Spark Therapeutics partners with SpliceBio for gene therapy
Pharmaceutical Technology
OCTOBER 17, 2023
SpliceBio will license Spark Therapeutics’ propriety protein splicing platform to develop a gene therapy for renal disease.
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Pharmaceutical Technology
OCTOBER 17, 2023
SpliceBio will license Spark Therapeutics’ propriety protein splicing platform to develop a gene therapy for renal disease.
Pharmaceutical Technology
DECEMBER 22, 2023
Discover Amicus Therapeutics' groundbreaking gene therapy vectors and methods of use. Enhance protein expression and cellular targeting for effective treatment of genetic disorders. Explore the patent claim now!
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Bio Pharma Dive
NOVEMBER 19, 2020
The results are first to emerge from a late-stage test of a hemophilia B gene therapy, and show UniQure's treatment can restore clotting protein levels to mild or even near normal levels.
Camargo
JULY 27, 2021
How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.
Pharmaceutical Technology
MARCH 10, 2023
On 10 March, the National Health Service Blood and Transplant (NHSBT) opened a new Clinical Biotechnology Centre (CBC) with the aim of improving the UK’s ability to develop and manufacture cell and gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
Pharmaceutical Technology
MAY 25, 2023
ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. ElevateBio intends to use the funds to advance its genetic medicine current good manufacturing practice (cGMP) and process development business, BaseCamp.
Pharmaceutical Technology
NOVEMBER 9, 2022
On November 2, the Institute for Clinical and Economic Review (ICER) released its updated evidence aimed at measuring the clinical effectiveness and cost of the two haemophilia gene therapies. Known by the brand name Roctavian, BioMarin’s haemophilia A therapy valoctocogene roxaparvovec could be fairly priced in the range of $1.95–1.96
Pharmaceutical Technology
JULY 7, 2022
For many decades, investigators have been working on innovative therapeutic modalities known as cell and gene therapies, which use modified versions of the body’s own cellular and genetic material to treat and potentially cure these diseases. A new frontier in cancer research.
Bio Pharma Dive
DECEMBER 12, 2022
New data at ASH show the therapy can maintain levels of a key blood-clotting protein for years. Roche’s subsidiary Spark has offered few updates on its hemophilia A treatment since being acquired in 2019.
Pharmaceutical Technology
JUNE 8, 2023
GenScript ProBio has collaborated with the New York Blood Center Enterprises’ business unit, called Comprehensive Cell Solutions (CCS), to accelerate the development and manufacture of cell and gene therapies. The core objective of the collaboration is to make the therapies affordable and accessible to patients.
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.
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 accelerated approval follows a vote of support from the FDA Cellular, Tissue and Gene Therapies Advisory Committee and the granting of priority review status.
Bio Pharma Dive
JUNE 23, 2022
After one year, researchers detected important protein changes in patients who received a low dose of the experimental treatment. Further testing and functional data are needed to assess its potential, however.
Pharmaceutical Technology
APRIL 13, 2023
Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER). Also known as lovo-cel, bluebird bio’s product is a lentiviral gene therapy.
Bio Pharma Dive
DECEMBER 9, 2021
The amount of blood clotting protein found in patients who received the medicine was nearly the same at six months and 18 months post-treatment, according to results disclosed Thursday.
pharmaphorum
AUGUST 23, 2022
France’s BrainVectis, a subsidiary of Bayer’s Asklepios BioPharma (AskBio) unit, has been given the green light by regulators in France to start dosing patients with its gene therapy candidate for devastating neurodegenerative disorder Huntington’s disease.
Drug Discovery World
OCTOBER 18, 2023
Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies.
Pharmaceutical Technology
MARCH 28, 2024
Discover how 4D Molecular Therapeutics Inc's patented variant AAV capsid proteins enhance muscle cell infectivity for gene therapy, revolutionizing treatment for genetic disorders.
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. . The disease is caused by anomalous repeating mutations in the huntingtin gene leading to abnormal protein aggregates in nerve cells.
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. It also affects Hispanic Americans, but at a lower prevalence.
Pharmaceutical Technology
JUNE 15, 2023
Japan-based Astellas Pharma has entered a partnership with Cullgen to discover and develop multiple targeted protein degraders through the latter’s proprietary uSMITE targeted protein degradation platform, in a deal potentially worth $1.9bn. This method can target enzymes, transcription factors, scaffold proteins and more.
XTalks
JUNE 28, 2023
Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). DMD is caused by the absence of dystrophin, a protein that helps maintain the integrity of muscle cells.
XTalks
JUNE 30, 2023
It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).
pharmaphorum
DECEMBER 2, 2021
Astellas has signed another bolt-on deal to build its gene therapy pipeline, agreeing a deal with Dyno Therapeutics to tap into its adeno-associated virus (AAV) vector platform for delivering genes to skeletal and cardiac muscle. Those safety issues have hit Astellas’ aspirations in gene therapy directly.
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.
pharmaphorum
MAY 13, 2021
The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. Regardless of cohort, eligible participants are scheduled to receive the investigational gene therapy, either at the start of the study or after one year following treatment with placebo.
Drug Discovery World
SEPTEMBER 6, 2023
The funding will support the development of the company’s gene therapy in a powder, a non-viral gene delivery system to improve tissue regeneration following surgery. The technology allows rapid transfection of patients’ cells with the gene(s) of interest.
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.
Drug Discovery World
MAY 23, 2023
The US Food and Drug Administration (FDA) Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) have voted eight to six in favour of a new gene therapy for Duchenne muscular dystrophy. SRP-9001 is intended to deliver a gene that codes for a shortened, functional form of dystrophin to muscle cells.
Pharmaceutical Technology
MAY 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 Immuno-oncology in Pharmaceuticals: Oncolytic viral proteins. Sillajen and Advaxis are the other key patent filers of oncolytic viral proteins.
Drug Discovery World
DECEMBER 19, 2022
Researchers have found that a topical gene therapy gel helps heal wounds in patients with life-threatening blistering skin disease epidermolysis bullosa (EB). . The post ‘Life-changing’ topical gene therapy gel heals EB wounds appeared first on Drug Discovery World (DDW).
Drug Discovery World
JANUARY 25, 2023
The post Gene therapy for cystic fibrosis receives EC orphan drug designation appeared first on Drug Discovery World (DDW). The European Commission has granted orphan designation for Krystal Biotech’s KB407 for the treatment of cystic fibrosis (CF).
pharmaphorum
JANUARY 12, 2022
The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it may have the clinical data needed to file for regulator approval. point increase on the NSAA scale with the gene therapy, while the control group saw a 0.7-point
BioPharma Reporter
OCTOBER 22, 2020
Scientists have used gene therapy and a newly developed light-sensing protein to restore sight in mice: with the technology to start clinical trials in the US later this year.
pharmaphorum
JULY 7, 2022
New data on Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have introduced a concern about its safety. ” The post Myocarditis case mars Sarepta DMD gene therapy readout appeared first on.
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. The post FDA slaps clinical hold on BioMarin’s PKU gene therapy appeared first on.
Drug Discovery World
MAY 29, 2023
The US Food and Drug Administration has approved Krystal Biotech’s Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of dystrophic epidermolysis bullosa (DEB). DEB is a genetic disorder that affects the connective tissue in the skin and nails and results from mutation(s) in the COL7A1 gene.
BioTech 365
AUGUST 19, 2021
Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: CRISPR pioneer Feng Zhang’s latest work delivers mRNA, gene therapy with a human protein.CRISPR pioneer Feng Zhang’s latest work delivers mRNA, gene therapy with a human protein … Continue reading →
XTalks
JUNE 8, 2021
Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. Related: Is $2 Million Too Much For FDA-Approved SMA Gene Therapy? With a price tag of over $2.5
pharmaphorum
DECEMBER 22, 2021
Pfizer has called a halt to a clinical trial of its gene therapy for Duchenne muscular dystrophy as it investigates the unexpected death of a young male patient. AAV vectors are well-suited to gene therapies for DMD because they are effective at targeting muscle tissue.
Roots Analysis
NOVEMBER 30, 2021
Bioassay – A Prerequisite for the Cell and Gene Therapy Development. Over the past few years, investigational new drug (IND) filings for cell and gene therapy product have significantly increased. Companies Offering Bioassay Services for Cell and Gene Therapies.
Drug Discovery World
SEPTEMBER 6, 2022
DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. million price point per dose.
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.
Medical Xpress
MARCH 18, 2023
University of Utah Health scientists have corrected abnormal heart rhythms in mice by restoring healthy levels of a protein that heart cells need to establish connections with one another.
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