Medical Xpress

MARCH 27, 2023

Food and Drug Administration (FDA) applications for Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), a new article can help developers of gene therapies for rare genetic diseases. Providing an overview of the submissions process and examples of U.S.

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Drug Discovery World

DECEMBER 5, 2022

A new partnership between industry and three rare disease foundations has formed with the goal of accelerating the development of precision drugs to treat rare neurodevelopmental disorders. . The post Partnership takes on mission to advance precision drug development appeared first on Drug Discovery World (DDW).

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Pharmaceutical Technology

MAY 4, 2023

Vertex Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) for KALYDECO (ivacaftor) to treat children with cystic fibrosis (CF) aged between one and four months. CF is a rare, life-shortening genetic disease that is estimated to impact over 88,000 people across the globe.

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Drug Discovery World

MAY 3, 2024

Dr Incerti has more than three decades of experience in the biopharmaceutical industry and brings an extensive track record in global drug development, including from his time at Sanofi Genzyme where he played a leading role in pioneering therapies for rare and genetic diseases.

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XTalks

MARCH 21, 2024

The US Food and Drug Administration (FDA) has awarded approval to Orchard Therapeutics for its gene therapy Lenmeldy (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).

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Drug Discovery World

JANUARY 24, 2023

DMD and FA are rare, degenerative, genetic diseases that affect movement and eventually lead to paralysis. This is particularly important for rare diseases where it can be hard to identify suitable patients. In the FA study, teams worked with patients to identify key movement patterns and predict genetic markers of disease.

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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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Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy is a new therapeutic approach in which genes are used to treat or prevent diseases. Viruses and bacteria can be first modified to prevent them from causing infectious diseases and then implemented into human tissues as therapeutic gene vectors.

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Drug Discovery World

NOVEMBER 7, 2022

Genomic healthcare data is critical to identify disease risk, ancestry, traits and response to medicines and aids in the development of new targeted therapies – precision medicines. DDW’s Megan Thomas observes developments of accessibility in this sector and the potential impact. . The origins . The current landscape .

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Pharmaceutical Technology

APRIL 6, 2023

GRIA disorder is a family of rare genetic diseases. Through the study, Dr Coombs expects to characterise the genetic variants underlying various behavioural and cognitive symptoms in children suffering from GRIA disorder. He will further employ this diagnostic to select drugs for restoring patients’ normal functioning.

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Drug Discovery World

DECEMBER 15, 2022

Metrion will develop cell lines for both wild type and variant Kv3.1, The KCNC1 Foundation was created by the parents of a child diagnosed with KCNC1-related DEE to support research aimed at accelerating the development of a cure, as well as to raise awareness to identify and connect patients and provide support for families. . “We

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Pharmaceutical Technology

MAY 22, 2023

The US Food and Drug Administration (FDA) has granted approval for Krystal Biotech’s Vyjuvek (beremagene geperpavec-svdt) to treat dystrophic epidermolysis bullosa (DEB) in patients aged six months and above. Vyjuvek is a non-invasive, topical, re-dosable gene therapy that delivers functional human COL7A1 gene copies to offer wound healing.

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XTalks

APRIL 15, 2021

Experts from Servier and Genuity Science recently spoke on a webinar about using genomics data to drive drug development in heart failure and identify new targets for novel therapeutics. However, these drugs have shown limited effectiveness in treating HFpEF. Watch this on-demand webinar to hear from these experts.

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Drug Discovery World

JUNE 19, 2023

Cystic fibrosis is a progressive genetic disease that affects the lungs, pancreas and other organs. AdBio Partners’ mission is to support founding scientists in the development of new therapeutic approaches,” said Alain Huriez, Managing Partner of AdBio Partners. “We

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Drug Discovery World

DECEMBER 19, 2023

Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication. MPS I is a rare, genetic disease that affects the body’s ability to produce the enzyme alpha-L-iduronidase (IDUA).

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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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pharmaphorum

JULY 20, 2022

Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on. .

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Drug Discovery World

APRIL 17, 2024

What are the challenges of drug discovery in this area? DS: You say on your website that your approach is applicable to any disease where increasing protein expression is beneficial. What diseases are you currently targeting? JMB: Hundreds of genetic diseases involve a missing or deficient protein and/or enzyme.

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Drug Discovery World

DECEMBER 5, 2022

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases. . Many distinct types of parvoviruses infect mammals – including humans – without causing disease.

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Pharmaceutical Technology

JULY 29, 2022

In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs). However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea.

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XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). After further discussion with Orchard Therapeutics, the NHS negotiated an additional, undisclosed discount on the drug, allowing the health agency to now offer it to patients.

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World of DTC Marketing

JANUARY 24, 2022

Venture capitalists have poured $42 billion into drug development over the past three years. Most small biotech companies rely on venture capitalist funding to develop new drugs but is that a good way to go? Yet nearly half the money has flooded into cancer and rare diseases with expensive cures. Only about $2.2

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XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. Food and Drug Administration (FDA). Born with spinal muscular atrophy (SMA), the little boy was saved by the drug Zolgensma, a one-time intravenous infusion that restored the child’s defective SMN1 gene.

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Drug Discovery World

NOVEMBER 7, 2022

In 2018, the UK’s department of health announced an NHS Genomic Medicine Service, which allows patients with rare diseases to have their entire genetic code read in the hope of providing a much-needed diagnosis. That said, some people with rare genetic diseases remain without a molecular diagnosis after their genome is analysed. . “We

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The Pharma Data

NOVEMBER 19, 2020

MD, FAAP, Cincinnati Children’s Hospital, who will discuss genetic forms of hearing loss in children. MD, FAAP, Cincinnati Children’s Hospital, who will discuss genetic forms of hearing loss in children. The call will feature a presentation by KOL John Greinwald Jr., To register for the call, please click here.

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pharmaphorum

AUGUST 12, 2020

The enzyme replacement therapy (ERT) – also known as PRX-102 – has been granted a priority review by the US regulator, and is the top prospect in Chiesi’s recently formed rare diseases division. Market research firm Optima Insights has predicted that sales of Fabry disease drugs will more than double from around $1.8

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The Pharma Data

OCTOBER 15, 2020

Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a small molecule drug for the rare genetic disease Alpha-1 antitrypsin deficiency (AATD), canning the drug’s development after seeing lackluster results from an early phase 2 trial. James Miessler.

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XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.

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Drug Discovery World

NOVEMBER 17, 2022

Andrew Roddam, CEO of Our Future Health, shares the impact research programmes such as this will have on drug discovery. . Our Future Health aims to tackle this and help people live healthier lives for longer by creating the UK’s largest ever health research programme to prevent, detect and treat diseases.? The challenge .

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Drug Discovery World

AUGUST 28, 2023

And we’ve shown that, at least in animal models of these diseases, our drug molecules work really well. DS: Your research has so far focused on Parkinson’s disease and Amyotrophic Lateral Sclerosis (ALS). PH : This is a relatively new area of biology and it takes many years to get a new drug to the market.

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pharmaphorum

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. billion deal in 2018. The aim will be to identify promising candidates in areas of “unmet patient need.”

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pharmaphorum

SEPTEMBER 17, 2020

AstraZeneca has bought rights to an orally-active drug that inhibits PCSK9, offering a patient-friendly alternative to current cholesterol-lowering therapies that have to be delivered via a needle. The post AZ buys oral PCSK9 cholesterol drug from Dogma Therapeutics appeared first on. Novartis paid a hefty $9.7

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XTalks

MAY 17, 2023

The FNIH will focus on advancing clinical trials for the eight diseases through gene therapy research partnerships with the goal of accelerating the path of new therapies to clinical trials. It included formulating a standardized therapeutic development model that would help accelerate clinical trial processes for new treatments.

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pharmaphorum

OCTOBER 4, 2022

US-based LogicBio is a clinical-stage genetic medicine company focused on genome editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood. Instead of traditional CRISPR- based gene therapy, the company uses homologous recombination as the basis of its genome editing platform, ‘GeneRide’.

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Pharmaceutical Technology

MAY 8, 2023

Innovation S-curve for the pharmaceutical industry Zinc-finger nucleases is a key innovation area in pharmaceutical Zinc finger nucleases (ZFNs) are tools used in genetic engineering to cleave a chosen genomic sequence. ZFNs are also used to create a new generation of genetic disease models called isogenic human disease models.

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Drug Discovery World

FEBRUARY 15, 2024

In mouse models, the team found that S-Ac7-DOG encapsulated mRNA demonstrated markedly higher transfection, lower reactogenicity, and higher accumulation in the vaccine draining lymph nodes, tissues where immune responses against microbial antigens and tumour antigens are typically induced. Hypothetically, any mRNA-based drug using S-Ac7-DOG as (..)

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XTalks

FEBRUARY 28, 2022

Related: UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD. Below are some facts and information about rare diseases, including rare disease clinical trials and orphan drugs. How is a Rare Disease Defined? Are Most Rare Diseases Genetic?

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