pharmaphorum

JANUARY 18, 2023

First, start co-development with participants early on in the process. And if participants have opted in to be made aware of their risks of genetic diseases, then communication has to be carefully considered. At the same time, the fraction of clinical trials that include genetic markers is increasing. About the author.

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Genetic Disease Genetics Research Genome 97

Medical Xpress

NOVEMBER 10, 2022

Using a protocol developed at UC San Francisco, physicians have successfully treated a fetus with a devastating genetic disorder for the first time, and the child is now thriving as a toddler, a case study in the New England Journal of Medicine reports.

Genetics 98

Genetics Genetic Disease Medicine Development 98

Scienmag

MARCH 30, 2021

Credit: Gretel Nicholson, EXRC Scientists have discovered a new genetic disease, which causes some children’s brains to develop abnormally, resulting in delayed intellectual development and often early onset cataracts.

Genetic Disease 52

Genetic Disease Genetics Scientist Development 52

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. As a result, the diversity of CFTR mutations presents significant challenges in developing therapies that can benefit all CF patients.

Gene Therapy 105

Gene Therapy Protein Genetic Disease Gene 105

Worldwide Clinical Trials

JANUARY 24, 2024

Much of what we discuss I covered in my presentation, “Leveraging Genetics to Support Rare Disease Clinical Trials,” at last year’s World Orphan Drug Congress (WODC) EU. As a board-eligible genetic counselor, I believe that genetic counselors should also be involved since they are integral to the patient’s diagnostic odyssey.

Genetics 189

Genetics Trials Clinical Trials Clinical Trials 189

Pharmaceutical Technology

MAY 4, 2023

Vertex global medicines development and medical affairs executive vice-president and chief medical officer Carmen Bozic stated: “Treating the underlying cause of cystic fibrosis as early as possible is important, and this approval, the first for a CFTR modulator in this age group, means families will now have a medicine for eligible infants.”

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In-Vitro Genetic Disease Medicine Genetics 246

Pharma Times

AUGUST 16, 2021

VHL is a rare genetic disease which puts patients at risk of developing benign blood vessel tumours and several cancers

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Genetic Disease Genetics Development 123

Pharmaceutical Technology

MARCH 1, 2023

Tevard Biosciences and Vertex Pharmaceuticals have announced a four-year global research partnership to develop new tRNA-based therapies to treat Duchenne muscular dystrophy (DMD) patients. The deal offers access to the platforms of Tevard to discover and develop tRNA-based therapies.

Development 130

Development Gene Therapy Genetics Genetic Disease 130

XTalks

DECEMBER 4, 2024

CAMP4’s RNA-based therapies focus on genetic diseases like urea cycle disorders, while Rapport’s small molecules aim to address epilepsy, pain and bipolar disorder. Jupiter joins a wave of biotech IPOs this year, stepping into a competitive space while broadening treatment options for CNS patients.

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Bioavailability Genetic Disease Marketing RNA 111

Pharma Mirror

APRIL 21, 2024

With the rapid development of biotechnology and molecular medicine, the introduction of mRNA as a vaccine or therapeutic agent enables the production of almost any desired functional protein/peptide within the human body.

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In-Vitro Drugs Genetic Disease RNA 130

BioTech 365

DECEMBER 23, 2020

UCSF and BridgeBio Pharma Collaborate to Accelerate the Development of Therapies for Genetic Diseases UCSF and BridgeBio Pharma Collaborate to Accelerate the Development of Therapies for Genetic Diseases SAN FRANCISCO, Dec.

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Genetic Disease Genetics Development 40

Scienmag

JULY 1, 2022

Curing debilitating genetic diseases is one of the great challenges of modern medicine. During the past decade, development of CRISPR technologies and advancements in genetics research brought new hope for patients and their families, although the safety of these new methods is still of significant concern.

Genetics 97

Genetics Genetic Disease Medicine Development 97

Medical Xpress

MARCH 15, 2023

Scientists have developed a test that could greatly improve quality of life for infants with homocystinuria (HCU), a congenital disease that—if not treated early—causes serious complications. Research demonstrating the efficacy of this test was published today in Clinical Chemistry.

Genetic Disease 75

Genetic Disease Genetics Scientist Development 75

STAT News

FEBRUARY 22, 2023

The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions. Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines.

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Gene Editing Gene Genetic Disease Research 126

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. “They called him ‘The Boy.’

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Development Gene Therapy Clinical Trials Clinical Trials 98

Scienmag

JUNE 16, 2021

million grant from The National Institutes of Health to develop methods that will improve genetic risk estimates – polygenic risk scores – for specific diseases in people from diverse populations and mixed ancestries. LOS ANGELES (June 16, 2021) – UCLA Health will receive a $4.8

Genetics 70

Genetics Genetic Disease Development Bioinformatics 70

Pharmaceutical Technology

AUGUST 17, 2022

Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. These programmes will include therapies and vaccines in infectious disease and oncology areas.

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RNA Vaccination Vaccine In-Vivo 147

STAT News

APRIL 19, 2023

But after Cheung herself developed a genetic condition so rare it doesn’t have an official name, causing her to start losing her vision, HHMI decided to stop funding her.

Scientist 136

Scientist Genetic Disease Genetics Research 136

World of DTC Marketing

JANUARY 24, 2022

Venture capitalists have poured $42 billion into drug development over the past three years. Most small biotech companies rely on venture capitalist funding to develop new drugs but is that a good way to go? billion, which was much lower than previous studies, which have placed the average cost of drug development as $2.8

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Life Science Drugs Development Genetic Disease 225

Scienmag

MAY 2, 2022

WINSTON-SALEM, NC – May 2, 2022 — Wake Forest Institute for Regenerative Medicine (WFIRM) scientists working on CRISPR/Cas9-mediated gene editing technology have developed a method to increase efficiency of editing while minimizing DNA deletion sizes, a key step toward developing gene editing therapies to treat genetic diseases.

Gene Editing 58

Gene Editing DNA Gene Development 58

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. While most gene therapy clinical studies are ongoing, a number of products are in advanced clinical development, and several are approved by FDA. PK Planning for Gene Therapy Development Programs. Exposure-Response Considerations.

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Gene Therapy Gene Gene Editing Genetics 238

Pharmaceutical Technology

MAY 22, 2023

It is claimed to be both the first re-dosable gene therapy and the first and only FDA-approved treatment for both recessive and dominant types of DEB, a rare and serious genetic disease affecting the skin and mucosal tissues.

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FDA Approval Gene Therapy Genetic Disease Gene 130

Pharmaceutical Technology

FEBRUARY 28, 2023

Under the deal, Rhythm will acquire Xinvento’s 100% fully diluted equity for a $5m upfront payment and pay up to an additional $6m in preclinical development milestones. The acquisition of Xinvento is a solid strategic fit with the rare endocrinology focus of Rhythm and is expected to offer new development opportunities.

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Genetic Disease Genetics Sales Development 130

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Moderna will also assume further development, manufacturing, and commercialisation responsibilities on exercising a target option.

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Gene Editing Gene In-Vivo Gene Therapy 147

STAT News

SEPTEMBER 12, 2022

The company is developing treatments for diseases caused by broken mitochondria — the main energy source for human cells. Breakdowns in these microscopic power plants are linked to dozens of diseases, including Alzheimer’s, cancer, metabolic conditions, and several rare genetic diseases.

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Scienmag

SEPTEMBER 29, 2021

JUPITER, FL – The American Chemical Society announced that its prestigious 2022 Nobel Laureate Signature Award for Graduate Education in Chemistry honors a student from the Skaggs Graduate School of Chemical and Biological Sciences at Scripps Research and her faculty advisor, for their pioneering work developing RNA-targeting medications for genetic (..)

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RNA Genetic Disease Scientist Genetics 77

BioPharma Reporter

OCTOBER 17, 2024

Wave Life Sciences reports that it has successfully carried out RNA editing in two patients with alpha-1 antitrypsin deficiency, the first time this kind of clinical RNA editing has been completed in humans according to the company.

RNA 52

RNA Genetic Disease Genetics Life Science 52

BioSpace

OCTOBER 13, 2021

Rectify emerged with a goal to develop disease-modifying precision therapies that will restore ABC transporter function in order to address the underlying cause of serious genetic disease.

Genetic Disease 69

Genetic Disease Genetics Development 69

XTalks

APRIL 15, 2021

Experts from Servier and Genuity Science recently spoke on a webinar about using genomics data to drive drug development in heart failure and identify new targets for novel therapeutics. The reverse could also be true whereby the development of coronary artery disease may impact heart rate. HFrEF vs. HFpEF.

Development 98

Development Drugs Genome Genomics 98

pharmaphorum

OCTOBER 25, 2022

The Japanese firm has agreed to make a $50 million investment in Dallas-based Taysha in exchange for a 15% stake in the company, plus exclusive options to license two clinical-stage, single-gene therapies for rare genetic diseases. ” The post Astellas makes another gene therapy play, takes stake in Taysha appeared first on. .

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Gene Therapy Gene Genetic Disease Genetics 103

STAT News

FEBRUARY 3, 2023

The Boston drugmaker’s medicines for the genetic disease — which damages the lungs and other organs — have transformed the lives of patients, helping them abandon other time-consuming treatments, shrug off what would have been dangerous respiratory infections, and just live breathing easier.

Genetic Disease 98

Genetic Disease Genetics Medicine Drugs 98

BioSpace

JUNE 20, 2022

Therorna brought in $42 million to advance its circular RNA platform, while Carbon Biosciences banked $38 million to develop therapies for genetic diseases, beginning with cystic fibrosis.

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Genetic Disease RNA Genetics Development 64

Pharmaceutical Technology

APRIL 6, 2023

GRIA disorder is a family of rare genetic diseases. Through the study, Dr Coombs expects to characterise the genetic variants underlying various behavioural and cognitive symptoms in children suffering from GRIA disorder. He will further employ this diagnostic to select drugs for restoring patients’ normal functioning.

Genetic Disease 130

Genetic Disease Genetics Gene Drugs 130

BioPharma Reporter

FEBRUARY 23, 2023

The messenger RNA (mRNA) specialist Moderna has teamed up with ElevateBio-owned Life Edit Therapeutics to develop gene editing therapies that are delivered into patients in vivo.

Gene Editing 52

Gene Editing Genetic Disease Gene In-Vivo 52

Pharmaceutical Technology

JANUARY 12, 2023

Solid Biosciences has entered a strategic research collaboration with Phlox Therapeutics to expedite the development of new therapies for rare cardiac diseases. Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM).

RNA 130

RNA Gene Therapy Genetics Genetic Disease 130

Scienmag

FEBRUARY 17, 2021

A leading cause of SCD in young athletes is arrhythmogenic cardiomyopathy (ACM), a genetic disease in which healthy heart muscle is replaced over time […]. Credit: Mark Bauer/FSU College of Medicine Nearly a half-million people a year die from sudden cardiac death (SCD) in the U.S.

Medicine 55

Medicine Genetic Disease Development Genetics 55

Scienmag

APRIL 6, 2021

Researchers developed a new animal model to study a rare genetic disease that can lead to blindness at the age of 40-50.

Genetic Disease 52

Genetic Disease Genetics Development Research 52