New Resource for the Galactosemia Community Launches to Empower Families as They Navigate Lifelong Genetic Disease

BioTech 365

New Resource for the Galactosemia Community Launches to Empower Families as They Navigate Lifelong Genetic Disease New Resource for the Galactosemia Community Launches to Empower Families as They Navigate Lifelong Genetic Disease – The handbook presents information for each life … Continue reading → Business Wire

Imaging in Drug Development to Treat Rare Genetic Diseases

ERT

The post Imaging in Drug Development to Treat Rare Genetic Diseases appeared first on ERT. Application Brief - Cardiac Safety - Imaging - Endocrine - Metabolic (Hypophosphatemia). Resources

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#news #biotech Improving RNA editing for genetic diseases by recruiting the body’s natural protein

BioTech 365

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Improving RNA editing for genetic diseases by recruiting the body’s natural protein.Improving RNA editing for genetic diseases by recruiting the body’s natural protein aliu Mon, 02/14/2022 – … Continue reading → #news

#news #biotech Simplifying RNA editing for treating genetic diseases

BioTech 365

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Simplifying RNA editing for treating genetic diseases.New research led by bioengineers at the University of California San Diego could make it much simpler to repair disease-causing mutations … Continue reading → #news

Rare genetic disease caused by mutations in protein that controls RNA metabolism

Scienmag

Medicine & Health Biology Cell Biology Developmental/Reproductive Biology Genes Genetics Medicine/Health Molecular Biology Musculature Pediatrics

New approach to diagnosing genetic diseases using RNA sequencing increases yield

Scienmag

In the world of rare genetic diseases, exome and genome sequencing are two powerful tools used to make a diagnosis. A recent addition to the toolkit, RNA sequencing, has been demonstrated to help researchers narrow down disease candidate variants identified first on exome and genome sequencing. Technology and Engineering Bioinformatics Biotechnology Diagnostics Genetics Medicine/Health

Scientists discover new genetic disease that delays brain development in children

Scienmag

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.

US regRNA player gets $100m in Series A funding to treat genetic diseases at their core

BioPharma Reporter

US biotech, CAMP4 Therapeutics, has secured US$100m in a Series B financing round that it says will be used to accelerate expansion of its regulatory RNA (regRNA) platform. Markets & Regulations

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UCSF and BridgeBio Pharma Collaborate to Accelerate the Development of Therapies for Genetic Diseases

BioTech 365

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. 23, 2020 (GLOBE NEWSWIRE) — UC San Francisco (UCSF) … Continue reading → GlobeNewswire

Insights on the Umbilical Cord Blood Banking Global Market to 2027 – Rising Applications of Cord Blood and Stem Cells in Genetic Disease Treatment is Driving Growth – ResearchAndMarkets.com

BioTech 365

Insights on the Umbilical Cord Blood Banking Global Market to 2027 – Rising Applications of Cord Blood and Stem Cells in Genetic Disease Treatment is Driving Growth – ResearchAndMarkets.com Insights on the Umbilical Cord Blood Banking Global Market to 2027 … Continue reading → Business Wire

AavantiBio and Catalent Announce Partnership to Support Development and Manufacturing of Gene Therapies for Rare Genetic Diseases

Pharma Mirror

The post AavantiBio and Catalent Announce Partnership to Support Development and Manufacturing of Gene Therapies for Rare Genetic Diseases appeared first on Pharma Mirror Magazine.

#news #biotech Penn team uses CRISPR to edit out genetic disease before and after birth in mice

BioTech 365

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Penn team uses CRISPR to edit out genetic disease before and after birth in mice.Penn team uses CRISPR to edit out genetic disease before and after birth … Continue reading → #news

UCLA receives $4.8M to improve genetic disease risk estimates in diverse populations

Scienmag

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. Medicine & Health Bioinformatics Genetics Health Care Systems/Services Medicine/Health MinoritiesLOS ANGELES (June 16, 2021) – UCLA Health will receive a $4.8

Orphazyme’s Stocks Tumble After FDA Rejects Genetic Disease Drug

BioSpace

The FDA said that the drug, which has been studied and submitted for NPC indication, needs further data to support its use in this rare, progressive genetic disorder

Fabric Genomics Launches GEM Algorithm to Accelerate Genetic Disease Diagnosis, Provide Comprehensive Clinical-Decision Support

BioTech 365

Fabric GEM’s accuracy validated across five leading genomics centers of excellence in the U.S. and abroad OAKLAND, Calif.–(BUSINESS

RNAi Technology Market- by Application (Cancer, Infectious Diseases, Ophthalmic Diseases, Genetic Diseases, and Other Diseases) and by Region – Global Forecasts 2020-2024 | Technavio

BioTech 365

RNAi Technology Market- by Application (Cancer, Infectious Diseases, Ophthalmic Diseases, Genetic Diseases, and Other Diseases) and by Region – Global Forecasts 2020-2024 | Technavio RNAi Technology Market- by Application (Cancer, Infectious Diseases, Ophthalmic Diseases, Genetic Diseases, and Other Diseases) and … Continue reading → Business Wire

Agios to Present Broad Set of Clinical and Translational Data for Oncology and Rare Genetic Disease Programs at the Virtual 2020 ASH Annual Meeting

BioTech 365

Agios to Present Broad Set of Clinical and Translational Data for Oncology and Rare Genetic Disease Programs at the Virtual 2020 ASH Annual Meeting Agios to Present Broad Set of Clinical and Translational Data for Oncology and Rare Genetic Disease … Continue reading → GlobeNewswire

Merck wins approval for cancer drug acquired in 2019 biotech buyout

Bio Pharma Dive

The FDA cleared Welireg, which Merck picked up in its $1 billion takeover of Peloton Therapeutics, to treat certain tumors associated with a rare genetic disease

Illumina and the University Hospital of Tübingen Evaluate Potential of Whole Genome Sequencing to Improve Diagnosis of Full Range of Genetic Diseases

BioTech 365

Illumina and the University Hospital of Tübingen Evaluate Potential of Whole Genome Sequencing to Improve Diagnosis of Full Range of Genetic Diseases Illumina and the University Hospital of Tübingen Evaluate Potential of Whole Genome Sequencing to Improve Diagnosis of Full … Continue reading → Business Wire

Cancer drug can rebalance kidney function in a devastating genetic disease

Scienmag

Researchers at the University of Cambridge and the University of Zurich have discovered that a drug newly approved for cancer improves kidney dysfunction in a mouse model of Dent disease 2 and Lowe syndrome The study is published today in Kidney International and offers hope for the first disease-modifying treatment. Biology Biochemistry Cell Biology Developmental/Reproductive Biology Genetics Medicine/Health Molecular Biology Physiology Sex-Linked Conditions Urogenital System

UniQure moves Huntington's gene therapy to next phase of key trial

Bio Pharma Dive

Safety monitors cleared the company to test a higher dose after reviewing data from 10 patients, providing some hopeful news after recent setbacks for experimental drugs targeting the genetic disease

Sarepta says early filing for DMD gene therapy is back on

pharmaphorum

Sarepta’s gene therapy – like rivals from Pfizer and Solid Biosciences – codes for a shortened form of the dystrophin protein that is deficient in patients with the X-linked muscle-wasting disease, which occurs primarily in males.

Evidence suggests cancer is not as purely genetic as once thought

Scienmag

While cancer is a genetic disease, the genetic component is just one piece of the puzzle — and researchers need to consider environmental and metabolic factors as well, according to a research review by a leading expert at the University of Alberta.

“Soft” CRISPR may offer a new fix for genetic defects

Scienmag

Curing debilitating genetic diseases is one of the great challenges of modern medicine. Credit: Guichard/Bier Curing debilitating genetic diseases is one of […]. Technology and Engineering CRISPR defects fix genetic offer Soft

New screening technique could accelerate and improve mRNA therapies

Scienmag

Therapeutics based on messenger RNA, or mRNA, can potentially treat a wide range of maladies, including cancer, genetic diseases, and as the world has learned in recent years, deadly viruses.

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Welireg approved in US for rare VHL-associated cancers

Pharma Times

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

Rare Disease Diagnosis: Why Tackling the Genomic Analysis Bottleneck is Key to Advancing Precision Medicine

XTalks

Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare genetic diseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with genetic diseases.

EMA starts rapid review of Bluebird’s gene therapy for rare disease CALD

Pharma Phorum

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review. The post EMA starts rapid review of Bluebird’s gene therapy for rare disease CALD appeared first on.

Therorna, Carbon Reel in Combined $80M in Series A Financing

BioSpace

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

RNA 63

"Holy Grail" Transplantation Technique Offers Hope to Patients with Rare Kidney Disease

BioSpace

In a study conducted by researchers at Stanford University, three pediatric patients with a rare genetic disease that often results in kidney failure received kidney transplants

Acadia closer to bringing first Rett syndrome drug to market

Pharma Phorum

News R&D Acadia Pharmaceuticals genetic diseases Neuren Pharmaceuticals rare disease Rett Syndrome trofinetide

Gene Therapy Continues Edging into the Mainstream

BioSpace

Gene therapy is still very much cutting-edge with the potential to cure incurable genetic diseases. Here’s some recent news in the space

Rectify Nets $100 Million to Help the Body Relearn its ABCs

BioSpace

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

Catabasis craters after giving up on muscular dystrophy lead

Pharma Phorum

The PolarisDMD study failed across the board, leaving Catabasis with no choice but to abandon the drug, including an ongoing open-label extension study from an earlier failed trial in the muscle-wasting disease. News R&D Catabasis Duchenne muscular dystrophy edasalonexent genetic disease

Study in mice shows genes may be altered through drug repurposing

Scienmag

Researchers at the University of Illinois Chicago have published a study showing a promising approach to using drug repurposing to treat genetic diseases. Biology Genes Genetics Medicine/Health Ophthalmology

Population bottlenecks that reduced genetic diversity were common throughout human history

Scienmag

Human populations have waxed and waned over the millennia, with some cultures exploding and migrating to new areas or new continents, others dropping to such low numbers that their genetic diversity plummeted. Biology bottlenecks common diversity genetic History human population reduced

Bluebird bio's Oncology Business Leaves the Nest to Form New Company

BioSpace

Bluebird bio announced plans to split its genetic disease and oncology businesses. Bluebird bio will stay focused on severe genetic disease and spin out its oncology business into a new company

The answer to keeping moose populations healthy? Wolves

Scienmag

Predators may keep prey populations healthy by acting as a selective force against genetic diseases. A new study found that wolves select adult moose based on age and osteoarthritis, a chronic disease that can be influenced by genetics.

Regular cycling helps patients with ‘accelerated aging’ disease

Scienmag

Hamilton, ON (April 11, 2022) – A study led by McMaster University researchers has found that regular cycling can greatly improve mobility in patients with myotonic dystrophy (MD), a genetic disease that causes muscle degeneration. Medicine & Health accelerated Aging cycling Disease helps Patients regular

New perspective to understand and treat a rare calcification disease

Scienmag

Researchers developed a new animal model to study a rare genetic disease that can lead to blindness at the age of 40-50. Medicine & Health Biology Genes Genetics Metabolism/Metabolic Diseases

#news #biotech Stem cells reveal underpinnings of rare immune disease

BioTech 365

new stem cell study by KAUST researchers helps to explain a rare genetic disease called Wiskott-Aldrich syndrome (WAS), yielding … Continue reading → #newsBiotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Stem cells reveal underpinnings of rare immune disease.A

New insights into kidney disease with tropical frog models

Scienmag

An international team led by Soeren Lienkamp, professor at the Institute of Anatomy at UZH, has now exploited this similarity by using a tiny tropical frog called Xenopus tropicalis to model human genetic diseases. Technology and Engineering Disease frog insights kidney models Tropica

CNIO researchers shed light on the maturation of spliceosome, a cellular process involved in certain types of cancer

Scienmag

The assembly process is tightly controlled, and any failure can result in genetic diseases, including some […]. Alternative Splicing is an extraordinarily complex process that requires the coordinated action of multiple proteins, each specialised in very specific functions.