Pharmaceutical Technology

APRIL 4, 2023

Abacus Medicine Pharma Services (AMPS) and Laboratoires CTRS, marketing authorization holder of Orphacol®, are proud to announce a strategic partnership that ensures continuous stock of Orphacol® in Spain. Our ambition is to provide better access to medicines and better healthcare to patients around the world.

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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.

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Genetics Genetic Disease Medicine Development 97

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.

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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.

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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. HS: Absolutely.

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Genome Genomics Medicine Genetic Disease 98

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.

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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.”

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Gene Editing Genome Genomics Genetic Disease 119

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.

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Gene Therapy Gene Development Manufacturing 242

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.

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Genetic Disease Genetics Scientist Development 52

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.

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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.

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Genetic Disease Genetics Medicine Drugs 98

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.

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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

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Genetics Genetic Disease Development Bioinformatics 70

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.

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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.

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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.

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Scienmag

MAY 12, 2021

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

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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.

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Gene Editing Gene Gene Therapy Genetics 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).

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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.

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Gene Editing Genetic Disease Gene Genome 63

XTalks

DECEMBER 18, 2024

Trial data for both the pediatric and adult trials were published in The New England Journal of Medicine. Ingrezza (valbenazine), first approved in 2017 for tardive dyskinesia, received a key label expansion in 2023 to include the treatment of chorea in patients with Huntingtons disease.

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Genetics Hormones Trials Drugs 116

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.

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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.

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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.

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Gene Genetic Disease FDA Approval Genetics 134

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.

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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 (..)

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Genetic Disease Research Genetics Medicine 67

pharmaphorum

NOVEMBER 19, 2021

Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company. The post NICE reaches a deal with Roche on access to oral SMA drug Evrysdi appeared first on.

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Drugs Gene Therapy Genetic Disease Gene 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.

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Genetic Disease Genetics Development Research 52

XTalks

MAY 26, 2023

The regulatory nod “ushers in a whole new paradigm to treat genetic diseases” and is a major milestone for DEB patients and their families, Krystal Biotech CEO Krish Krishnan said in a statement from the company. Results of the trials are published in the journals Nature Medicine and The New England Journal of Medicine.

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Gene Therapy Gene FDA Approval Dermatology 97

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).

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RNA Gene Therapy Genetics Genetic Disease 130

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 […].

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Genetic Disease Genetics Gene Drugs 52

The Pharma Data

OCTOBER 21, 2020

NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that the company will host a conference call and live webcast on Thursday, November 5, 2020 at 8:00 a.m. CAMBRIDGE, Mass., 22, 2020 (GLOBE NEWSWIRE) — Agios Pharmaceuticals, Inc.

Genetic Disease 52

Genetic Disease Genetics Medicine Development 52

Scienmag

APRIL 11, 2022

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.

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Genetic Disease Genetics Research Medicine 61

pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. per share, a rare 660% premium on LogicBio’s share price. .

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Gene Editing Gene Genome Genomics 64

Scienmag

APRIL 21, 2021

New approach to gene therapy can correct any disease-causing mutation within a gene UNIVERSITY PARK, Pa.

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Gene Gene Editing Gene Therapy Genetic Disease 52

Scienmag

JUNE 7, 2021

CSHL is a world leader in basic biological research and in target discovery for human genetic diseases like cancer. Autobahn will provide […].

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Genetic Disease Life Science Genetics Research 52

Scienmag

SEPTEMBER 11, 2020

New haven, CT: Researchers at Yale have identified a possible treatment for Duchenne muscular dystrophy (DMD), a rare genetic disease for which there is currently no cure or treatment, by targeting an enzyme that had been considered “undruggable.” ” The finding appears in the Aug. 25 edition of Science Signaling.

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Genetic Disease Genetics Drugs Research 50