Onasemnogene abeparvovec

Solve GNE and Genosera Sign Agreement to Develop a Novel Bicistronic AAV Gene Therapy Targeting HIBM

Retrieved on: 
Monday, August 14, 2023
NCH, Gene, GNE, Myopathy, Research, DSM-IV codes, Doctor of Philosophy, AAV, Duchenne muscular dystrophy, Muscle weakness, Incidence, Spinal muscular atrophy, Genetic disorder, MD, Patient, HIBM, Lipase, Onasemnogene abeparvovec, Nationwide, Hereditary inclusion body myopathy, Disease, Pharmaceutical industry, Jews

LOS ANGELES, Aug. 14, 2023 (GLOBE NEWSWIRE) -- Solve GNE announced today that it has entered into a sponsored research agreement with Genosera to provide funding for a novel Adeno-Associated Virus (AAV) gene therapy aimed at curing GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM).

Key Points: 
  • LOS ANGELES, Aug. 14, 2023 (GLOBE NEWSWIRE) -- Solve GNE announced today that it has entered into a sponsored research agreement with Genosera to provide funding for a novel Adeno-Associated Virus (AAV) gene therapy aimed at curing GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM).
  • Genosera is developing novel bicistronic, or two gene, AAV treatments for muscle diseases.
  • Together with Solve GNE, Genosera will work to develop the first bicistronic gene therapy treatment for GNE myopathy patients.
  • Solve GNE is proud to be collaborating with Genosera and NCH towards finding a cure for GNEM.”

Gene therapy helps combat some forms of blindness – and ongoing clinical trials are looking to extend these treatments to other diseases

Retrieved on: 
Friday, May 12, 2023
Retina, Tissue, DNA, Conversation, University, Life, Pharmacy, Eye, Retinitis pigmentosa, Visual impairment, Cancer, RPE65, Hematology, Paratriathlon, Research, Brain, Cell, RNA, DSM-IV codes, Achromatopsia, Muscle weakness, System, CAR, Operating, Genetic disorder, Patient, Ophthalmology, Congenital blindness, Choroideremia, Virtual reality, FDA, Onasemnogene abeparvovec, Death, Gene, Conference, Eye disease, Disease, Pharmaceutical industry, Antibiotics, Vaccine

While not every form of blindness can be cured, recent scientific breakthroughs have uncovered new ways to treat some forms of inherited blindness through gene therapy.

Key Points: 
  • While not every form of blindness can be cured, recent scientific breakthroughs have uncovered new ways to treat some forms of inherited blindness through gene therapy.
  • Jean Bennett is a gene therapy expert and a professor emeritus of ophthalmology at the University of Pennsylvania.
  • She and her laboratory developed the first gene therapy drug for a genetic disease to be approved in the U.S.

What is gene therapy and how does it work?


    Gene therapy is a set of techniques that harness DNA or RNA to treat or prevent disease. Gene therapy treats disease in three primary ways: by substituting a disease-causing gene with a healthy new or modified copy of that gene; turning genes on or off; and injecting a new or modified gene into the body.

How has gene therapy changed how doctors treat genetic eye diseases and blindness?

    • In the past, many doctors did not think it necessary to identify the genetic basis of eye disease because treatment was not yet available.
    • Over time, we were able to create a treatment designed for individuals with particular gene defects that lead to congenital blindness.
    • Gene therapy treatments are now available in pharmacies and operating rooms all over the world.

You created one of the first gene therapies approved in the US. What is the current state of the clinical use of gene therapy?

    • There are now many approved gene therapies in the U.S., but the majority are combined with cell therapies in which a cell is modified in a dish and then injected back into the patient.
    • The majority of those therapies target different forms of cancer, although there are several for devastating inherited diseases.
    • The gene therapy that my team and I developed was the first FDA-approved project involving injection of a gene therapy directly into a person – in this case, into the retina.

What are you currently working on that you’re most excited about?

    • I am very excited about some upcoming clinical trials that my team will soon initiate to target some other devastating blinding diseases.
    • This test utilizes a virtual reality game that is not only fun for the user but promises to provide an objective measure of the person’s functional vision.

What are the biggest challenges gene therapy faces?

    • The biggest challenges involve systemic diseases, or diseases affecting the entire body rather than a single organ or body part.
    • For those diseases, super-high doses of gene therapy reagents must be delivered.
    • Cost remains a key issue in this effort – gene therapy drugs are enormously expensive.

Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset

Retrieved on: 
Sunday, March 19, 2023
Failure mode and effects analysis, Non-invasive ventilation, SMA, Interim, Gastroenteritis, SMN2, IV, Spinal muscular atrophy, Observational study, STRONG, Marketing, Dehydration, Survival, Clinical trial, Quality of life, Commonwealth of Australia (US securities entity), Research, Ventilation, Government, Sale, Walking, MD, Patient, Muscular Dystrophy Association, Regulation, Physician, Pandemic, Episcopal conference, Common, Viral, Diagnosis, MDA, Periodic breathing, Nationwide, Conference, Pneumonia, Safety, Disease, Birth control, Pharmaceutical industry, Nursing, Medical device, Sales, COVID-19, Novartis, Onasemnogene abeparvovec

“These children now have an improved quality of life, vastly different from what would have been expected for them if they had not received treatment.

Key Points: 
  • “These children now have an improved quality of life, vastly different from what would have been expected for them if they had not received treatment.
  • Results from the IV cohort, which included 63 patients, demonstrated how a single administration of Zolgensma provided consistent, substantial and durable efficacy over time.
  • “Data from the LT-001 and LT-002 studies showed that, regardless of the patient’s symptomatic status at the time of treatment, Zolgensma IV is an effective and durable treatment option.
  • Nor can there be any guarantee that such products will be commercially successful in the future.

Spinal Muscular Atrophy Treatment Market Worth $18.0 Billion by 2030: Grand View Research, Inc.

Retrieved on: 
Wednesday, January 11, 2023
Clinical trial, Ageing, SMA, Gene therapy, EC, UNC Medical Center, Food, Therapy, Research, Spinal muscular atrophy, Genentech, Royal commission, USD, SAN, Disease, Growth, Patient, Novartis, MAP, Revenue, Treatment, Route of administration, European Commission, Food and Drug Administration Amendments Act of 2007, EMA, Prevalence, Diagnosis, PTC Therapeutics, Pharmaceutical industry, Medical device, Medical imaging, Nusinersen, Onasemnogene abeparvovec, Risdiplam

SAN FRANCISCO, Jan. 11, 2023 /PRNewswire/ -- The global spinal muscular atrophy treatment market size is expected to reach USD 18.0 billion by 2030, according to a new study by Grand View Research Inc.

Key Points: 
  • SAN FRANCISCO, Jan. 11, 2023 /PRNewswire/ -- The global spinal muscular atrophy treatment market size is expected to reach USD 18.0 billion by 2030, according to a new study by Grand View Research Inc.
  • For instance, in July 2021, F. Hoffmann-La Roche Ltd launched Evrysdi (risdiplam) for the treatment of patient with spinal muscular atrophy in India.
  • Thus, increasing number of product approvals is expected to drive the spinal muscular atrophy treatment market.
  • Grand View Research has segmented the global spinal muscular atrophy treatment market based on type, application, technology, end-use, and region:
    Spinal Muscular Atrophy Treatment Market - Type Outlook (Revenue, USD Million, 2018 - 2030)

Spinal Muscular Atrophy Treatment Market Report 2022: Continued Product Approvals Bolsters 18.6% Annual Growth - ResearchAndMarkets.com

Retrieved on: 
Thursday, December 29, 2022
Health, Pharmaceutical, Clinical trial, Ageing, SMA, Gene therapy, EC, UNC Medical Center, Administration, Food, Therapy, Spinal muscular atrophy, Research, Genentech, Royal commission, USD, Disease, Growth, Patient, Novartis, MAP, Treatment, Route of administration, European Commission, Segmental analysis (biology), Food and Drug Administration, EMA, Diagnosis, PTC Therapeutics, Medical device, Pharmaceutical industry, Medical imaging, Coal, Nusinersen, Onasemnogene abeparvovec, Risdiplam

The global spinal muscular atrophy treatment market size is expected to reach USD 18.0 billion by 2030.

Key Points: 
  • The global spinal muscular atrophy treatment market size is expected to reach USD 18.0 billion by 2030.
  • In March 2021, PTC Therapeutics, Inc., received marketing approval for Evrysdi (risdiplam) from the EMA for the treatment of patient with type1, 2, and 3 spinal muscular atrophy.
  • Thus, increasing number of product approvals is expected to drive the spinal muscular atrophy treatment market.
  • For instance, in July 2021, F. Hoffmann-La Roche Ltd launched Evrysdi (risdiplam) for the treatment of patient with spinal muscular atrophy in India
    Chapter 4 Global Spinal Muscular Atrophy Treatment Market - Segment Analysis, By Type, 2018 - 2030 (USD Million)
    Chapter 5 Global Spinal Muscular Atrophy Treatment Market - Segment Analysis, by Treatment, 2018 - 2030 (USD Million)
    Chapter 6 Global Spinal Muscular Atrophy Treatment Market - Segment Analysis, By Drug, 2018 - 2030 (USD Million)
    Chapter 7 Global Spinal Muscular Atrophy Treatment Market - Segment Analysis, by Route of Administration, 2018 - 2030 (USD Million)
    Chapter 8 Spinal Muscular Atrophy Treatment Market: Segment Analysis, by Region, 2018 - 2030 (USD Million)
    Chapter 9 Global Spinal Muscular Atrophy Treatment Market: Competitive Analysis

Spinal Muscular Atrophy Treatment Market Size, Share & Trends Analysis Report By Type, By Treatment, By Drug, By Route Of Administration, By Region And Segment Forecasts, 2022 - 2030

Retrieved on: 
Thursday, December 15, 2022
Therapy, Spinal muscular atrophy, Ageing, SMA, Food, Gene therapy, UNC Medical Center, MAP, EC, Diagnosis, Royal commission, EMA, Patient, Treatment, Authorization, Research, Genentech, Disease, European Commission, Route of administration, PTC Therapeutics, Food and Drug Administration Safety and Innovation Act, Growth, Clinical trial, Novartis, USD, Medical device, Pharmaceutical industry, Nusinersen, Onasemnogene abeparvovec, Risdiplam

View original content:https://www.prnewswire.com/news-releases/spinal-muscular-atrophy-treatme...

Key Points: 
  • Spinal Muscular Atrophy Treatment Market Size, Share & Trends Analysis Report By Type (Type1, Type 2), By Treatment (Gene Therapy, Drug), By Drug (Spinraza, Zolgensma), By Route Of Administration, By Region, And Segment Forecasts, 2022 - 2030
    The global spinal muscular atrophy treatment market size is expected to reach USD 18.0 billion by 2030.
  • In March 2021, PTC Therapeutics, Inc., received marketing approval for Evrysdi (risdiplam) from the EMA for the treatment of patient with type1, 2, and 3 spinal muscular atrophy.
  • Thus, increasing number of product approvals is expected to drive the spinal muscular atrophy treatment market.
  • For instance, in July 2021, F. Hoffmann-La Roche Ltd launched Evrysdi (risdiplam) for the treatment of patient with spinal muscular atrophy in India
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Aetna launches Gene-Based, Cellular and Other Innovative Therapies™ (GCIT) designated network

Retrieved on: 
Wednesday, October 27, 2021
Safety, Case management, Genomics, Travel, Cellular, Onasemnogene abeparvovec, Patient, CVS Health, FDA, Pharmacist, NYSE, Chimeric antigen receptor T cell, MPH, Growth, Aetna, Environment, Lists of diseases, Health, CVS Caremark, Solution, Conditional sentence, Nursing, Retina, Heart, Pharmacy, Physician, Practitioner, Pharmaceutical industry, Medical device, Health insurance, Hospital

WOONSOCKET, R.I., Oct. 27, 2021 /PRNewswire/ --Aetna, a CVS Health company (NYSE CVS), announced the launch of its designated Gene-based, Cellular, and Other Innovative Therapies (GCIT) network.

Key Points: 
  • WOONSOCKET, R.I., Oct. 27, 2021 /PRNewswire/ --Aetna, a CVS Health company (NYSE CVS), announced the launch of its designated Gene-based, Cellular, and Other Innovative Therapies (GCIT) network.
  • This network is designed to enable members' access to new therapies that treat and potentially cure rare genetic diseases, while helping to manage the high cost of these therapies.
  • Through Aetna's designated GCIT network, members and customers benefit from:
    A comprehensive strategy to support access and manage the cost of current and future FDA-approved gene therapies.
  • A network of more than 75 designated GCIT service providers that have demonstrated quality and value in the delivery of GCIT services.

GenSight Biologics Appoints Françoise de Craecker to its Board of Directors

Retrieved on: 
Monday, May 17, 2021
Health, Genetics, General Health, Clinical trials, Pharmaceutical, Optical, Biotechnology, Life sciences, Branches of biology, Biology, Biotechnology, Molecular biology, Gene therapy, Biopharmaceutical, Retina, Onasemnogene abeparvovec

b"GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced the appointment of Fran\xc3\xa7oise de Craecker to its Board of Directors, replacing Natalie Mount.

Key Points: 
  • b"GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced the appointment of Fran\xc3\xa7oise de Craecker to its Board of Directors, replacing Natalie Mount.
  • She built from scratch the multifunctional team launching Zolgensma, the first one-shot Gene Therapy for the treatment of Spinal Muscular Atrophy, in the EMEA region.
  • She paved the way for the approval, distribution, market access and commercialization, working with authorities on innovative funding pathways and early access.
  • GenSight Biologics\xe2\x80\x99 pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases.

REGENXBIO Announces Agreement to Monetize Portion of Zolgensma® Royalties for $200 Million

Retrieved on: 
Tuesday, December 22, 2020
Branches of biology, Neurological disorders, Biology, Motor neuron diseases, Neurogenetic disorders, Biotechnology, Spinal muscular atrophy, Gene therapy, Onasemnogene abeparvovec, Covington & Burling, SMA, AveXis

If either cap is met, the royalty rights would revert to REGENXBIO.

Key Points: 
  • If either cap is met, the royalty rights would revert to REGENXBIO.
  • Zolgensma is currently approved for the treatment of Spinal Muscular Atrophy (SMA) in the United States, Japan, Europe, Brazil and Canada.
  • Morgan Stanley & Co. LLC served as sole structuring agent and Covington & Burling LLP served as counsel to REGENXBIO.
  • REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy.

Health Canada approves Zolgensma®, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA)1

Retrieved on: 
Wednesday, December 16, 2020
Neurological disorders, Neurodegenerative disorders, Branches of biology, Motor neuron diseases, Neuromuscular Disorders, Neurogenetic disorders, Onasemnogene abeparvovec, Gene therapy, Neuromuscular disease, SMN1, AveXis, Spinal muscular atrophies

In Canada each year, approximately one in 10,000 babies are born with SMA,a rare, genetic neuromuscular disease caused by a defective or missingSMN1gene3.

Key Points: 
  • In Canada each year, approximately one in 10,000 babies are born with SMA,a rare, genetic neuromuscular disease caused by a defective or missingSMN1gene3.
  • Zolgensma is the only gene therapy approved by Health Canada for the treatment of SMA1.
  • Advances and challenges in developing a therapy for spinal muscular atrophy.
  • Time Is Motor Neuron: Therapeutic Window and Its Correlation with Pathogenetic Mechanisms in Spinal Muscular Atrophy.