Drug Discovery World

DECEMBER 5, 2022

A recent study revealed that traditional research sources are missing evidence for up to 70% of clinically encountered variants, information that is essential to developing treatments for genetic disorders. This lack of comprehensive data increases the risk of program failure in clinical trials. .

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Delveinsight

AUGUST 17, 2021

Few of the common adverse reactions observed in patients who underwent Keytruda and Lenvima combination in clinical trials are fatigue , diarrhea , musculoskeletal pain , hypothyroidism, hypertension , stomatitis , decreased appetite, rash, nausea and decreased weight. The rarity of disease – 1.36

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Pharmaceutical Technology

JUNE 19, 2023

Pharmaceutical companies are putting their trust in the immense potential this new generation of medicine has for treating individuals with rare genetic diseases, which currently affect an estimated 280 million patients worldwide. Cell and gene therapies are not your typical clinical supply chain.

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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.

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Drug Discovery World

MARCH 8, 2023

The manufacturer, Genethon, is a non-profit gene therapy R&D organisation founded by the French Muscular Dystrophy Association (AFM-Telethon). PRIME status was granted to GNT-0003 following promising results from early phases of the European trial currently underway in collaboration with the CureCN consortium, and sponsored by Genethon.

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The Pharma Data

NOVEMBER 27, 2020

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. With this approval, Imcivree becomes the first-ever FDA approved therapy for these rare genetic diseases of obesity. BOSTON, Nov.

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XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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XTalks

NOVEMBER 27, 2023

“FDA approval of Pombiliti and Opfolda is a testament to the power of science, medicine and our passionate determination to improve the lives of people living with Pompe disease. I am just so immensely proud of our team, and so very grateful to everyone who has worked to bring this medicine to this approval.

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Drug Discovery World

AUGUST 28, 2023

DDW’s Diana Spencer speaks to Peter Hamley , Chief Scientific Officer of Samsara Therapeutics, to understand the role autophagy plays in neurodegenerative diseases and how boosting this process could increase healthspan. DS: Samsara’s autophagy inducing candidate SAM001 will be the first agent of its class to enter clinical trials.

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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. Gene-altering and other advanced medicines are radically rewriting the future for people with some of the rarest, and most devastating, diseases on earth. Integrating patients into all trial functions.

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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. Longer trials are needed to fully understand the effects and safety of Spevigo in those affected by GPP using the treatment long-term.

FDA Approval 52

FDA Approval Clinical Trials Clinical Trials Dermatology 52

Delveinsight

AUGUST 26, 2021

Shape’s RNA editing technology could potentially modify the amount of a key regulatory protein in the body or treat genetic diseases. The company then utilizes the de-identified records to improve the design of clinical trials and quality of life measurements. and Leila Zegna, director of the Kabuki Syndrome Foundation.

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pharmaphorum

JUNE 28, 2022

Xenpozyme – developed by Sanofi’s rare disease business Genzyme – replaces an enzyme that is deficient in ASMD, reducing fatty accumulation within cells with a dose delivered intravenously every two weeks. “The approval of Xenpozyme by the European Commission represents a transformational shift in what we can offer to patients.

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Genetic Disease Clinical Trials Clinical Trials Genetics 52

Drug Discovery World

NOVEMBER 14, 2023

Adzynma provides patients with a treatment option that replaces their deficient ADAMTS13 enzyme and offers a favourable efficacy and safety profile and reduced administration time and volume compared to current plasma-based therapies.

FDA Approval 52

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XTalks

MARCH 3, 2021

The US Food and Drug Administration (FDA) has granted approval to Nulibry (fosdenopterin) for reducing the risk of death associated with molybdenum cofactor deficiency type A (MoCD Type A), making it the first treatment for the ultra-rare, genetic metabolic disorder. Nulibry Trial Results.

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The Pharma Data

MARCH 16, 2021

This NDA is based on data from the Phase 2 Study-004 trial, in which belzutifan showed a confirmed overall response rate of 36.1% (n=22/61) (95% CI: 24.2-49.4) in patients with VHL disease-associated RCC. was awarded the Nobel Prize in Physiology or Medicine in 2019. About Von Hippel-Lindau Disease and Renal Cell Carcinoma.

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The Pharma Data

OCTOBER 27, 2020

The company plans to tackle the existing challenges of precision medicine with a three-prong approach – developing better medicines for known cancer-driving mutations, developing drugs for targets previously thought to be undruggable, and finding new, untapped targets that could lead to even better drugs. Sirnaomics. Be Biopharma .

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The Pharma Data

JANUARY 17, 2021

n-Lorem selects patients based on their genetic mutation and the organ affected rather than their specific diseases. “We It’s the cause of the mutation, not the name of the disease, that matters.”. They’re basically the same,” but each one uses a particular nucleotide to bind to specific genetic sequence.

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Genetics Clinical Trials Clinical Trials Drugs 52

Drug Discovery World

MAY 11, 2023

The RSV candidate is the lead program, and the company is working towards generating the pre-clinical data to support a Phase I clinical trial.

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The Pharma Data

NOVEMBER 11, 2020

Received Rare Pediatric Disease and Fast Track Designations for INZ-701 for the treatment of ENPP1 deficiency. Expect to initiate INZ-701 Phase 1/2 clinical trials for ENPP1 and ABCC6 deficiencies in first half of 2021. H1 2021: Initiation of Phase 1/2 clinical trial.

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Drug Discovery World

APRIL 3, 2024

A global health and economic issue Haemorrhage accounts for up to 50% of trauma deaths occurring worldwide within 24 hours of traumatic injury, according to SEMICYUC-Spanish Society of Intensive and Critical Care Medicine and Coronary Units, and up to 80% of intraoperative trauma mortality. Our initial focus will be on the US FDA.

Genetic Disease 52

Genetic Disease Development Research Clinical Trials 52

XTalks

NOVEMBER 10, 2023

Takeda’s application for Adzynma was granted a rare pediatric disease priority review voucher, priority review, fast track and orphan designations. It’s also been granted orphan designation by the European Medicines Agency (EMA) and Japan’s Ministry of Health, Labour and Welfare (MHLW) for the treatment of TTP.

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XTalks

DECEMBER 29, 2022

This includes the first ever CRISPR gene editing therapy from Vertex and CRISPR therapeutics for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Given their precise targeting, gene therapies have largely been focused on oncology indications followed by rare genetic diseases.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

Drug Discovery World

JULY 11, 2022

These challenges exist across the translation cycle from early research to clinical trials, manufacturing, and ultimately reimbursement. The complexities of sample management can significantly extend the timelines for clinical trials. Regulatory requirements. Conclusion. The promise of gene therapy is substantial.

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Drug Discovery World

JUNE 22, 2023

The proposed joint clinical assessment methodology would have rejected nearly 90% of the ATMPs currently authorised in the EU, claims the Alliance for Regenerative Medicine (ARM). ARM reviewed the 18 ATMPs with EU marketing authorisation, including cell and gene therapies for serious, sometimes fatal genetic diseases and blood cancers.

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The Pharma Data

DECEMBER 8, 2020

The assumption is SciNeuro will start with Parkinson’s disease and pain in addition to rare genetic diseases, after scaling up staffing. . A supporting pipeline has medicines to target these key pathways to reverse or eradicate disease and extend the years of healthy life remaining.

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The Pharma Data

AUGUST 15, 2021

Until today, there were no systemic therapies approved to help treat patients diagnosed with certain types of VHL-associated tumors,” said Dr. Eric Jonasch, principal investigator of Study 004 and professor, Department of Genitourinary Medical Oncology, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center.

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The Pharma Data

DECEMBER 3, 2020

Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life. The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease.

Medicine 52

Medicine Genetics Clinical Trials Clinical Trials 52

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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The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. Novartis today announced that the U.S. SVP, Chief Medical Officer, Novartis Gene Therapies. “We

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Clinical Trials Clinical Trials Gene Therapy Trials 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). In the UK, Libmeldy will be administered by a specialist service through the Centre for Genomic Medicine at Saint Mary’s hospital in Manchester. million (approximately $3.8

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The Pharma Data

NOVEMBER 2, 2020

We are grateful to the families who have participated in the clinical trials, the advocacy groups from around the world, and the study investigators, all who have been on this journey with us to deliver a treatment choice and contribute to further understanding achondroplasia medically and scientifically.” and Europe. In the U.S.,

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Drug Discovery World

MARCH 6, 2024

The development of effective new disease-modifying treatments in neurodegenerative and neuropsychiatric disorders has been hindered by their inherent genetic complexity, environmental influences, and clinical variability. This is comparatively easier to accomplish in these diseases because they are relatively monogenic.

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Genome Genomics Clinical Trials Clinical Trials 64

The Pharma Data

JULY 6, 2021

The STASEY study is one of the largest open-label studies primarily assessing the safety and tolerability of a medicine for adults and adolescents with haemophilia A with factor VIII inhibitors. 1 These results confirm the favourable safety profile of Hemlibra, as previously demonstrated in the phase III HAVEN clinical trials.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. We are grateful to all the investigators, staff and patients who participated in the ILLUMINATE clinical studies, and to their families, caregivers and patient advocates. President of R&D at Alnylam.

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XTalks

DECEMBER 22, 2021

RNA therapeutics hold promise as powerful treatments for diseases, including those that are genetic-based. We talked about the emergence of dozens of CRISPR biotechs last year and the growing number of CRISPR-based therapies in clinical trials for conditions ranging from Duchenne muscular dystrophy (DMD) and sickle cell disease (SCD).

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The Pharma Data

JANUARY 25, 2021

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development.

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