Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

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Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

Pharmaceutical Technology

FEBRUARY 2, 2023

The regulatory approval is based on the data obtained from the ASCEND-D trial, which is evaluating Jesduvroq’s safety and efficacy to treat patients. The ASCEND programme included five Phase III trials that enrolled more than 8,000 patients who received treatment for up to 4.26 dL) for the patients.

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FDA Approval Life Science Genetics Trials 264

XTalks

APRIL 3, 2025

Eli Lilly shared new Phase II trial data showing that its experimental siRNA therapy, lepodisiran, leads to reductions in levels of lipoprotein(a) (Lp(a)) a genetically inherited risk factor for heart disease in adults with elevated levels of the marker. The 400 mg dose was the highest lepodisiran dose in the trial.

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Trials Clinical Trials Clinical Trials Genetics 59

XTalks

JANUARY 6, 2025

CF is a progressive genetic disease caused by defective CFTR proteins, which are crucial for regulating salt and water movement in cells. In clinical trials, Alyftrek demonstrated non-inferiority to Trikafta, showing similar improvements in lung function and a reduction in sweat chloride levels.

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XTalks

DECEMBER 9, 2024

Despite its potential to slow cognitive decline, the delays highlight the challenges of timely drug approval for life-threatening conditions. This initiative is particularly impactful for rare diseases and genetic disorders, where traditional endpoints may be infeasible.

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Clinical Trials Clinical Trials Compliance Trials 105

XTalks

SEPTEMBER 14, 2022

In this episode, Ayesha discussed the FDA approval of Sanofi’s enzyme replacement therapy Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare genetic lysosomal storage disease, in adults and pediatric patients.

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XTalks

MAY 23, 2024

One of the more intriguing developments in cancer research in recent years is the growing understanding of clonal hematopoiesis, a phenomenon where blood cells expand from a single clone due to genetic mutations. Clonal hematopoiesis increases in prevalence with age and can precede hematologic malignancies.

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Genetics Research Genome Genomics 114

XTalks

NOVEMBER 15, 2024

Drug resistance in DEEs often stems from genetic and molecular factors unique to each syndrome, limiting standard treatments’ effectiveness. Current treatment options remain limited, and many patients have not had opportunities to participate in trials specifically tailored to their condition.

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Trials Clinical Trials Clinical Trials Genetics 97

XTalks

DECEMBER 4, 2024

Proceeds from the IPO will propel key initiatives, including the Phase II clinical trial of Jotrol in Parkinson’s disease. While Jupiter faces financial uncertainties and challenges in scaling operations in competitive markets, it remains committed to refining grant applications for proof-of-concept trials.

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Bioavailability Genetic Disease Marketing RNA 111

XTalks

NOVEMBER 14, 2024

This therapy, the first of its kind to be administered directly into the brain, addresses a rare genetic disorder that severely affects motor function and dopamine production. Kebilidi has a known safety profile from clinical trials. for this purpose. Common side effects include involuntary movements, fever and low blood pressure.

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Gene Therapy FDA Approval Gene Clinical Trials 105

XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio. Of the more than 10,000 rare diseases, more than 80 percent are caused by genetic defects.

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Gene Therapy Clinical Trials Clinical Trials Gene 98

XTalks

MARCH 2, 2022

The editorial team discussed the encouraging growth seen in recent years in rare disease clinical research in the life science industries, providing hope for improved diagnosis, care and treatments for patients with rare diseases. In the US, Black people only constitute five percent of all clinical trial participants.

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Life Science Clinical Trials Clinical Trials Trials 52

XTalks

DECEMBER 12, 2023

The world of neuroscience trials is rapidly evolving, presenting new challenges and opportunities for researchers and clinicians. The worldwide market for neurology clinical trials stood at a valuation of $5.24 There is currently a heightened emphasis on imaging and liquid biomarkers in neuroscience clinical trials.

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Trials Clinical Trials Clinical Trials Genetics 111

XTalks

FEBRUARY 21, 2025

Current Share Price: PWS is a complex, genetically determined neurodevelopmental disorder that affects multiple organ systems. And hyperphagia is common not only to PWS but also to hypothalamic obesity, binge-eating disorder and certain rare genetic disorders like Bardet-Biedl syndrome.

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XTalks

AUGUST 7, 2024

Lawreen is a seasoned biotech and life science professional with over 25 years of experience. Tune into the episode to hear Lawreen’s perspectives as both a life sciences professional and rare disease patient advocate. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

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XTalks

DECEMBER 12, 2024

In clinical research, this includes designing trials that elevate the patient voice, reduce barriers to participation, and represent the diverse populations who will ultimately use these medical products. Access to information: Limited resources and educational materials hinder informed decision-making about trial participation.

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Clinical Trials Clinical Trials Clinical Research Trials 59

XTalks

APRIL 15, 2025

The approval marks the first FDA-approved therapy specifically targeting hyperphagia in this rare genetic disorder. Vykay XR Clinical Trial Results Data from a randomized, placebo-controlled Phase III trial (Study 2-RWP or Study C602-RWP) support Vykat XRs approval. The FDA rejected it in 2022.

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FDA Approval Clinical Trials Clinical Trials Trials 59

XTalks

DECEMBER 28, 2023

BillionToOne, a company specializing in molecular diagnostics, has partnered with Janssen Research & Development, LLC, a Johnson & Johnson division, to conduct the AZALEA Phase III clinical trial globally. The trial is set to occur across the US, the European Union (EU) and selected global locations.

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Trials Clinical Trials Clinical Trials Antibody 97

XTalks

DECEMBER 31, 2024

Related: Neurocrine Biosciences Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH In a statement announcing the trial results, Hitto Kaufmann, chief R&D officer, Hansa Biopharma, said, Our Phase II study results and the indirect treatment comparison with IGOS are critically important.

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Life Science Clinical Trials Clinical Trials Antibody 52

XTalks

FEBRUARY 28, 2024

Advancements in rare disease research are leading to innovative new treatments for various rare conditions, including genetic disorders, which make up just over 70 percent of all rare diseases. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

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Life Science Research Genetics Drugs 52

pharmaphorum

DECEMBER 9, 2022

has announced that the pivotal phase 2 FELIX clinical trial of obecabtagene autoleucel (obe-cel) in relapsed/refractory (r/r) adult Acute Lymphoblastic Leukaemia (ALL) patients has met its primary endpoint of overall remission rate (ORR) at interim analysis. (Nasdaq: AUTL), a clinical-stage biopharma and a portfolio company of Syncona Ltd.,

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Trials Clinical Trials Clinical Trials Gene Therapy 98

Pharmaceutical Technology

JANUARY 22, 2023

While in the US, the therapy is already being prescribed under an expanded access program , the FDA has requested additional data from a randomised, placebo-controlled trial before it will allow Egetis to schedule a PDUFA date. In clinical trials, the drug showed a 3.8-point point improvement (unadjusted means) and a 3.2-point

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XTalks

DECEMBER 2, 2024

The approval is grounded in data from five randomized, double-blind, placebo-controlled trials involving 317 adults. He expressed optimism about the therapy’s potential to enhance outcomes for patients in the US.

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Trials Marketing FDA Approval Life Science 104

Pharmaceutical Technology

NOVEMBER 29, 2022

The company now plans to move this approach into a Phase III trial. PharmaTher also has an agreement in place with Case Western Reserve University to develop ketamine as a treatment for Rett syndrome, a rare genetic neurological disorder. PharmaTher is not the only player in this space. Still, Funding is not the only issue.

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Research Trials Drugs Generic Drugs 275

XTalks

APRIL 5, 2024

Amylyx Pharmaceuticals announced yesterday that it will be pulling its ALS (amyotrophic lateral sclerosis) drug Relyvrio from the market after failing in a confirmatory trial. Last month, Amylyx reported results from the global PHOENIX Phase III clinical trial that were unable to confirm the drug’s effectiveness.

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Trials Drugs Marketing FDA Approval 98

XTalks

SEPTEMBER 17, 2020

In rare disease trials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. IGF-1 is an acceptable surrogate endpoint in trials of growth hormone receptor agonists like Somavert, and is preferred for pediatric patient populations.

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Trials Gene Hormones Clinical Trials 98

XTalks

APRIL 13, 2022

Pediatric rare disease clinical trials are vital to determining the safety and efficacy of medications for children living with a rare disease. About 80 percent of rare diseases have a genetic basis. The World Health Organization (WHO) estimates that there are between 5,000 and 8,000 rare diseases worldwide.

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Clinical Trials Clinical Trials Clinical Research Trials 98

XTalks

MARCH 21, 2025

The company is planning Phase III trials in bipolar disorder and Alzheimers. The company recently reported topline results from its Phase I/II trial of AURN001, an allogeneic cell therapy for corneal edema. The Phase I/II trial showed significant improvements in vision and corneal clarity. million in Q4 2024.

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Genome Genomics FDA Approval Trials 59

XTalks

FEBRUARY 24, 2023

When life sciences industry partners with patients and their advocacy communities, the patient voice becomes integrated into the drug development process. It’s an increasingly common and evolving practice that has brought about meaningful change in the clinical trial process.

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Development Clinical Trials Clinical Trials Gene Therapy 98

XTalks

MARCH 17, 2025

Recent pre-mass production trials tests conducted to ensure that the devices meet performance and manufacturing quality standards before full-scale production demonstrated that the A+Pre chip recovered 94% of tumor cells, while the AC-1000 achieved a 79.5% recovery rate, surpassing their performance targets.

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Production Manufacturing DNA Genetics 71

XTalks

APRIL 17, 2025

Approximately 80% of these diseases have a genetic basis , and many manifest in childhood , often with significant challenges in achieving a timely and accurate diagnosis. By exploring rare diseases and focusing on HPP, we can better understand the complexities of genetic disorders and the need for patient-centric research and collaboration.

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Clinical Research Research Clinical Trials Clinical Trials 52

pharmaphorum

OCTOBER 7, 2020

There is enormous power in this genetic tool, which affects us all. It has not only revolutionised basic science, but also resulted in innovative crops and will lead to ground-breaking new medical treatments,” said Claes Gustafsson, chair of the Nobel Committee for Chemistry. What started as a curiosity?driven,

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Gene Editing Bacteria DNA Gene 105

XTalks

NOVEMBER 6, 2020

Canadian clinical-stage biotech company Symvivo Corporation has developed an oral COVID-19 vaccine that entered clinical trials this week. The first healthy volunteer was dosed with the vaccine in Australia as part of the bacTRL-Spike COVID-19 Phase I clinical trial. COVID-19 Clinical Trials.

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Clinical Trials Clinical Trials Vaccination Vaccine 75

pharmaphorum

FEBRUARY 17, 2021

One group excelling in this space is the Genetic Alliance. Their work ranges from developing new services in partnership with the National Health Service (NHS) to working with the All Party Parliamentary Group on Rare, Genetic and Undiagnosed Conditions, and the Department of Health on the UK Strategy for Rare Diseases.

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XTalks

DECEMBER 1, 2020

As the research and medical community learns more about population variances in disease outcomes, whether as a result of regional genetic profiles, socioeconomic factors, or environmental inputs, it’s become necessary to ensure that clinical research trials include broad swaths of global participants. billion in 2019.

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Clinical Trials Clinical Trials Trials Clinical Research 96

XTalks

FEBRUARY 19, 2025

It occurs when a genetic error causes the overproduction of a protein called CSF1, which attracts inflammatory cells to the joint, often causing pain, swelling and reduced mobility. Looking ahead, Deciphera is exploring additional clinical trials and potential collaborations in the TGCT space.

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FDA Approval Drugs Trials Clinical Trials 59

pharmaphorum

OCTOBER 16, 2020

In the age of artificial intelligence, no trial data should be going to waste. These might be licensed drugs that could hold potential for a patent extension, or drugs which failed efficacy trials for an intended indication. Over the last five years we have focused on the power of drug repurposing for rare genetic diseases.

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Trials Clinical Trials Clinical Trials Life Science 105