Drug Discovery World

MARCH 19, 2024

Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system. This is the first FDA-approved treatment option for children who have this rare genetic disease,” said Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research (CBER). “We

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Drug Discovery World

DECEMBER 5, 2022

AI-driven genomics company Genomenon will partner with COMBINEDBrain, SynGAP Research Fund, and SLC-6A1 Connect and their pharma partners to deliver genomic data on their diseases of focus. . Understanding rare diseases remains a significant challenge.

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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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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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Drug Discovery World

NOVEMBER 7, 2022

A new study led by Medical Research Council-funded researchers from UCL has found that tailoring the analysis of whole genome sequencing to individual patients could double the diagnostic rates of rare diseases. . Dr Pitceathly continued: “In this study, every new genetic diagnosis had a direct impact on patient care.

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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

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. However, researchers are determined to overcome this hurdle.

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XTalks

FEBRUARY 28, 2022

In the official Rare Disease Day 2022 video above, patients from all around the world are heard sharing their stories in their native languages about living with rare diseases like Duchenne muscular dystrophy, cystic fibrosis and Gaucher’s disease among others. How is a Rare Disease Defined?

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Drug Discovery World

JANUARY 30, 2024

Lentivirus strategies are one of the most widely used tools in biomedical research and clinical testing, says Stella Vnook, CEO, Likarda. “Dr Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said.

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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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Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. Whilst research into mRNA has been ongoing for decade, interest has sparked in recent years and research has begun to expand into areas of vaccinology, infectious diseases and other indications.

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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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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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Drug Discovery World

FEBRUARY 22, 2024

Recent advances in biomarkers, research into the central nervous system and breakthroughs in human iPSC cell models, have bolstered neuroscience drug discovery research. ARUK 3 adds that while the UK government increased funding in dementia research from £28.2m years it takes to complete an entire cancer clinical trial.

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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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Camargo

JULY 27, 2021

The technology of gene therapy is possible due to extensive DNA research and our resulting understanding of many diseases on the genetic level, and it encompasses several mechanisms such as introducing new genes (gene addition) and inactivating or replacing mutated genes (gene editing).

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Drug Discovery World

APRIL 3, 2024

A public-private consortium formed by the biotech Hemostatics, based at the Barcelona Science Park, the Clínica Universidad de Navarra (CUN) and the Institute of Chemical Research of Catalonia (ICIQ), has received €2.5 Our initial focus will be on the US FDA.

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pharmaphorum

OCTOBER 16, 2020

By definition, however, trial master files represent a much richer and more detailed source of data on a drug and how it performs. For instance, provided a drug has not failed a trial on safety, the side effects it caused in one population could constitute on-target effects in another.

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The Pharma Data

DECEMBER 3, 2020

Clinical Trials. December 5, 2:00 PM – 3:30 PM; Platform A: Translational Research / Genetics. Translational Research / 2B. New preclinical data provide additional evidence of the potential for TANGO antisense oligonucleotides (ASOs) to provide a gene-specific, disease-modifying treatment for Dravet syndrome.

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Delveinsight

FEBRUARY 11, 2021

Verily is partnering with Johnson & Johnson’s Janssen division to witness the body’s earliest immune responses to a coronavirus infection, with people participating in the research from within their homes. . However, they have only shrunk tumors in about half of patients who took them, along with chemotherapy, in the clinical trials.

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Drug Discovery World

NOVEMBER 14, 2023

“The FDA remains deeply committed in our efforts to help facilitate the development and approval of safe and effective therapies for patients with rare diseases,” said Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research. Without treatment, cTTP is ultimately fatal.

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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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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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The Pharma Data

NOVEMBER 25, 2020

Ongoing research was put on the backburner and scientists began to focus on understanding the virus and assessing what medications could be used against it. The mRNA vaccine candidate developed by Pfizer and Germany-based BioNTech demonstrated 95% efficacy in clinical trials.

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Delveinsight

JANUARY 12, 2021

The duo will jointly develop the therapy, conducting clinical trials globally. Bluebird bio recently announced its plans to split its genetic disease and oncology businesses. The company has decided to prioritize its severe genetic disease unit and form its oncology business into a new company. and Europe.

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Drug Discovery World

JULY 11, 2022

Complexities arise while trying to establish a standard, cost-efficient, and expert process for research and manufacturing. These challenges exist across the translation cycle from early research to clinical trials, manufacturing, and ultimately reimbursement. 1: TBRC Business Research PVTLTD. Making CGT work.

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XTalks

MARCH 3, 2021

BridgeBio is dedicated to developing therapies for genetic diseases with unmet needs. Today’s action marks the first FDA approval for a therapy to treat this devastating disease,” said Hylton V. Nulibry Trial Results. The approval was granted to BridgeBio Pharma, Inc.

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The Pharma Data

JULY 6, 2021

1 These results confirm the favourable safety profile of Hemlibra, as previously demonstrated in the phase III HAVEN clinical trials. Spark Therapeutics will share updated data from the ongoing phase I/II clinical trial of SPK-8011, an investigational AAV-based gene therapy developed for the treatment of haemophilia A.

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XTalks

DECEMBER 29, 2022

Given their precise targeting, gene therapies have largely been focused on oncology indications followed by rare genetic diseases. As such, most gene therapies in late-stage clinical trials are in oncology or rare disease indications. 3. Manufacturing & Supply Chain: Addressing Drug Shortages.

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The Pharma Data

OCTOBER 14, 2020

As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.

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Delveinsight

AUGUST 17, 2021

The efficacy and safety results were produced from a broad research study, multicenter, open-label, randomized phase 3 trial data from the Keynote-581 trial of the first-line therapy indicated to the patients that resulted in an extension of their survival. The rarity of disease – 1.36

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The Pharma Data

MARCH 16, 2021

“Von Hippel-Lindau disease is a rare genetic condition for which there is no systemic treatment option available and is associated with a high risk of cancer development in multiple organs. Research into VHL biology that led to the discovery of HIF-2? About Von Hippel-Lindau Disease and Renal Cell Carcinoma.

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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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The Pharma Data

JANUARY 17, 2021

Safety is critical in all clinical trials, but with ultra-rare diseases, even greater care is taken. We’ve created safety databases that begin with non-human primates and extend to controlled clinical trials.” Genetic diseases are vastly more common and more complex than we used to think.”.

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pharmaphorum

APRIL 20, 2022

As is typical, the research built on work going back years prior, specifically to 1987 when the first CRISPR mechanism was identified in E. coli, and their research in turn formed the foundation for the CRISPR-based therapies being explored to create new treatment options. Why cancer? The pipeline.

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Drug Discovery World

MAY 11, 2023

In The Evolving Role of the CRO, panellists showed that research partner organisations and their service provision to R&D companies is essential. 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

OCTOBER 27, 2020

AavantiBio’s strategic partnership with University of Florida’s Powell Gene Therapy Center provide their foundational research in rare genetic disorders. The company’s lead program is aimed at Friedrich’s Ataxia, a rare inherited genetic disease that causes cardiac and central nervous system dysfunction.

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