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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The Pharma Data

NOVEMBER 27, 2020

FDA Approves Imcivree (setmelanotide) for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency. Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. BOSTON, Nov.

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pharmaphorum

AUGUST 13, 2020

Viltepso can be used to treat around 8% of DMD patients and previously approved DMD drugs has not been shown to change the course of the disease. It’s bad form to compare performance of drugs in separate clinical trials, but results suggest that Viltepso helps patients produce more dystrophin than Sarepta’s rival Vyondys 53 (golodirsen).

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Drug Discovery World

OCTOBER 18, 2023

We’re beginning to see their potential come to fruition, with the FDA having approved three treatments as of January 2023 — for retinal dystrophy 1 spinal muscular atrophy, 2 and haemophilia B. However, NHP models cannot fully predict AAV transduction in the human liver.

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Advarra

JANUARY 11, 2024

Personalized medicine using genetic resequencing techniques, such as cell and gene therapies, enables researchers to create more customized therapies for combatting cancer. Many monoclonal antibodies are used to treat cancer by targeting specific cell structures regulating cell activity, causing the death of tumor cells.

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XTalks

SEPTEMBER 17, 2021

The ALS Association is pushing for rapid FDA approval of Amylyx Pharmaceuticals Inc.’s Accelerated FDA Approval Modeled on Aduhelm. This has brought criticism that US regulators are moving too slow on a drug that has clinical promise and is being backed by other international regulators.

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Drug Discovery World

APRIL 4, 2023

Orserdu (elacestrant), Stemline Therapeutics The first and only treatment specifically indicated for patients with ESR1 mutations in ER+, HER2- advanced or metastatic breast cancer was approved in the US in February. This made Jesduvroq is the first oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) to be approved in the US.

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XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies.

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XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Daybue (trofinetide) is the first and only FDA-approved drug for Rett syndrome.

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XTalks

JUNE 28, 2023

The approval is restricted to ambulatory patients within this narrow age range due to uncertainty around its effectiveness in older children, which Sarepta hopes to clarify in a confirmatory trial. DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness.

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Drug Discovery World

NOVEMBER 14, 2023

A very rare, inherited blood clotting disorder, cTTP is caused by a disease-causing mutation in the ADAMTS13 gene, which is responsible for making an enzyme, also named ADAMTS13, that regulates blood clotting. A deficiency in this enzyme causes blood clots to form in the small blood vessels throughout the body.

FDA Approval 52

FDA Approval Gene Clinical Trials Clinical Trials 52

pharmaphorum

DECEMBER 9, 2021

An FDA advisory committee has delivered a blow to Reata Pharma, after voting unanimously that the drugmaker’s data on bardoxolone – a drug for kidney disease – did not show it is effective. Advisors also had some concerns about the safety of the drug and the design of the pivotal trial used to support the marketing application.

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Drugs Clinical Trials Clinical Trials Genetic Disease 52

Pharmaceutical Technology

APRIL 5, 2023

It will be six years since the US FDA approval of Kymriah in August this year. Bruce Levine [BL]: We now have six approved CAR-T therapies. And my estimate is that between 25,000 and 30,000 patients have been treated with those therapies globally, which does not include patients treated in clinical trials.

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Drug Discovery World

AUGUST 14, 2023

Yet, there remains important regulatory differences between the EMA and the FDA, such as varying standards, approaches and even nomenclature for CGT. More importantly, the FDA and EMA have divergent interpretations of Chemistry, Manufacturing, and Controls (CMC) requirements, though one difference was recently clarified by the EMA.

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STAT News

MARCH 6, 2023

Unlike most people who receive medications yet to be cleared by the Food and Drug Administration, none of these patients is getting the vaccines as part of a clinical trial. Instead, the 26 patients paid the foundation to create the vaccines and provide them through the FDA’s expanded access program.

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Drug Discovery World

MAY 24, 2023

G protein-coupled receptors (GPCRs) are a large family of proteins that regulate many processes in the body and are the target of one third of clinically used drugs. Several FDA-approved drugs that target the NK1 receptor are used to prevent nausea and vomiting associated with chemotherapy or surgery.

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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. In 1970, the FDA approved ketamine as an anesthetic.

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The Pharma Data

JANUARY 29, 2021

Additional safety and efficacy data for the vaccine will continue to accumulate from ongoing clinical trials and is expected to be published in the coming weeks. The safety data published so far is from over 20,000 participants enrolled across four clinical trials in the UK, Brazil and South Africa. Source: AstraZeneca

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The Pharma Data

JANUARY 29, 2021

Additional safety and efficacy data for the vaccine will continue to accumulate from ongoing clinical trials and is expected to be published in the coming weeks. The safety data published so far is from over 20,000 participants enrolled across four clinical trials in the UK, Brazil and South Africa. Source: AstraZeneca.

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XTalks

FEBRUARY 5, 2024

Revlimid (Lenalidomide) Revlimid 2022 sales: $9.978 billion Company/Developer: Bristol Myers Squibb Date of first FDA approval: December 27, 2005 Indications Revlimid is FDA-approved for: Myelodysplastic disease (MDS), multiple myeloma, mantle cell lymphoma (MCL), follicular lymphoma and marginal zone lymphoma.

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Drug Discovery World

APRIL 14, 2023

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

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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. Most rare diseases are chronic conditions, and many are clinically progressive and potentially fatal. adults).

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Drug Discovery World

AUGUST 3, 2023

Medical cannabis is often a first-thought when regarding plant-based medicines, but there remains public scepticism, arguably due to the regulations relating to the substance. For example, the FDA has not approved a marketing application for cannabis for the treatment of any disease or condition to date 1.

Medicine 52

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Cloudbyz

MAY 19, 2021

Moreover, 12% of industrial clinical pipeline products and at least 16% of preclinical pipeline products consist of CGT. The primary drivers for growth include increasing cancer and genetic cases and predicated applicability of CGT as a treatment. 2 CAR-T cell therapies proposed by Bristol-Myers Squibb were approved by FDA in 2020.

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XTalks

DECEMBER 29, 2022

As of December 16, 2022, there are seven US Food and Drug Administration (FDA) approved gene therapies. In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. In 2023, a number of gene therapies are expected to get the FDA green light.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

XTalks

FEBRUARY 5, 2024

Revlimid (Lenalidomide) Revlimid 2022 sales: $9.978 billion Company/Developer: Bristol Myers Squibb Date of first FDA approval: December 27, 2005 Indications Revlimid is FDA-approved for: Myelodysplastic disease (MDS), multiple myeloma, mantle cell lymphoma (MCL), follicular lymphoma and marginal zone lymphoma.

FDA Approval 52

FDA Approval Drugs Sales Development 52

The Pharma Data

JANUARY 6, 2021

The vaccine was shown in clinical trials to be safe and effective at preventing symptomatic COVID-19, with no severe cases and no hospitalisations more than 14 days after the second dose. The approval in India is an important milestone as it will enable to supply India but also a large number of countries around the world.

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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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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. Surrogate endpoints were used as the basis for approval of 45 percent of new drugs reviewed by the FDA between 2010 and 2012. Duchenne Muscular Dystrophy. Prevalence: 1 in 3,500.

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The Pharma Data

DECEMBER 18, 2020

In making this determination, the FDA can assure the public and medical community that it has conducted a thorough evaluation of the available safety, effectiveness, and manufacturing quality information. The Moderna COVID-19 Vaccine contains messenger RNA (mRNA), which is genetic material. The vaccine was 94.1% ModernaTX, Inc.

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Vaccination Vaccine Production Manufacturing 52

XTalks

MARCH 13, 2023

The US Food and Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) held a recent town hall where three experts from the regulator provided guidance on how to design and conduct gene therapy clinical trials for rare diseases.

Gene Therapy 52

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XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. In 2020, 31 out of 53 novel drug approvals were for rare or orphan diseases​​. The FDA introduced its Orphan Drug Act in 1983.

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XTalks

NOVEMBER 20, 2023

The newly approved Truqap and Faslodex combo is restricted to patients with a genetic alteration in one or more of the following PI3K/AKT pathway genes: PIK3CA , AKT1 or PTEN. PI3K/AKT is a key intracellular signaling pathway that regulates cell survival and proliferation. It was also given FDA priority review.

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HR FDA Approval Gene Trials 98

XTalks

JULY 27, 2022

The exact causes of ADHD are unknown, but genetics may play a key role. XTALKS WEBINAR: Delivering Patient Data through a Regulated Platform to Improve Trial Oversight. Register for this free webinar to learn about how one can increase operational efficiencies and enhance the patient experience in clinical trials.

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Clinical Trials Clinical Trials Trials FDA Approval 98

The Pharma Data

JANUARY 21, 2021

INSTIs, like cabotegravir, inhibit HIV replication by preventing the viral DNA from integrating into the genetic material of human immune cells (T-cells). Pregnancy: In a clinical trial, total rilpivirine exposures were generally lower during pregnancy compared to the postpartum period.

FDA Approval 52

FDA Approval RNA Drugs Containment 52

pharmaphorum

FEBRUARY 11, 2022

Three patients from the phase 1/2 trial – two aged under six at the time of treatment and one aged 13 – showed improvements in skills like cognition, expressive language and fine motor skill acquisition than would be expected if untreated. The post Regenxbio expands MPS I gene therapy trial after first look at data appeared first on.

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XTalks

MARCH 25, 2021

Since joining Xtalks in 2015, she has written over 1,000 articles on diverse topics from remote clinical trials and rare disease to medical device sterilization and drug pricing. Ayesha obtained an MSc in Cancer & Genetics from McMaster University and a PhD in Medical Biophysics from the University of Toronto.

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