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.

Genetic Disease 97

Genetic Disease Genetics Research Genome 97

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.

Genetic Disease 40

Genetic Disease Genetics Development 40

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

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

Drug Discovery World

JUNE 22, 2023

This is the latest episode of the free DDW narrated podcast, “Challenges and tactics for transformative drug development” It covers two articles written for Volume 23, Issue 2 – Spring 2022 of DDW. They are called “ Balancing speed and quality in CLD for faster drug development ”, and “ Sitting down with BridgeBio Pharma ”.

Development 52

Development Genetic Disease Drugs Genetics 52

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 168

Genetics Trials Clinical Trials Clinical Trials 168

Drug Discovery World

MAY 3, 2024

Dr Incerti has more than three decades of experience in the biopharmaceutical industry and brings an extensive track record in global drug development, including from his time at Sanofi Genzyme where he played a leading role in pioneering therapies for rare and genetic diseases.

Development 52

Development Genetic Disease Genetics Drugs 52

Medical Xpress

MARCH 27, 2023

Food and Drug Administration (FDA) applications for Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), a new article can help developers of gene therapies for rare genetic diseases. The article is published in the journal Human Gene Therapy.

Gene Therapy 60

Gene Therapy Gene Development Genetic Disease 60

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

Drug Discovery World

JUNE 22, 2023

This is the latest episode of the free DDW narrated podcast, “Challenges and tactics for transformative drug development” It covers two articles written for Volume 23, Issue 2 – Spring 2022 of DDW. They are called “ Balancing speed and quality in CLD for faster drug development ”, and “ Sitting down with BridgeBio Pharma ”.

Development 52

Development Drugs Genetic Disease Genetics 52

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

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

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

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

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

Genetic Disease 122

Genetic Disease Genetics Development 122

Drug Discovery World

JUNE 19, 2023

Cystic fibrosis is a progressive genetic disease that affects the lungs, pancreas and other organs. AdBio Partners’ mission is to support founding scientists in the development of new therapeutic approaches,” said Alain Huriez, Managing Partner of AdBio Partners. “We

Development 52

Development Life Science In-Vivo In-Vitro 52

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.”

In-Vitro 246

In-Vitro Genetic Disease Medicine Genetics 246

Drug Discovery World

DECEMBER 5, 2022

A new partnership between industry and three rare disease foundations has formed with the goal of accelerating the development of precision drugs to treat rare neurodevelopmental disorders. . Understanding rare diseases remains a significant challenge.

Development 52

Development Genome Genomics Drugs 52

Drug Discovery World

NOVEMBER 7, 2022

Genomic healthcare data is critical to identify disease risk, ancestry, traits and response to medicines and aids in the development of new targeted therapies – precision medicines. DDW’s Megan Thomas observes developments of accessibility in this sector and the potential impact. . The origins . The current landscape .

Genome 98

Genome Genomics Development Genetics 98

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.

Gene Editing 126

Gene Editing Gene Genetic Disease Gene Therapy 126

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.

Genetic Disease 76

Genetic Disease RNA Genetics Development 76

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.

FDA Approval 130

FDA Approval Gene Therapy Genetic Disease Gene 130

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 82

Genetic Disease Genetics Development 82

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.’

Development 98

Development Gene Therapy Clinical Trials Clinical Trials 98

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.

Genetic Disease 130

Genetic Disease Genetics Sales Development 130

Drug Discovery World

JANUARY 24, 2023

DMD and FA are rare, degenerative, genetic diseases that affect movement and eventually lead to paralysis. This is particularly important for rare diseases where it can be hard to identify suitable patients. The post AI and wearable tech could speed up drug development appeared first on Drug Discovery World (DDW).

Development 52

Development Clinical Trials Clinical Trials Drugs 52

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

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

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.

RNA 147

RNA Vaccination Vaccine In-Vivo 147

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 (..)

RNA 77

RNA Genetic Disease Scientist Genetics 77

Drug Discovery World

DECEMBER 19, 2023

Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication. MPS I is a rare, genetic disease that affects the body’s ability to produce the enzyme alpha-L-iduronidase (IDUA).

Engineer 113

Engineer Trials Genetic Disease Gene Therapy 113

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.

Genetic Disease 98

Genetic Disease Life Science Genetics Branding 98

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

Scienmag

MAY 26, 2022

A line of powerful tools has been developed from the popular CRISPR-Cas9 to cure genetic diseases. ITHACA, N.Y. — CRISPR has ushered in the era of genomic medicine.

Gene Editing 63

Gene Editing Genetic Disease Gene Genome 63

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

Drug Discovery World

DECEMBER 5, 2022

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases. . Many distinct types of parvoviruses infect mammals – including humans – without causing disease.

Gene Therapy 52

Gene Therapy Gene Genetic Disease Genetics 52

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.

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147