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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Broad Institute

FEBRUARY 7, 2024

However, researchers lack efficient ways to explore how these genetic variants are molecularly connected to cardiovascular disease, limiting efforts to develop therapeutics. The team found that a key biological mechanism involved in a rare vascular disease may influence CAD risk.

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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. In 2023, two gene-based treatments became available.

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FDA Law Blog: Biosimilars

APRIL 13, 2025

LYTGOBI was granted accelerated approval for the treatment of adult patients with previously treated, unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma (iCCA) with FGFR2 gene fusions or rearrangements. DCR is defined as the sum of complete response (CR), partial response (PR) and stable disease (SD).

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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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Elrig

MARCH 25, 2025

Prof Rory Johnson, Associate Professor, University College Dublin, and Dr Shalini Andersson, Vice President Nucleic Acid Therapeutics, AstraZeneca will lead this years event focussed on drugging the undruggable.

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Broad Institute

DECEMBER 18, 2023

But a new path for bringing treatments to patients is starting to emerge. To achieve this, the authors called for more genetics and longitudinal data types from patients of diverse ancestries, more data- and tool-sharing across sectors, the development of quantitative biomarkers for measuring disease mechanisms, and more.

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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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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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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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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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DrugBank

OCTOBER 17, 2024

Bayesian adaptive randomization , for example, can dynamically allocate patients to different treatment arms based on accumulating data, ensuring that more patients receive the most promising treatment. Machine learning algorithms can be trained on historical data to identify patterns and anomalies indicating potential safety concerns.

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Drug Target Review

JANUARY 2, 2024

We are studying the placement of organelles within cells and how they communicate to help better treat disease,” said Coskun. Scrutinising these distinctions aids scientists in comprehending cellular operations, thereby fostering enhanced therapeutic approaches for diverse diseases.

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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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Conversations in Drug Development Trends

FEBRUARY 28, 2023

95% of rare diseases don’t have a treatment approved and won’t anytime soon. Miraculously, there was an approved treatment. This treatment only exists because a small charity in the ’70s donated the funds needed for the researcher to discover it. Uplifting Athletes (UA) is on a mission to change that. It saved my life.”

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The Pharma Data

AUGUST 30, 2021

ALXN1840 demonstrated approximately three times greater copper mobilisation from tissues than standard-of-care treatments. Wilson disease is a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised.

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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.

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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. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options.

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KIF1A

FEBRUARY 24, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. Read the Article Rare Roundup Rare Disease Day is this Thursday, February 29th!

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DrugBank

SEPTEMBER 19, 2024

It offers convenience for patients, promotes medication adherence, and improves treatment outcomes. Achieving a sustained and targeted drug release profile is essential for maximizing therapeutic effects while minimizing the risk of adverse events. This could aid in treatment for diabetic patients who currently rely on injections.

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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

AUGUST 29, 2024

By: Nathan Chadwick, Therapeutic Strategy Lead, Rare Disease Over the last few years in clinical trials, particularly within the rare disease community, a notable shift is underway, where patients and caregivers are taking the lead in reaching out to clinical trial sites rather than the other way around.

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The Pharma Data

FEBRUARY 25, 2021

Food and Drug Administration for investigational AGN-190584 (pilocarpine 1.25%) ophthalmic solution for the treatment of presbyopia. There were no treatment emergent serious adverse events observed in any AGN-190584 treated participants. The most common treatment emergent non-serious adverse events occurring at a frequency of ?5%

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Conversations in Drug Development Trends

AUGUST 10, 2023

As one of the more recent fields to emerge in the clinical space, psychedelics have gained significant traction as a potential treatment for various neuropsychiatric diseases. I was fortunate to have the opportunity to attend Psychedelic Science 2023 during the third week of June in Denver, Colorado.

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Drug Target Review

APRIL 7, 2025

The challenge of GPCR drug discovery G protein-coupled receptors (GPCRs) are one of the most desirable and challenging target classes in drug discovery, as their mutation can lead to a wide range of diseases such as cancer, cardiovascular disorders and neurological conditions.

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The Pharma Data

MARCH 26, 2021

Despite continuous innovations in the treatment landscape, unmet needs remain. Overall, the number of treatment-emergent adverse events reported was similar between the ponesimod and teriflunomide treated groups, and the majority were mild/moderate and did not warrant treatment discontinuation. vs. 10.4%).

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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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The Pharma Data

AUGUST 22, 2021

The findings, published online in The American Journal of Geriatric Psychiatry , evaluated the long-term use of AUSTEDO with the total motor Abnormal Involuntary Movements Scale (AIMS) score, and measures of treatment success and quality of life. IMPORTANT SAFETY INFORMATION.

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The Pharma Data

MARCH 2, 2021

Boehringer Ingelheim and Gubra today announced a new research and licensing agreement focused on the identification and validation of targets and innovative peptide compounds for the treatment of obesity. Senior Vice President and Global Head of Cardiometabolic Diseases Research, Boehringer Ingelheim. population will have obesity.

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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. However, it is often the case that the smaller marginal benefit of new treatments leads to a reduction in relevant outcomes that can be used as endpoints. Historically, the available drugs and U.S.

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Conversations in Drug Development Trends

FEBRUARY 22, 2023

Rare Disease Day on February 28th is a chance for rare disease communities to come together as one and join in conversation with stakeholders searching for treatments, raising money to support research, and changing the way healthcare and governments interact with patients. In my case, my daughter Iris has GM1 gangliosidosis.

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Conversations in Drug Development Trends

DECEMBER 19, 2024

At the recent World Orphan Drug Congresses (WODC) in Europe and the United States, Worldwide Clinical Trials Derek Ansel , Vice President, Therapeutic Strategy Lead, Rare Disease, facilitated roundtable discussions to explore the operational challenges and ethical barriers surrounding genetic testing.

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PPD

JULY 29, 2024

The benefits of utilizing AI in clinical research Patient recruitment and diversity AI is increasingly being utilized in the recruitment process for clinical trials , aiming to connect individuals with potential benefits from investigational treatments.

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The Pharma Data

JANUARY 14, 2021

Department of Health and Human Services’ (HHS) decision to effectively remove the “X-waiver” requirement for physicians, expanding their ability to utilize medication-assisted treatment (MAT) for patients struggling with opioid use disorder (OUD). Added Dr. Rosenberg, “This is a great day for our patients.

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The Pharma Data

MARCH 27, 2021

Novo Nordisk today announced that the Committee for Medicinal Products for Human Use (CHMP) under the European Medicines Agency (EMA) has recommended that the use of Saxenda ® is expanded for the treatment of obesity in adolescents aged 12–17 years. If approved, Saxenda ® will be the first EU-approved treatment for obesity in adolescents.

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The Pharma Data

JULY 25, 2021

Allergan, an AbbVie company, today announced new data, including the full results from the Phase 3 GEMINI 1 clinical study, evaluating the efficacy, safety and tolerability of investigational AGN-190584 (pilocarpine 1.25%) ophthalmic solution for the treatment of presbyopia. of patients discontinuing due to adverse events.

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Drug Target Review

JULY 1, 2024

As a non-opioid, SRP-001 also eliminates abuse potential, positioning it as a safer and effective drug candidate for the treatment of acute and neuropathic pain and migraine headache. In Phase I clinical trials, SRP-001 showed no serious adverse events among 56 healthy volunteers, highlighting its safety. South Rampart Pharma, Inc.

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