Drug Discovery World

FEBRUARY 1, 2024

These deposits can cause progressive damage to the brain and other areas of the body. Disease symptomatology is characterised by chronic diarrhea during infancy and cataracts in late childhood. At present there is no cure for CTX, only treatment to slow disease progression. Vivet Therapeutics has received €4.9

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Drug Discovery World

SEPTEMBER 4, 2024

Evommune’s pre-clinical data demonstrates that by blocking activation of MRGPRX2 via all disease relevant ligand categories, EVO756 can prevent the degranulation of mast cells.

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Drug Discovery World

OCTOBER 3, 2023

The US Food and Drug Administration (FDA) has announced a pilot programme with the aim of accelerating the development of novel drug and biological products for rare diseases. The post FDA pilot programme to boost rare disease therapies appeared first on Drug Discovery World (DDW).

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Drug Discovery World

DECEMBER 4, 2023

Smoking continues to be the leading cause of preventable death and disease, leading to almost eight million direct and indirect deaths annually. The post Inhaled smoking cessation therapy superior to other treatments appeared first on Drug Discovery World (DDW).

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Drug Discovery World

JULY 6, 2023

The first patient has been dosed in the clinical Phase I evaluation of the Sema3A monoclonal antibody, part of the Evotec-Bayer multi-target research collaboration in kidney diseases. Sema3A is upregulated in injured human kidneys and implicated in the development and progression of acute and chronic kidney diseases.

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Drug Discovery World

JUNE 21, 2023

Citius Pharmaceuticals has revealed positive results from its Phase IIb Study of Halo-Lido (CITI-002) for the treatment of haemorrhoids. Following the 21 st Century Cures Act, higher emphasis is placed on using Patient Reported Outcome (PRO) instruments in clinical trials.

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Drug Discovery World

JUNE 6, 2023

BenevolentAI, developers of AI that accelerates biopharma discovery, has delivered BEN-34712, its pre-clinical candidate for the potential treatment of amyotrophic lateral sclerosis (ALS). ” The post BenevolentAI progresses drug for the potential treatment of ALS appeared first on Drug Discovery World (DDW).

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Drug Discovery World

OCTOBER 4, 2022

William Erhardt , President, Head of Development & Operations of biotech Oligomerix, tells Lu Rahman about the company’s differentiated approach to developing disease-modifying therapeutics for neurodegenerative diseases. . With its recent award of $3.35 Overcoming sector challenges .

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Drug Discovery World

SEPTEMBER 14, 2023

6,7 “For patients with advanced UC, including FGFR-driven tumours, outcomes remain poor and treatment options are limited; therefore, there is a need for novel, targeted therapies,” said Martin Vogel, EMEA Therapeutic Area Lead Oncology, Janssen-Cilag GmbH. “We Erdafitinib for the treatment of metastatic bladder cancer. J Clin Med.

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Drug Target Review

AUGUST 28, 2024

What are the main challenges currently faced in the treatment of chronic liver diseases, and how does Resolution Therapeutics aim to address these challenges? Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years.

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Drug Target Review

JUNE 9, 2023

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

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Drug Discovery World

JULY 12, 2024

The US Food and Drug Administration (FDA) has granted Fast Track Designation for Asklepios BioPharmaceutical’s (AskBio) AB-1005 which is being developed for moderate Parkinson’s disease. AB-1005 is an investigational adeno-associated virus 2 glial cell line-derived neurotrophic factor (AAV2-GDNF) neurorestorative gene therapy.

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Drug Discovery World

FEBRUARY 27, 2024

The results support the continued clinical development of VXX-401 as a candidate for the treatment of hypercholesterolemia and prevention of atherosclerotic cardiovascular disease. VXX-401 is currently in a Phase I clinical trial for safety and tolerability, with topline data expected in mid-2024.

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Drug Discovery World

JUNE 26, 2024

Biotechnology company EG 427 has revealed plans to start a Phase Ib/IIa clinical study in neurogenic detrusor overactivity (NDO), following Investigational New Drug (IND) clearance from the US Food and Drug Administration (FDA). It is a step forward in bringing the potential benefits of gene therapy to large disease populations in neurology.

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Drug Discovery World

DECEMBER 22, 2022

Shelley McLendon , SVP of Vaccine and Infectious Diseases Program Management at ICON, attended the World Vaccine Congress Europe from 16-19 October 2022 in Barcelona, Spain. Disease surveillance and reporting . Disease surveillance is the collection and monitoring of data on disease within a population.

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Conversations in Drug Development Trends

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment.

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Conversations in Drug Development Trends

MAY 14, 2024

By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare disease research. Common goals empower change.

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FDA Law Blog: Drug Discovery

JUNE 12, 2024

Sasinowski — Hyman, Phelps & McNamara (HPM) would like to congratulate the 7 rare disease programs selected for the inaugural class of the FDA’s “Support for clinical Trials Advancing Rare disease Treatment” (START) pilot program. HPM is proud to aid 5 of the 7 selected participants for the START pilot program.

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Cytel

MAY 28, 2024

Written by Susannah Sadler and Stacy Grieve Launching a treatment for inflammatory diseases can be a make-or-break endeavor. A robust evidence base and a compelling value story are crucial to success, but current challenges and unexpected bumps in the road will require creative solutions.

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Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

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Drug Discovery World

JULY 5, 2024

People with FTD who have disease-causing GRN mutations produce a reduced amount of progranulin protein. AVB-101 is a potential one-time therapy designed to stop disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels to affected areas of the brain.

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Cytel

APRIL 10, 2024

Orphan drug designation is a regulatory status granted to pharmaceuticals developed for the treatment of rare diseases. It provides incentives to encourage the research, development, and approval of therapies targeting small patient populations.

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Drug Discovery World

FEBRUARY 26, 2024

The US Food and Drug Administration (FDA) has granted Fast Track Designation for Certa Therapeutics’ investigational therapy FT011 for the treatment of systemic sclerosis (scleroderma). The post Scleroderma drug gets fast-tracked in the US appeared first on Drug Discovery World (DDW).

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Drug Discovery World

SEPTEMBER 5, 2023

BenevolentAI has dosed the first patients in Phase I first-in-human studies of its oral phosphodiesterase 10 (PDE10) inhibitor, BEN-8744, intended for the treatment of ulcerative colitis (UC). UC is a chronic disease that causes inflammation and ulceration of the inner lining of the colon and rectum. UC affects 0.4%

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Drug Discovery World

NOVEMBER 30, 2023

Treatment with FT011 for 12 weeks resulted in a clinically meaningful improvement in 60% of patients treated with FT011 400mg and 20% of patients in the FT011 200mg group compared with 10% in the placebo group. I look forward to collaborating with the company in the FT011 development program ahead.”

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Drug Discovery World

JANUARY 25, 2024

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to Pleco Therapeutics’s lead compound PTX-252 for the treatment of acute myeloid leukaemia (AML). PTX-252 is a novel molecular entity developed to increase the sensitivity of cancer cells to chemotherapy.

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The Pharma Data

SEPTEMBER 29, 2021

to enhance outcomes, new treatments which will target kidney-specific disease mechanisms are highly desired,” said Hiddo L. Christian Rommel, Member of the chief Committee of Bayer AG’s Pharmaceutical Division and Head of Research and Development. 1 in 3 adults is at risk for the disease. In the U.S.,

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Drug Discovery World

MAY 29, 2024

All 11 trial participants had experienced disease progression in prior immunotherapy treatment before joining the trial. These preliminary findings represent some of the initial positive evidence emerging from the burgeoning field of clinical research on cancer therapies leveraging gut microbiota.

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Conversations in Drug Development Trends

MAY 14, 2024

By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare disease research. Common goals empower change.

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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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Drug Target Review

JUNE 3, 2024

How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? Essentially, our CNPs reprogramme the immune system by restoring balance and returning the body to a homeostatic state, offering a precise approach to treating immune-mediated diseases.

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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).

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LifeSciVC

JULY 17, 2024

By Mike Cloonan, Chief Executive Officer of Sionna Therapeutics, as part of the From The Trenches feature of LifeSciVC The drug development process in rare diseases is rife with challenges especially when companies target significant differentiation or first-in-class targets.

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The Pharma Data

NOVEMBER 29, 2020

Prior to joining Gannex, Dr. Palmer was Head of Liver Disease Clinical Development at Takeda Pharmaceuticals. She has also held senior leadership positions at Shire Pharmaceuticals and Kadmon Corporation, where she was in charge of the global development of NASH and other liver disease programs.

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Drug Discovery World

OCTOBER 14, 2022

“We have been collaborating with Merck on PCVs since 2016, and together we have made significant progress in advancing mRNA-4157 as an investigational personalised cancer treatment used in combination with KEYTRUDA,” said Stephen Hoge, President of Moderna.

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Drug Discovery World

MARCH 7, 2024

It has potential to be the first approved treatment for HSCT-TMA, a rare complication of stem cell transplantation that has an 80% mortality rate among severe adult and paediatric patients. Akari’s long-acting version of nomacopan (PASylated-nomacopan) is in the final stages of pre-clinical development for geographic atrophy (GA).

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Drug Discovery World

JANUARY 10, 2024

The acquisition includes Aiolos’ AIO-001, a potentially best-in-class, long-acting anti-thymic stromal lymphopoietin (TSLP) monoclonal antibody ready to enter Phase II clinical development for asthma, with potential for additional indications.

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