmacology Donello highlights the growing recognition of synaptic plasticity’s crucial role in the biology of depression. .

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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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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.

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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).

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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.

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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.

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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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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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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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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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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.

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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.

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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.

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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.

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The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

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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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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.

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The Pharma Data

MARCH 30, 2021

Novartis Oncology continues to reimagine cancer care through development of robust radioligand therapy portfolio. We continue to invest in radioligand therapy as one of the four unique platforms of Novartis Oncology. Broad expression of FAP demonstrated in tumors or in tumor stroma across many solid tumors 1 ,2 ,3.

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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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The Pharma Data

SEPTEMBER 30, 2021

Anderson Professor of Chemical and Biomolecular Engineering, and his colleagues, are reporting in iScience the event of an intranasal subunit vaccine that gives durable local immunity against inhaled pathogens. But now, we’re one step closer. Navin Varadarajan, University of Houston M.D.

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The Pharma Data

JANUARY 27, 2021

. – Preclinical Data Underscore Treatment Potential for PBFT02 in Frontotemporal Dementia with Granulin (GRN) Mutations, a Devastating, Progressive Disorder Impacting Adults with No Approved Disease-Modifying Therapy Options. PHILADELPHIA, Jan. 28, 2021 (GLOBE NEWSWIRE) — Passage Bio , Inc. chief executive officer of Passage Bio.

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Drug Target Review

MAY 6, 2024

The clinical efficacy of BRAF inhibitors, like most other targeted cancer therapies, is inevitably limited by resistance development. Melanoma was an ideal disease for studying this phenomenon because cutaneous tumour biopsies are readily available. This caused many scientists to study the mechanisms that drive drug resistance.

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The Pharma Data

JANUARY 3, 2021

(Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that U.S. GM1 is a rare and often life-threatening CNS disorder with no approved disease-modifying therapies available. chief medical officer of Passage Bio.

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PPD

SEPTEMBER 16, 2024

Real-world data paired with machine learning is a game changer in drug development RWD is becoming increasingly important in influencing the drug development landscape, particularly when used to develop the natural history and patient journey through disease. AI is also proving useful in drug repurposing or indication selection.

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