Voretigene neparvovec

ALSA Ventures launches novel Gene Therapy portfolio company Axovia Therapeutics to treat Ciliopathies

Retrieved on: 
Tuesday, September 19, 2023
Tissue, University College London, UCL, PRV, BBS1, Research, Paratriathlon, Disease, AAV, BBS, Acquisition, ALSA, Patient, Syndrome, Vazgen Safarian, Ciliopathy, FDA, Therapy, Health, Pharmaceutical industry, Vaccine, Nursing, Child, Voretigene neparvovec

LONDON, Sept. 19, 2023 (GLOBE NEWSWIRE) -- ALSA Ventures , a UK-based, therapeutics-focused venture capital fund, announces the acquisition of Axovia Therapeutics Inc and the launch of a new portfolio company Axovia Therapeutics Ltd .

Key Points: 
  • LONDON, Sept. 19, 2023 (GLOBE NEWSWIRE) -- ALSA Ventures , a UK-based, therapeutics-focused venture capital fund, announces the acquisition of Axovia Therapeutics Inc and the launch of a new portfolio company Axovia Therapeutics Ltd .
  • Axovia is developing the first gene therapies for ciliopathies and has a pipeline of products for these devastating diseases, including Bardet-Biedl Syndrome (BBS).
  • AXV101 is an AAV9-based gene therapy targeting retinal dystrophy associated with BBS in patients carrying biallelic mutations in the BBS1 gene.
  • Axovia Acting CEO Professor Phil Beales said the Axovia gene therapy platform gives hope to BBS patients worldwide.

Global $70 Billion Regenerative Medicine Market Analysis to 2028 - ResearchAndMarkets.com

Retrieved on: 
Tuesday, May 16, 2023
Health, Pharmaceutical, GSK, Amgen, The Global Institute for Tomorrow, COVID-19, Bayer, Medicine, Infection, Food, Takeda Pharmaceutical Company, Cancer, Foreign Affairs, Paratriathlon, Multinational corporation, DSM-IV codes, Prostate, United States Department of Health and Human Services, AstraZeneca, Lung, Stomach, Colon, Regenerative medicine, Ministry, Death, GSK plc, Criticism of the Food and Drug Administration, Rectum, Patent, Patient, Immune system, Voretigene neparvovec, Conditional sentence, Growth, Integra LifeSciences, Region, FDA, Vericel, B cell, Ministry of Economics (Latvia), United Arab Emirates, Skin, World Health Organization, Novartis, Pharmaceutical industry, Vaccine, Video game, Economics, Research

The "Global Regenerative Medicine Market Size, Segments, Outlook, and Revenue Forecast 2022-2028 by Product Type, Material, Application, and Region" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Regenerative Medicine Market Size, Segments, Outlook, and Revenue Forecast 2022-2028 by Product Type, Material, Application, and Region" report has been added to ResearchAndMarkets.com's offering.
  • The Global Regenerative Medicine Market - which grew from approximately US$ 15 billion in 2017 to approximately US$ 30 billion in 2022 - is forecasted to grow further into approximately US$ 70 billion opportunities by 2028, owing to the growing severe diseases, infections, and cancers.
  • Regenerative medicine market is driven by surging trauma cases, genetic disorders, chronic diseases, and other infectious viruses.
  • The rising number of cancer patients worldwide is driving the growth of the regenerative medicine market.

Ocugen to Present at Association for Research in Vision and Ophthalmology 2023 Annual Meeting

Retrieved on: 
Thursday, April 20, 2023
Vaccine, Medicine, ARVO, Vance DeGeneres, Association for Research in Vision and Ophthalmology, New Orleans, DME, Stargardt disease, CSO, Retinitis pigmentosa, Voretigene neparvovec, Diabetic retinopathy, Doctor of Philosophy, Development, Patient, Macular degeneration, Treatment, The Association, News, Leber congenital amaurosis, Retina, Pharmaceutical industry, Stargardt, DR, Ophthalmology, Research

“We are thrilled to share more detail on our unique modifier gene therapy platform, as well as our novel biologics ophthalmic product pipeline with the professional community at ARVO,” commented Arun Upadhyay, PhD, Chief Scientific Officer and Head of Research, Development and Medical at Ocugen.

Key Points: 
  • “We are thrilled to share more detail on our unique modifier gene therapy platform, as well as our novel biologics ophthalmic product pipeline with the professional community at ARVO,” commented Arun Upadhyay, PhD, Chief Scientific Officer and Head of Research, Development and Medical at Ocugen.
  • “It is especially exciting to be at ARVO just following positive preliminary safety and efficacy results from the Phase 1/2 trial of OCU400 for the treatment of retinitis pigmentosa.
  • We look forward to highlighting this most recent news along with the work we are doing across our ophthalmology portfolio to combat hard-to-treat blindness diseases affecting millions of patients globally,” Dr. Upadhyay concluded.
  • Title: Modifier Gene Approach Using OCU410 for Dry AMD Therapy: One Gene—Multiple Targets
    Title: Ocugen—OCU400—Modifier Gene Therapy for Treatment of Inherited Retinal Diseases: Retinitis Pigmentosa & Leber Congenital Amaurosis
    Presenter: Arun Upadhyay, PhD, CSO and Head of Research, Development and Medical, Ocugen
    Title: Ocugen—OCU200—A Novel Biologic for the Treatment of DME, DR, and Wet AMD

Ocugen Announces Positive Preliminary Safety and Efficacy Results from the Phase 1/2 Trial of OCU400, a Modifier Gene Therapy Product Candidate, for the Treatment of Retinitis Pigmentosa and Leber Congenital Amaurosis

Retrieved on: 
Friday, April 14, 2023
Vaccine, Retina, Medicine, Schepens Eye Research Institute, CEP290, LCA, Cohort, CMC, Clinical trial, Retinitis pigmentosa, CanSino Biologics, Voretigene neparvovec, Doctor of Philosophy, RP, NR2E3, Development, Patient, Webcast, RHO, Leber congenital amaurosis, Safety, Pharmaceutical industry, Toy, Rhodopsin

“It is very gratifying to see these positive preliminary results from our novel modifier gene therapy approach,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen.

Key Points: 
  • “It is very gratifying to see these positive preliminary results from our novel modifier gene therapy approach,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen.
  • A total of 18 RP subjects have been enrolled in this study—10 subjects in the dose escalation and 8 subjects in the expansion phase, respectively.
  • We further expanded this Phase1/2 trial to enroll LCA patients with CEP290 gene mutation and pediatric patients with NR2E3, RHO and CEP290 mutations.
  • CanSinoBIO, Ocugen’s strategic partner, provided all CMC development and clinical supplies for the Phase 1/2 trial of OCU400.

VintaBio Unveils $64M to Back Viral Vector Trailblazers Behind the First Approved Cell and Gene Therapies

Retrieved on: 
Tuesday, April 11, 2023
Analytical chemistry, ILC Dover, University, Spinal muscular atrophy, Eli Lilly and Company, Thermo Fisher Scientific, Child, Doctor of Philosophy, Eli Lilly, CDMO, Patient, Voretigene neparvovec, Hospital, FDA, Children's Hospital of Philadelphia, Vaccine, Liu Junwei, Sanofi

VintaBio was founded by Junwei Sun and Dr. Shangzhen Zhou, who developed the first viral vectors used for life-saving therapies, including the cell therapy that cured Emily Whitehead of a pediatric cancer, as well as the first two FDA-approved gene therapies.

Key Points: 
  • VintaBio was founded by Junwei Sun and Dr. Shangzhen Zhou, who developed the first viral vectors used for life-saving therapies, including the cell therapy that cured Emily Whitehead of a pediatric cancer, as well as the first two FDA-approved gene therapies.
  • "I co-founded VintaBio to make sure viral vectors never prevent someone from receiving a life-changing treatment.
  • "With VintaBio, we now want to provide the ability to scale cutting-edge therapies for thousands more in-need patients."
  • Radspinner joins VintaBio from ILC Dover, a New Mountain Capital portfolio company, where he served as president for the organization's biotherapeutics portfolio.

Avirmax CMC Inc. Successfully Manufactured and Delivered to a Client the First Lot of rAAV Drug Product for a Monkey Toxicology Study

Retrieved on: 
Thursday, December 15, 2022
Process, Toxicology, Knowledge, Cros, QA, HEK293, Rita Raave, VG, Public health, ACI, Documentation, CGMP, California Department, Vaccine, Alipogene tiparvovec, Voretigene neparvovec

The manufacturing process was conducted in ACI's cGMP facility, recently licensed by California Department of Public Health (CDPH)-Food & Drug Branch, for the production of rAAV drug products for use in clinical investigations.

Key Points: 
  • The manufacturing process was conducted in ACI's cGMP facility, recently licensed by California Department of Public Health (CDPH)-Food & Drug Branch, for the production of rAAV drug products for use in clinical investigations.
  • Release testing of the rAAV product were performed in-house and by selected contract research organizations (CROs) in the U.S., thus speeding up the product release process.
  • The manufacturing platform contains orthogonal virus clearance technologies to ensure product virological safety.
  • It offers its clients with great transparency and flexible services with solid process development expertise, skills and knowledge in rAAV manufacturing, testing and quality management.

Amwins Group Benefits Launches Gene Therapy Solutions Program Exclusively Available Through Stealth Partner Group

Retrieved on: 
Monday, October 10, 2022
Risk management, SMA, Gene, Program, Food and Drug Administration, Voretigene neparvovec, Food, TPA, Face, FDA, Severe cognitive impairment, Solution, Consultant, Medical device, Insurance, Amwin

CHARLOTTE, N.C., Oct. 10, 2022 /PRNewswire/ -- Amwins Group Benefits released its new Gene Therapy Solutions program exclusively available through Stealth Partner Group, an Amwins company. This new solution when coupled with stop-loss coverage, provides a sustainable, revolutionary strategy that prepares employers to face unexpected, high-dollar gene therapy claims with confidence.

Key Points: 
  • CHARLOTTE, N.C., Oct. 10, 2022 /PRNewswire/ -- Amwins Group Benefits released its new Gene Therapy Solutions program exclusively available through Stealth Partner Group , an Amwins company.
  • "Our goal is to help employer groups navigate the future of gene therapy while providing unrivaled knowledge and expertise to help mitigate their financial risk," said Harley Barnes, Jr., co-chief executive officer at Stealth Partner Group.
  • "We're launching the Gene Therapy Solutions program to empower employers as they implement smart, practical and complete risk management programs and strategies for their employees."
  • Stealth Partner Group, an Amwins Company, was founded in 2009 and has grown to be one of the largest specialized general agencies in the country.

Macular Degeneration Treatment Global Market Report 2022: Increasing Burden of Retinal Disorders to Bolster Sector Growth - ResearchAndMarkets.com

Retrieved on: 
Friday, September 9, 2022
Optical, Health, Other Health, Novartis, Retina, Ageing, American Academy, Severe cognitive impairment, RPE65, Disease, Man, Administration, Route of administration, Macular degeneration, Optogenetics, Prevalence, Spark Therapeutics, Therapy, Voretigene neparvovec, AMD, Woman, Intermediate, Retinopathy, Growth, Eye, Acquisition, Patient, Pharmaceutical industry

The "Macular Degeneration Treatment Market Global Market Report 2022" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Macular Degeneration Treatment Market Global Market Report 2022" report has been added to ResearchAndMarkets.com's offering.
  • In 2021, North America will be the largest region in the macular degeneration treatment market.
  • The increasing burden of retinal disorders is expected to propel the growth of the macular degeneration treatment market.
  • Therefore, the increasing burden of retinal disorders is driving the growth of the macular degeneration treatment market.

AscellaHealth Family of Companies is Founding Sponsor of Inaugural Gene Therapy and Specialty Pharmacy Awareness Month, September 2022

Retrieved on: 
Tuesday, August 30, 2022
Managed Care, Software, Other Health, Pharmaceutical, Data Management, Oncology, Technology, Hospitals, Genetics, Clinical Trials, Practice Management, Health, Diabetes, Health care, CEO, Specialty pharmacy, Awareness, SHSMD, SMA, Disease, Solution, US Foods, Food, Company, Gene therapy, Disorder, Calendar, Thalassemia, FDA, Therapy, American Hospital Association, Industry, Society, Patient, Pharmaceutical industry, Voretigene neparvovec

The AscellaHealth Family of Companies , including AscellaHealth, a global healthcare and specialty pharmacy solutions company, and subsidiaries Optime Care and Terebellum , announces its founding sponsorship of the inaugural Gene Therapy and Specialty Pharmacy Awareness Month, September 2022, which was accepted for inclusion in the annual Calendar of Health Observances & Recognition Days, published by The Society for Health Care Strategy & Market Development (SHSMD) of the American Hospital Association .

Key Points: 
  • The AscellaHealth Family of Companies , including AscellaHealth, a global healthcare and specialty pharmacy solutions company, and subsidiaries Optime Care and Terebellum , announces its founding sponsorship of the inaugural Gene Therapy and Specialty Pharmacy Awareness Month, September 2022, which was accepted for inclusion in the annual Calendar of Health Observances & Recognition Days, published by The Society for Health Care Strategy & Market Development (SHSMD) of the American Hospital Association .
  • In time, the gene and cell therapy market is expected to venture beyond targeting only rare conditions, says Belazi.
  • At AscellaHealth, we have introduced unique financial solutions that address concerns of access and affordability.
  • The AscellaHealth Family of Companies , comprised of AscellaHealth and subsidiaries Optime Care and Terebellum , is a global Healthcare and Specialty Pharmacy solutions organization serving specialty and rare disease patients, payers, life sciences manufacturers and providers with solutions addressing the unmet needs in this dynamic marketplace.

Gene Therapy Global Market Research Report 2022 by Gene Type (Antigen; Cytokine; Suicide Gene; Others) & Vector (Viral Vector; Non-Viral Vector) - ResearchAndMarkets.com

Retrieved on: 
Friday, June 17, 2022
Health, Genetics, Prostate, Population, COVID-19, Spinal muscular atrophy, American Cancer Society, Degenerative disease, Intelligence, Female, Gene, Artificial intelligence, Vector, Lists of diseases, Cancer, Pressure, Hospital, Growth, Infection, Disease, National Health Service, RMIT University, Lung, Machine learning, Neoplasm, Cell death, Application, Texas Homecare, Organization, Voretigene neparvovec, AI, Breast cancer, Medical device, Pharmaceutical industry, Vaccine

The "Gene Therapy Global Market Report 2022: By Gene, By Vector, By Application, By End-User" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Gene Therapy Global Market Report 2022: By Gene, By Vector, By Application, By End-User" report has been added to ResearchAndMarkets.com's offering.
  • The gene therapy market consists of sales of gene therapy related services by entities (organizations, sole traders and partnerships) that manufacture gene therapy drugs.
  • The main types of gene therapy are antigen, cytokine, suicide gene and others.
  • The high prices of gene therapy medicines are expected to limit the growth of the gene therapy market.