Genetic Disease Related Topics 
Pharma Mirror
APRIL 28, 2021
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.
Pharma Phorum
JANUARY 18, 2023
In 2016, scientists behind a study called the Resilience Project analysed genetic data from 589,000+ people and found 13 adults who carried genetic variants that should have resulted in serious – even deadly – childhood disease, but who were apparently healthy.
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BioSpace
OCTOBER 6, 2022
This week, researchers presented findings in how ancestry affects cancer treatment, nanotechnology and genetic diseases. Here's a look at that and more
The Pharma Data
DECEMBER 2, 2022
Lilly Completes Acquisition of Akouos Expanding Efforts to Help People with Genetic Diseases. The acquisition expands Lilly’s efforts in genetic medicines to include Akouos’s portfolio of potential first-in-class adeno-associated viral gene therapies for the treatment of inner ear conditions, including sensorineural hearing loss. senior vice president of genetic medicine at Lilly and co-director of the Institute for Genetic Medicine.
The Pharma Data
AUGUST 31, 2022
Moment, theU.S. Food and Drug Administration approved Xenpozyme( Olipudase alfa) for intravenous infusion in pediatric and adult cases with Acid Sphingomyelinase Deficiency( ASMD), a rare inheritable complaint that causes unseasonable death.
ERT
MARCH 29, 2021
The post Imaging in Drug Development to Treat Rare Genetic Diseases appeared first on ERT. Application Brief - Cardiac Safety - Imaging - Endocrine - Metabolic (Hypophosphatemia). Resources
BioTech 365
JULY 25, 2022
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
Scienmag
OCTOBER 27, 2020
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
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.
BioTech 365
FEBRUARY 14, 2022
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
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. Medicine & Health Bioinformatics Genetics Health Care Systems/Services Medicine/Health MinoritiesLOS ANGELES (June 16, 2021) – UCLA Health will receive a $4.8
BioSpace
JUNE 17, 2021
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
BioPharma Reporter
JULY 21, 2022
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
BioPharma Reporter
JULY 21, 2022
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
BioTech 365
FEBRUARY 10, 2022
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
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. 23, 2020 (GLOBE NEWSWIRE) — UC San Francisco (UCSF) … Continue reading → GlobeNewswire
BioTech 365
JULY 13, 2021
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
Bio Pharma Dive
AUGUST 16, 2021
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
BioTech 365
MAY 31, 2022
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
Scienmag
SEPTEMBER 9, 2020
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
BioTech 365
OCTOBER 21, 2020
Fabric GEM’s accuracy validated across five leading genomics centers of excellence in the U.S. and abroad OAKLAND, Calif.–(BUSINESS
BioTech 365
DECEMBER 3, 2020
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
BioTech 365
NOVEMBER 4, 2020
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
Pharma Phorum
JANUARY 9, 2023
First introduced in 2020, the global Managed Access Programme (gMAP) has provided Zolgensma (onasemnogene abeparvovec) free of charge to nearly 300 children with the genetic disorder across 36 countries where the therapy has not yet received approval or in which no formal access pathway exists.
BioTech 365
FEBRUARY 18, 2021
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
Bio Pharma Dive
MAY 27, 2021
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
Pharma Phorum
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.
XTalks
SEPTEMBER 14, 2022
In this episode, Ayesha discussed the FDA approval of Sanofi’s enzyme replacement therapy Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare genetic lysosomal storage disease, in adults and pediatric patients.
Pharma Phorum
OCTOBER 5, 2022
Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule.
XTalks
SEPTEMBER 12, 2022
ASMD is a rare genetic lysosomal storage disease caused by mutations in the sphingomyelin phophodiesterase-1 ( SMPD1 ) gene that codes for the acid sphingomyelinase (ASM) enzyme. Related: FDA’s CDER Introduces New ARC Program to Accelerate Rare Disease Cures.
Pharma Phorum
AUGUST 1, 2022
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.
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
Scienmag
APRIL 22, 2022
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.
XTalks
NOVEMBER 10, 2020
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.
Pharma Phorum
SEPTEMBER 14, 2022
SparingVision has raised €75 million in second-round funding that will be used to fund clinical trials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related macular degeneration (AMD).
Scienmag
JULY 1, 2022
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
Pharma Phorum
OCTOBER 4, 2020
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.
Scienmag
JULY 1, 2022
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.
BioSpace
OCTOBER 30, 2022
Platform technology company Replay, based in San Diego and London, has launched Eudora, a gene therapy company targeting genetic diseases of the retina
Pharma Phorum
DECEMBER 8, 2021
News R&D Acadia Pharmaceuticals genetic diseases Neuren Pharmaceuticals rare disease Rett Syndrome trofinetide
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
BioSpace
MAY 24, 2021
Gene therapy is still very much cutting-edge with the potential to cure incurable genetic diseases. Here’s some recent news in the space
BioSpace
JUNE 16, 2022
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
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