pharmaphorum

JANUARY 27, 2023

Developing medicines – for people living with disease Dr Mullen’s current role involves providing medical monitoring support, as well as safety, pharmacovigilance, scientific, and medical advice across a range of client projects, including advanced therapies and orphan drugs. I still feel that it is very important,” Dr Mullen said. “We’ve

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XTalks

MAY 26, 2023

The regulatory nod “ushers in a whole new paradigm to treat genetic diseases” and is a major milestone for DEB patients and their families, Krystal Biotech CEO Krish Krishnan said in a statement from the company. Results of the trials are published in the journals Nature Medicine and The New England Journal of Medicine.

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Gene Therapy Gene FDA Approval Dermatology 97

XTalks

DECEMBER 22, 2021

If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the life sciences. The development and widespread adoption of new technologies is key to revolutionizing the way we diagnose, prevent, treat and manage disease. The RNA Revolution: From mRNA Vaccines to RNA Editing.

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Life Science RNA Vaccination Vaccine 98

XTalks

NOVEMBER 27, 2023

Pompe disease primarily manifests as progressive muscle weakness, particularly affecting the skeletal muscles in regions such as the hips, legs, shoulders, arms and diaphragm. Sanofi also offers another ERT for Pompe disease known as Myozyme, which received FDA approval in 2006, and Lumizyme in 2010 for late-onset disease.

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XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. All clinical trials rely on a well-oiled machine of motivated professionals, but nowhere is this more true than in advanced-therapy studies in rare diseases where knowledge is sparse. Collaboration.

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XTalks

FEBRUARY 28, 2022

Related: UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD. Below are some facts and information about rare diseases, including rare disease clinical trials and orphan drugs. How is a Rare Disease Defined? Are Most Rare Diseases Genetic?

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Clinical Trials Clinical Trials Genetics Trials 98

XTalks

FEBRUARY 27, 2023

Attendees will learn about the types of assessments and scales that are feasible to conduct remotely in rare disease trials. Attendees will learn about the types of assessments and scales that are feasible to conduct remotely in rare disease trials.

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XTalks

DECEMBER 13, 2023

This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader.” FDA officials said it had not seen such cases in Vertex patients, but would look out for side effects.

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XTalks

SEPTEMBER 22, 2022

Clinical Trial Behind Spevigo. The efficacy of Spevigo (spesolimab) was determined based on the results of the Effisayil 1 double-blind, randomized, Phase II clinical trial. GPP occurs in individuals with specific gene variations as it is a genetic disease , and variations in the genes IL36RN and AP1S3 are known to cause it.

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FDA Approval Clinical Trials Clinical Trials Dermatology 52

pharmaphorum

DECEMBER 8, 2021

Acadia Pharma’s trofinetide has hit its efficacy targets in a phase 3 trial involving girls with the devastating neurological disorder Rett syndrome, although safety has dampened enthusiasm a little. Results from a phase 3 trial of blarcamesine are due next year. At 12 weeks, trofinetide-treated patients had a 5.1-point

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Marketing Drugs Life Science Trials 59

XTalks

MAY 12, 2023

What’s been shown in mouse models of Rett syndrome which also have the genetic disease, is that trofinetide helps strengthen those connections between the neurons. So, although it’s not a genetic treatment for Rett syndrome, it does act on the core pathophysiology behind Rett syndrome, which is that synaptic loss.

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Delveinsight

FEBRUARY 11, 2021

Verily, Google’s life-science-focused sibling company and Janssen will also seek to tap into the data generated by people during their everyday lives to seek for any previous health-related signals in the two years leading up to the point they consented to participate in the study as well as in the two years after.

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Gene Therapy Gene Immune Response In-Vivo 52

XTalks

NOVEMBER 10, 2023

Related: Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio The FDA approval of Adzynma was based on its demonstrated safety and efficacy in a Phase III global cross-over study, the first of its kind for cTTP. Adzynma’s approval is the second for Takeda this week.

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FDA Approval Gene Therapy Gene Clinical Trials 59

The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Of course, this is how biotech investments work.

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In-Vivo Marketing Engineer Genetics 52

Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. These challenges exist across the translation cycle from early research to clinical trials, manufacturing, and ultimately reimbursement.

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Gene Therapy Gene Development Production 52

XTalks

SEPTEMBER 12, 2022

Patients in the trial received Xenpozyme or placebo for 52 weeks. In the trials, 75 percent of pediatric patients and 50 percent of adult patients experienced adverse reactions while receiving Xenpozyme that included headaches, nausea and vomiting. Treatment with Xenpozyme improved lung function and also reduced liver and spleen size.

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The Pharma Data

DECEMBER 8, 2020

December is bringing frost in the North and plenty of cold hard cash for these life sciences companies. . European venture capital firm Forbion rounded up $545 million for its fifth life sciences fund. Silverback’s lead candidate is currently in a Phase I trial in adults with HER2-expressing solid tumors.

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RNA Antibody Life Science Protein 52

XTalks

MARCH 3, 2021

BridgeBio is dedicated to developing therapies for genetic diseases with unmet needs. Nulibry Trial Results. The efficacy of Nulibry in the treatment of MoCD Type A was demonstrated in data from three different clinical trials that was compared to data from a natural history study.

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XTalks

AUGUST 6, 2020

TSC is a rare genetic disease that affects approximately 1 in 6,000 people. The effectiveness of Epidiolex for the treatment of TSC-associated seizures was demonstrated in a randomized, double-blind, placebo-controlled trial.

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XTalks

MARCH 21, 2024

MLD is a rare genetic disease affecting the brain and nervous system caused by a deficiency of the enzyme arylsulfatase A (ARSA), which results in the accumulation of sulfatides (fatty substances) in cells. The transaction has proven fruitful with the milestone US approval of the gene therapy just a few months later.

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XTalks

DECEMBER 13, 2023

The company’s sustained investment in R&D has led to a rapid expansion of its pipeline in 2023, with notable milestones such as four infectious disease vaccines in Phase III trials, including the recently submitted respiratory syncytial virus (RSV) vaccine for regulatory approval.

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Drug Discovery World

APRIL 30, 2024

At the start of 2024, the first patients in a clinical trial investigating an mRNA cancer therapy were dosed. The Mobilize trial is investigating the mRNA-4359 therapy and its potential for treating melanoma, lung cancer and other solid tumour cancers. We believe it can now do the same for rare diseases.” “We

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RNA Protein Vaccination Vaccine 52

XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). He added that, “A deep body of evidence now points to the potential for durable effects in HSC gene therapy for certain severe genetic diseases including MLD.

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XTalks

SEPTEMBER 13, 2021

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

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RNA Genetics Genetic Disease Medicine 105

Drug Discovery World

MARCH 6, 2024

Guiding neurological drug discovery and development to success The 20 years since the Human Genome Project has seen transformational advances in the molecular understanding of cancers and rare genetic diseases, leading to genetically informed, personalised selection of therapies and massively improved outcomes for many patients.

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XTalks

APRIL 15, 2021

million people in the US, yet there is no treatment for the most common form of the disease: heart failure with preserved ejection fraction (HFpEF). A lack of efficacy is the greatest reason for clinical trial failures, especially in Phase II and Phase III,” says Dr. Tyl. Heart failure affects about 6.2

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Development Drugs Genome Genomics 98

XTalks

AUGUST 29, 2022

CRISPR technology has begun to enter clinical trials due to emerging therapeutic applications, but the technology still has limitations, primarily because it is difficult to safely make large quantities of precisely edited cells.

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XTalks

OCTOBER 1, 2020

The drug is currently in clinical trials for alcoholic hepatitis (AH), nonalcoholic steatohepatitis (NASH), as well as in COVID-19 patients with acute liver or kidney injury. Working on finding therapies for genetic diseases at NeuBase, Dr. Sooter says, “Everyday, we get up and that’s our goal and it really drives everyone.

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Scientist In-Vitro Research Life Science 52

The Pharma Data

OCTOBER 15, 2020

Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Prior to joining Inozyme, Sabbagh served as the head of rare renal and musculoskeletal diseases research at Sanofi.

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