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 broken new boundaries in pricing, too.

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BioSpace

NOVEMBER 15, 2023

The Japanese pharma contends that an analysis of the four deaths in its AT132 gene therapy clinical trial shows it is still viable as a potential treatment for a fatal, rare genetic disease.

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Worldwide Clinical Trials

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment.

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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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Pharmaceutical Technology

JUNE 19, 2023

Although only a small number of gene therapies have reached the market thus far, the industry is poised to grow quickly over the next few years. According to GlobalData’s clinical trials database, there are currently 1,231 planned and ongoing trials for gene therapies and gene-modified cell therapies alone.

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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.

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pharmaphorum

AUGUST 10, 2021

Europe market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according to Andrew Obenshain, president of its severe genetic diseases unit. market and will wind down in Europe.

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XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

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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.

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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. The article is published in the journal Human Gene Therapy.

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XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Both therapies will be available in early 2024.

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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). It has been modified so that it cannot replicate in normal cells.

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Drug Discovery World

AUGUST 23, 2022

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . A rare inherited neurodegenerative disease, it’s thought to affect 62,000 people in the European Union.

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XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday. How Can Study Protocols Be More Effective?

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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. Parvovirus gene therapy is a cutting-edge biomedical technology.

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pharmaphorum

SEPTEMBER 6, 2021

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal.

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Drug Discovery World

FEBRUARY 27, 2023

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study. Patients with MPS-IIIA have a mutation in the SGSH gene, causing them to lack an enzyme which normally breaks down large sugar molecules.

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pharmaphorum

AUGUST 1, 2022

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a view to making it available in sometime around the middle of 2023.

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STAT News

FEBRUARY 22, 2023

Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines. The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions.

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Drug Discovery World

MARCH 8, 2023

If successful, GNT-0003 would be the first gene therapy for Crigler-Najjar syndrome.” PRIME status was granted to GNT-0003 following promising results from early phases of the European trial currently underway in collaboration with the CureCN consortium, and sponsored by Genethon.

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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.

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Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. It has been estimated that by 2025 the FDA will be approving 10 to 20 cell and gene therapy products per year 2.

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pharmaphorum

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. CALD is caused by mutations in the ABCD1 gene located on the X chromosome, which provides instructions for the production of the ALD protein.

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pharmaphorum

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). The post SparingVision raises €75m for eye disease gene therapies appeared first on.

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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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Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”

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Pharmaceutical Technology

MAY 22, 2023

Vyjuvek is a non-invasive, topical, re-dosable gene therapy that delivers functional human COL7A1 gene copies to offer wound healing. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The company intends to commercialise Vyjuvek in the US in the third quarter of 2023.

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STAT News

JANUARY 30, 2023

Wiseman, 51, who lives near Sacramento, was diagnosed as an infant with hemophilia, the rare genetic disease that prevents blood from clotting. Given everything he’s been through, Bobby Wiseman says he should have been dead instead of celebrating the birth of his first grandchild several days ago.

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pharmaphorum

JANUARY 11, 2021

Under the plans the company’s rare disease drugs will remain under the aegis of bluebird with current genetic disease president Andrew Obenshain taking the reins as CEO. The post bluebird bio to split into oncology and gene therapy specialists appeared first on. million price tag.

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BioPharma Reporter

FEBRUARY 23, 2023

The messenger RNA (mRNA) specialist Moderna has teamed up with ElevateBio-owned Life Edit Therapeutics to develop gene editing therapies that are delivered into patients in vivo.

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BioTech 365

OCTOBER 5, 2020

Diamond Pharma Services has provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight, leading to the Marketing Authorisation Application for LUMEVOQ LUMEVOQ is a gene therapy to treat vision loss due to the rare, mitochondrial genetic disease, Leber Hereditary … Continue reading →

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Drug Discovery World

JULY 3, 2024

As a result, the data provides an important proof-of-concept for Circio’s circular RNA platform, which we expect will translate into improved gene therapies for patients in the future,” said Dr Thomas Hansen, CTO at Circio. “In In parallel, we have deployed machine learning to optimise circVec sequence composition.

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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. The post AstraZeneca pays record 660% premium for gene editing company LogicBio appeared first on.

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Pharmaceutical Technology

JULY 29, 2022

However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea. The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder.

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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

Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM). Solid Biosciences stated that the partnership boosts its scientific capabilities along with commercial potential in cardiac therapy. By Cytiva Thematic.

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BioTech 365

SEPTEMBER 25, 2020

NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that Emil D. NOVATO, Calif., 25, 2020 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. Kakkis, M.D., Ph.D., … Continue reading →

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