Fierce Pharma

MARCH 20, 2024

After scoring an FDA nod Monday for the first gene therapy to treat the rare genetic disease metachromatic leukodystrophy (MLD) in the U.S., Kyowa Kirin and its subsidiary Orchard Therapeutics have | After scoring an FDA nod Monday for the first gene therapy to treat the rare genetic disease metachromatic leukodystrophy in the U.S.,

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Gene Therapy Gene Genetic Disease Genetics 125

Drug Discovery World

JUNE 11, 2024

Researchers have discovered a new biological pathway in inflammatory bowel disease (IBD) and related conditions that can be targeted using existing drugs. Despite increasing prevalence, current treatments do not work in every patient and attempts to develop new drugs often fail due to an incomplete understanding of what causes IBD.

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Genetics Research Gene Scientist 59

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. and Europe. Catalent will further support process optimization and look to reduce material.

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Gene Therapy Genetic Disease Genetics Gene 130

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. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene Development Manufacturing 242

Pharmaceutical Technology

SEPTEMBER 30, 2022

After several setbacks, bluebird bio bounces back with two major FDA gene therapy approvals. Last month, Zynteglo (betibeglogene autotemcel), or beti-cel, was approved as a one-time potentially curative gene therapy for patients with beta-thalassaemia who require regular blood transfusions.

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Gene Therapy FDA Approval Gene Genetic Disease 289

Worldwide Clinical Trials

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

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Gene Editing Gene Gene Therapy In-Vivo 189

Worldwide Clinical Trials

OCTOBER 24, 2023

While this is a small number of people in each rare disease patient community, the significant and continued growth of rare disease drug development is unsurprising given that there are over 10,000 rare diseases , with more being discovered every year. Roughly 80% of rare diseases are due to a known genetic driver.

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Gene Gene Editing Gene Therapy Clinical Trials 130

Pharmaceutical Technology

JULY 7, 2022

Genetic mutations, both germline and acquired, are behind a large proportion of the most debilitating and sometimes life-threatening human diseases. Recent years have seen a steadily growing number of approvals for cell and gene therapies, which has spurred on the community to continue innovating in this space.

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Gene Therapy Gene Medicine Genetics 245

Medical Xpress

FEBRUARY 3, 2023

Patients can experience 30% fewer serious adverse reactions if their drugs are tailored to their genes, reports a study published in The Lancet. A European collaboration involving researchers from Karolinska Institutet suggests that a genetic analysis prior to drug therapy could significantly reduce suffering and health care costs.

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Genetic Analysis Genetics Drugs Gene 97

Pharmaceutical Technology

MAY 26, 2023

Krystal Biotech has received approval from the US Food and Drug Administration for topical gene therapy VYJUVEK to treat dystrophic epidermolysis bullosa (DEB) in adults and in children aged six months and above. VYJUVEK is designed to address the underlying genetic cause of the disease.

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Gene Therapy Gene FDA Approval Manufacturing 264

pharmaphorum

JANUARY 24, 2023

Drug development has long been an issue for the pharma industry, due to the expense and the high failure rate of potential treatments. Ben Hargreaves finds that the vast amount of genetic data that exists today could help provide a faster, more targeted way of developing new drug candidates.

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Genetics Drugs Genome Project Biobanking 98

Bio Pharma Dive

APRIL 6, 2022

Facing mounting competition to its top-selling drug Eylea, Regeneron is teaming up with ViGeneron to develop a genetic medicine for an inherited retinal disease.

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Gene Therapy Gene Genetics Medicine 344

Pharmaceutical Technology

JANUARY 5, 2023

Capsida Biotherapeutics and Eli Lilly and Company ’s wholly owned subsidiary Prevail Therapeutics have announced a partnership for the development of non-invasive gene therapies for central nervous system (CNS) diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.

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Gene Therapy Gene Development Engineer 237

Drug Discovery World

JUNE 14, 2024

With a lead story on the restoration of congenital hearing loss following gene therapy, the news highlights selection this week focuses on cell and gene therapies (CGT) and genetics, particularly some key progress in relation to CAR-T therapies.

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Gene Therapy Drugs Gene Genetics 52

Pharma Mirror

FEBRUARY 23, 2021

The post Catalent to Acquire Delphi Genetics and Launch US Plasmid Manufacturing Site appeared first on Pharma Mirror Magazine. SOMERSET, N.J. SOMERSET, N.J. Catalent is also announcing the launch of pDNA development and manufacturing services at its Rockville, Maryland facility.

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Pharmaceutical Technology

MAY 25, 2023

Pushing back an initial deadline, the US Food and Drug Administration (FDA) has proposed a new regulatory action date of 22 June, by which time the agency will assess the logistics of a possible approval for Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy.

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Gene Therapy Gene Clinical Trials Clinical Trials 130

Pharmaceutical Technology

MAY 24, 2023

It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene. It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene.

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Gene Therapy Gene Licensing Licensing 130

XTalks

MARCH 28, 2024

Italy-based drugmaker Italfarmaco has won US Food and Drug Administration (FDA) approval for its oral medication Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older. The global incidence of DMD is approximately one in every 3,500 to 6,000 male births.

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FDA Approval Genetics Gene Therapy Gene 105

Pharmaceutical Technology

JUNE 8, 2023

The US Food Drug Administration (FDA) has granted orphan drug designation to DTx Pharma’s investigational DTx-1252 for the treatment of Charcot-Marie-Tooth disease Type 1A (CMT1A). DTx-1252 targets the underlying genetic lesion of the disease and leverages our FALCON platform to unlock the promise of RNAi therapeutics. “We

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Drugs Gene Therapy Gene RNA 246

Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Genetically modified animal models. Regeneron Pharmaceuticals is one of the leading patent filers in the generation of genetically modified animal models.

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Genetics DNA Antibody Genome 130

Drug Discovery World

JULY 14, 2023

University of Queensland (UQ) researchers have used genetics to reveal that much of the risk of developing the common and sometimes fatal diverticular disease of intestine (DivD) is inherited. The study has identified new drug targets and opened up possibilities for novel treatment strategies.

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Genetics Drugs Gene Genome 52

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.

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Gene Therapy Genetic Disease Gene Genetics 310

Drug Discovery World

JANUARY 19, 2024

UK scientists have uncovered the interplay between cancer-driving genetic mutations and inherited genetic variants in a rare type of blood cancer. In the future, this knowledge could aid drug development and interventions that reduce the risk of disease. MPNs can also progress into other forms of blood cancer, such as leukaemia.

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Genetics Gene DNA Development 59

Worldwide Clinical Trials

MARCH 20, 2024

In the increasingly advancing world of medicine, personalized care is a key factor that can progress your drug development efforts. As the study of how an individual’s genes affect their response to drugs, pharmacogenomics is a critical factor in developing effective and safe drugs and dosages in clinical trials.

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Clinical Trials Clinical Trials Trials Genetics 130

BioSpace

JULY 11, 2022

Epic Bio aims to transform genetic medicine by developing a new class of drugs that target the epigenome to alter gene expression.

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Genetics Gene Expression Medicine Engineer 106

Bio Pharma Dive

SEPTEMBER 22, 2022

The approval is another example of so-called tissue-agnostic indications, which are based on a cancer’s genetic signature rather than where it is in the body.

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Gene Genetics Drugs 183

pharmaphorum

MARCH 10, 2022

18 months after signing its first partnering deal with Genentech, Scenic Biotech has raised $31 million in first-round financing that it will use to take its own genetic modifier-targeted medicines into clinical trials. Scenic has developed a series of small-molecule drugs targeting QPTCL that block the expression of CD47 on cancer cells.

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Genetics Gene Sequencing Gene Clinical Trials 98

Drug Discovery World

JUNE 5, 2024

million to fund a new two-year collaborative partnership with King’s College London (KCL) to validate a portfolio of human genetic driven therapeutic targets for motor neuron disease (MND). Historical pipeline data suggests that drug targets with human genetic disease association are twice as likely to lead to approved drugs.

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Research Bioinformatics Drugs Genetics 69

Drug Discovery World

JUNE 23, 2023

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD.

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Gene Therapy FDA Approval Gene Protein 97

Drug Discovery World

OCTOBER 5, 2023

A research team at the Francis Crick Institute and Great Ormond Street Hospital (GOSH)/UCL Great Ormond Street Institute of Child Health have identified potential treatments for children with rare genetic conditions. Both treatments were able to improve the calcium problems, but the genetic therapy was the most effective.

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Genetics Research Gene Doctors 59

STAT News

DECEMBER 13, 2022

A new study suggests that the root of the problem is buried in our genetic code. Researchers uncovered four gene variants connected with the immune system that significantly increase a person’s risk of developing the disease, known as progressive multifocal leukoencephalopathy or PML.

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Genetics Immune Response Gene Drugs 105

BioPharma Reporter

AUGUST 18, 2022

Food and Drug Administration (FDA) has approved bluebird bioâs Zynteglo (betibeglogene autotemcel), also known as beti-cel: a one-time gene therapy custom-designed to treat the underlying genetic cause of beta thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.

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Gene Therapy Gene FDA Approval Genetics 105

Pharmaceutical Technology

APRIL 28, 2023

Ocugen has received orphan drug designation from the US Food and Drug Administration for its OCU410ST (AAV5-hRORA) to treat ABCA4 – linked retinopathies. Utilising an AAV delivery platform, OCU410ST enables the delivery of the RAR-related orphan receptor A (RORA) gene to the retina.

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Drugs Gene Protein Genetics 130

Drug Discovery World

DECEMBER 6, 2023

Gene editing company Eligo Bioscience has announced a successful $30 million Series B funding round, led by Sanofi Ventures. “We We feel this reflects the strong support for our vision and confirms the potential of Eligo to create a novel class of transformative genetic medicines.”

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Gene Editing Gene In-Vivo Genetics 52

Drug Discovery World

JANUARY 23, 2024

The world’s most comprehensive database for understanding microbial distribution and function in the ocean has been released, opening up new opportunities for drug discovery. The post World’s largest catalogue of ocean DNA could boost drug discovery appeared first on Drug Discovery World (DDW).

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DNA Drugs Gene Scientist 103

Drug Discovery World

JANUARY 25, 2023

The US Food and Drug Administration has also granted Orphan Drug designation for KB407 to treat patients with CF. The US Food and Drug Administration has also granted Orphan Drug designation for KB407 to treat patients with CF.

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Gene Therapy Gene Drugs Genetics 52

Scienmag

SEPTEMBER 29, 2021

Describing a previously unknown genetic condition that affects children, researchers at University of California San Diego School of Medicine and Rady Children’s Institute for Genomic Medicine say they also found a potential method to prevent the gene mutation by administering a drug during pregnancy.

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Genetics Research Genome Genomics 84