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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Gene Therapy Gene Dermatology FDA Approval 97

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. The post Sarepta says early filing for DMD gene therapy is back on appeared first on.

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Gene Therapy Gene Genetics Trials 105

Pharmaceutical Technology

AUGUST 17, 2022

Merck will also make royalty payments on any approved products developed out of the partnership. 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.

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RNA Vaccine Vaccination In-Vivo 147

pharmaphorum

JANUARY 11, 2021

US biotech bluebird bio has announced plans to split into two this year, with a separate oncology business spinning off as the company prepares to bring its products to market. The gene therapy firm will be focused on its most important therapies in beta-thalassemia, cerebral adrenoleukodystrophy and sickle cell disease in the US And Europe.

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Gene Therapy Gene Genetic Disease Manufacturing 66

Pharmaceutical Technology

MARCH 1, 2023

Under the agreement terms, Tevard will receive up-front, option-exercise, as well as milestone payments, along with royalties on any products that are approved. The company is pioneering tRNA-based therapeutics for modulating mRNA function and curing several genetic diseases.

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Development Gene Therapy Genetics Genetic Disease 130

Pharmaceutical Technology

JANUARY 12, 2023

Solid Biosciences president and CEO Bo Cumbo said: “This collaboration with Phlox Therapeutics is an exciting opportunity to work with a company that shares our commitment and innovative approach to bringing transformative therapies to patients with rare genetic diseases. By Cytiva Thematic.

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RNA Gene Therapy Genetics Genetic Disease 130

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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Gene Therapy Gene Gene Sequencing Gene Editing 52

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. million price tag make it is the most expensive treatment ever approved for NHS funding.

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Drugs Gene Therapy Genetic Disease Gene 98

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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Gene Therapy Genetic Disease Gene Genetics 40

Delveinsight

AUGUST 26, 2021

Shape Therapeutics inks gene therapy deal with Roche worth up to USD 3 Billion. Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease.

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Vaccine Vaccination Gene Therapy RNA 98

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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RNA Genetic Disease Drugs Protein 52

pharmaphorum

MAY 6, 2021

Thalassaemia is a severe genetic disease that is characterised by significantly reduced production of functional beta-globin, a component of haemoglobin, the oxygen-carrying protein in the blood. Severely-affected patients need regular blood transfusions to maintain their haemoglobin levels. .

Genetic Disease 104

Genetic Disease Protein RNA Doctors 104

pharmaphorum

DECEMBER 6, 2020

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Roots Analysis

FEBRUARY 21, 2022

Since the introduction of a gene sequencing method by Frederick Sanger in 1977, the field of genomic data collection and analysis has evolved significantly. Moreover, the manual protocols require extensive manipulation, costly reagents and long duration of skilled genomic library production. Product Competitiveness Analysis.

Marketing 52

Marketing Gene Sequencing DNA Genomics 52

XTalks

NOVEMBER 27, 2023

Pompe disease, also referred to as acid-maltase disease and glycogen storage disease II, is an uncommon genetic disorder characterized by the gradual weakening of both cardiac and skeletal muscles. The body relies on this enzyme to break down glycogen, a stored form of sugar utilized for energy production.

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FDA Approval Clinical Trials Clinical Trials Trials 104

XTalks

SEPTEMBER 17, 2020

In the case of the former, pharmaceutical and biologics companies would be eligible for approval of their product through traditional regulatory pathways; the latter could support accelerated approval. Symptoms and Etiology: Cushing’s disease is a disorder caused by excess cortisol which is often the result of a pituitary adenoma.

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Trials Gene Hormones Clinical Trials 98

Pharmaceutical Technology

APRIL 24, 2023

Due to their size, cells can absorb them where they release products. Conde says the use of nanoparticle delivery for gene therapies has become more common over recent years. Lipid nanoparticles are spherical vehicles made from ionizable lipids. Before this, people would say nanotechnology is not going to work well.

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Drug Delivery Vaccine Vaccination Research 147

The Pharma Data

JANUARY 9, 2021

(NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants.

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Gene Therapy Gene Trials Clinical Trials 52

XTalks

NOVEMBER 10, 2023

The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

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FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

AUGUST 6, 2020

TSC is a rare genetic disease that affects approximately 1 in 6,000 people. The disease usually presents within the first year of life, displaying subtle signs that can take years to develop. Due to this, the disease can be misdiagnosed or not even recognized for a while.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

The Pharma Data

JULY 6, 2021

Data will include the final analysis from the phase IIIb STASEY study of Hemlibra® (emicizumab) and updated data from the phase I/II study of SPK-8011, an AAV-based gene therapy in development by Spark Therapeutics (a member of the Roche Group). Roche’s Chief Medical Officer and Head of Global Product Development.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

pharmaphorum

APRIL 20, 2022

The potential to develop treatments with CRISPR gene editing is hinged on its ability to add, delete or replace elements within a target DNA sequence. Cancer is a genetic disease, caused by certain changes in the way that genes control cell function, such as how they grow and divide. Why cancer? The pipeline.

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Gene Editing DNA Gene Genetics 52

The Pharma Data

DECEMBER 13, 2020

“Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies.

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Business Development Development Gene Editing In-Vivo 40

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.

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Gene Therapy Gene Gene Editing Genetics 238

The Pharma Data

NOVEMBER 29, 2020

In rare cases it is caused by a point mutation in the RAI1 gene in the deleted region. Oxlumo decreases oxalate production. It works by degrading HAO1 mRNA and decreasing the synthesis of glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1. The most common is a severe sleep disorder.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 52

Roots Analysis

AUGUST 12, 2024

According to the World Health Organization, there are over 40,000 in vitro diagnostics products available in the market. This vast array of products highlights the critical role of diagnostics in healthcare. Such test can be used for gene expression profiling, genotyping and detecting chromosomal abnormalities.

Genetics 40

Genetics Gene Expression RNA DNA 40

The Pharma Data

NOVEMBER 19, 2020

He has pioneered the establishment of diagnostic evaluation algorithms for children with sensorineural hearing loss and developed a next generation sequencing platform to determine the genetic causes of hearing loss in children. He has 92 peer review articles published with the majority related to hearing loss. www.sensorion-pharma.com.

Genetics 52

Genetics Containment Genetic Disease Gene 52

The Pharma Data

DECEMBER 9, 2020

Rocket intends to use the net proceeds from this offering to further fund the development of its pipeline of gene therapies for rare diseases, including filing for marketing authorization for RP-L201 in the United States and Europe, accelerating the buildout of in-house manufacturing capabilities, and for general corporate purposes.

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Gene Therapy Genetics Genetic Disease Sales 40

The Pharma Data

DECEMBER 27, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869).

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Gene Therapy Clinical Trials Clinical Trials In-Vivo 40

XTalks

AUGUST 7, 2024

She began her career as a research scientist, working for more than eight years in this role before transitioning into product management and marketing. Throughout her career, Lawreen has launched over 50 products and worked with various companies, from biotech start-ups to a Fortune 100 company.

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Life Science Scientist Production Research 52

The Pharma Data

DECEMBER 17, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (Paris:LYS) (FR0013233475 – LYS), a phase 3 gene therapy platform Company targeting central nervous system (CNS) diseases, announces a change in the governance and control of KGA, a company co-owned by Karen Aiach and which currently owns approximately 6% of Lysogene’s capital.

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Gene Therapy Clinical Trials Clinical Trials Gene 40

The Pharma Data

NOVEMBER 11, 2020

. Submitted Clinical Trial Application (CTA) for INZ-701 for the treatment of ENPP1 deficiency – Inozyme recently submitted its first CTA to initiate a Phase 1/2 clinical trial of INZ-701 for the treatment of ENPP1 deficiency to the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA).

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Clinical Trials Clinical Trials Trials Development 40

The Pharma Data

NOVEMBER 2, 2020

The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. This condition is caused by a mutation in the fibroblast growth factor receptor 3 gene ( FGFR3 ), a negative regulator of bone growth.

Drugs 40

Drugs Containment Trials Gene 40

The Pharma Data

OCTOBER 28, 2020

Five patients between the ages of 5 and 15 with deletions in the maternal UBE3A gene region were enrolled in the first three cohorts and are included in the interim data analysis. Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. About Angelman Syndrome.

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Protein Production Development Drugs 40

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.

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Gene Therapy Gene Trials Clinical Trials 95

The Pharma Data

JANUARY 25, 2021

The Company may also use net proceeds to fund potential acquisitions of, or investments in, complementary businesses, products, services and technologies. BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source.

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Sales Marketing Development Containment 52

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.

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Gene Therapy Gene FDA Approval In-Vivo 98