Pharmaceutical Technology

MAY 4, 2023

The medicine is currently available for use in more than 30 countries. CF is a rare, life-shortening genetic disease that is estimated to impact over 88,000 people across the globe. The progressive multi-organ disease affects the patients’ lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract.

In-Vitro 246

In-Vitro Genetic Disease Medicine Genetics 246

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.

Genetic Disease 69

Genetic Disease RNA Genetics Genomics 69

Pharmaceutical Technology

JUNE 8, 2023

Biotechnology company Hopewell Therapeutics has raised $25m in seed financing to accelerate the development of next-generation lipid nanoparticles for targeted delivery of genomic medicines. Hopewell Therapeutics is engaged in discovering, synthesising and developing advanced ttLNPs to provide next-generation genomic medicines.

Genome 130

Genome Genomics Medicine Development 130

XTalks

DECEMBER 4, 2024

In the Asia-Pacific market, Jupiter has negotiated partnerships with companies such as Sichuan Kelun and Tianjin Pharmaceuticals, focusing on Jotrol’s integration into traditional Chinese medicine frameworks. International patents valid until 2036 further strengthen its global positioning.

Bioavailability 111

Bioavailability Genetic Disease Marketing RNA 111

Scienmag

MAY 7, 2021

Credit: UPMC PITTSBURGH, May 7, 2021 – In a paper published today in Nature Communications, an international group of collaborators led by researchers at UPMC Children’s Hospital of Pittsburgh have identified a genetic cause of a rare neurological disorder marked by developmental delay and loss of coordination, or ataxia.

Genetics 95

Genetics Genetic Disease RNA Protein 95

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.

Genetics 95

Genetics Genetic Disease Research Medicine 95

Pharmaceutical Technology

JUNE 16, 2023

The Abu Dhabi Department of Health (DoH) in the UAE has made a declaration of collaboration with Mass General Brigham’s (MGB) International Center for Genetic Disease (iCGD) to advance life sciences.

Life Science 130

Life Science Genetic Disease Genome Genomics 130

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.

In-Vitro 130

In-Vitro Drugs Genetic Disease RNA 130

pharmaphorum

APRIL 30, 2024

Prime Medicine has been given the go-ahead by the FDA for the first human trial of its prime editing technology, which promises to deliver one-shot therapies for a range of severe genetic diseases.

Genetic Disease 105

Genetic Disease Trials Genetics Medicine 105

Pharmaceutical Technology

MAY 24, 2023

Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases.

Gene Therapy 242

Gene Therapy Gene Development Manufacturing 242

STAT News

MARCH 1, 2023

Tessera Therapeutics chief scientist Michael Holmes had just finished presenting a much-anticipated peek at a technology the company had previously said could “ revolutionize genetic medicine ” and “cure nearly any genetic disease.”

Gene Editing 119

Gene Editing Genome Genomics Genetic Disease 119

Pharmaceutical Technology

APRIL 26, 2023

MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The new deal excludes oncology and other therapeutic areas outside the scope of genetic disease.

Development 130

Development Genetic Disease Genetics Gene Therapy 130

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

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

Medical Xpress

MARCH 6, 2023

This has been shown in a recent study by Martin Luther University Halle-Wittenberg (MLU), University Medicine Halle and University of Leipzig Medical Center. The movement disorder ADCY5-related dyskinesia can be treated with the asthma drug theophylline.

Genetic Disease 70

Genetic Disease Genetics Research Medicine 70

Medical Xpress

MAY 16, 2023

That was the case when Thilini Gamage was to carry out one of the studies in her doctoral work with Professor Eirik Frengen at the Institute of Clinical Medicine, University of Oslo. They study gene variation and mutations that cause rare genetic diseases. A mutation is a permanent change in the genetic material.

In-Vitro 98

In-Vitro In-Vivo Genetic Disease Genetics 98

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.

Genome 98

Genome Genomics Medicine Genetic Disease 98

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

Pharmaceutical Technology

JANUARY 10, 2023

The multi-year collaboration will focus on discovering precision genetic medicines by developing a new platform for complete gene insertion by delivering a single vector of gene editing and DNA cargo. Beyond the liver, ReCode’s SORT LNP genetic medicines technology enables the delivery to target cells and organs.

Gene Editing 130

Gene Editing Gene Gene Therapy Genetics 130

Scienmag

FEBRUARY 17, 2021

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

Medicine 55

Medicine Genetic Disease Development Genetics 55

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. The regulatory approval was supported by data from the GEM-1/2 and GEM-3 clinical trials.

FDA Approval 130

FDA Approval Gene Therapy Genetic Disease Gene 130

Scienmag

MAY 12, 2021

This genetic disease leads to the formation of benign tumours which can massively impair the proper functioning of vital organs […].

Genetic Disease 94

Genetic Disease Research Genetics Protein 94

Pharmaceutical Technology

AUGUST 17, 2022

The rights to the oRNA-LNP technology platform of Orna will be retained by the company, which will also progress various other fully owned programmes in oncology and genetic disease areas. In addition, Merck will make a $100m equity investment in Orna’s Series B funding round concluded recently.

RNA 147

RNA Vaccination Vaccine In-Vivo 147

XTalks

SEPTEMBER 13, 2021

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

RNA 105

RNA Genetics Genetic Disease Medicine 105

World of DTC Marketing

JANUARY 24, 2022

Particularly attractive are medicines ahead of the curve, aimed at diseases likely to move into the spotlight over the next few years. ” “More than 50% of FDA-approved medicines now have their start in venture-backed private biotech companies in the United States. co-founder, and managing director at Vida Ventures.

Life Science 225

Life Science Drugs Development Genetic Disease 225

Pharmaceutical Technology

JANUARY 12, 2023

Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM). Our pipeline of programmes focused on neuromuscular and cardiac rare disease gives us deep insight into the diverse and complex nature of dilated cardiomyopathy.”.

RNA 130

RNA Gene Therapy Genetics Genetic Disease 130

Outsourcing Pharma

OCTOBER 22, 2024

The European Medicines Agencyâs Committee for Medicinal Products for Human Use (CHMP) has once again snubbed Translarna (ataluren), PTC Therapeuticsâ treatment for the genetic disease Duchenne muscular dystrophy (DMD).

Genetic Disease 52

Genetic Disease Genetics Medicine Production 52

Scienmag

MAY 26, 2022

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

Gene Editing 63

Gene Editing Genetic Disease Gene Genome 63

Delveinsight

AUGUST 17, 2021

Food and Drug Administration gave a red flag to FibroGen’s anemia pill roxadustat, for the use in patients suffering from kidney diseases owing to safety concerns that arose after results, which in turn are concluded for the presentation of further clinical studies to assess safety for future medicinal use.

FDA Approval 98

FDA Approval Medicine Vaccination Vaccine 98

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

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

OCTOBER 14, 2020

Known as myofibrillar myopathies, these rare genetic diseases lead to progressive muscle wasting, affecting muscle function and causing weakness. Credit: Credit: Stephen Greenspan.

Genetic Disease 67

Genetic Disease Genetics Protein Research 67

Scienmag

JULY 13, 2021

In proof-of-concept study, CHOP researchers used an AAV9 vector to edit a single base mutation, halting progression of a disease that causes irreversible damage before birth Philadelphia, July 13, 2021–Adding to the growing body of literature demonstrating the feasibility of correcting lethal genetic diseases before birth, researchers at Children’s (..)

Genetic Disease 67

Genetic Disease Research Genetics Medicine 67

Scienmag

DECEMBER 17, 2020

Researchers at the University of Illinois Chicago have published a study showing a promising approach to using drug repurposing to treat genetic diseases. A team from the […].

Genetic Disease 52

Genetic Disease Genetics Gene Drugs 52

XTalks

MAY 26, 2023

Vyjuvek is also the first drug approved to treat the disease and is Krystal’s first approved product. DEB is a genetic disorder characterized by very fragile skin that rips and blisters easily even from minor friction (like rubbing or scratching) or injury, resulting in open wounds that are prone to skin infections and fibrosis.

Gene Therapy 97

Gene Therapy Gene FDA Approval Dermatology 97

Pharmaceutical Technology

FEBRUARY 23, 2023

The collaboration will combine the mRNA platform of Moderna with the gene editing technologies suite, including the base editing capabilities of Life Edit for the development of curative therapies to treat challenging genetic diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

pharmaphorum

NOVEMBER 27, 2020

“Today’s approval brings us closer to our ultimate goal of providing medicines for all people with CF,” said Vertex’s chief executive Reshma Kewalramani. CF is a rare, life-shortening genetic disease that affects the lungs, liver, gastrointestinal tract, sinuses, sweat glands, pancreas and reproductive tract.

Gene 134

Gene Genetic Disease FDA Approval Genetics 134