Gene therapy

Outpace Bio, a Leader in Protein Design for Cell Therapy, Strengthens Executive Leadership Team with Key Appointments

Retrieved on: 
Thursday, May 4, 2023
Differentiable function, University, Biotechnology, Senior, IPSC, Center for Cell and Gene Therapy, Bone marrow, Baylor University, Cancer, Partner (business rank), Research, Infection, Bayer, Doctor of Philosophy, McKinsey & Company, Science, CAR, Rose–Hulman Institute of Technology, Acquisition, Patent, Cell, Drug discovery, Baylor College of Medicine, Cell engineering, Cardiology, Therapy, IND, Neurology, Gene therapy, Massachusetts Institute of Technology, Pharmaceutical industry

Seattle, WA, May 04, 2023 (GLOBE NEWSWIRE) -- Outpace Bio , a company using protein design and cell engineering to create advanced cell therapies, today announced two key leadership appointments.

Key Points: 
  • Seattle, WA, May 04, 2023 (GLOBE NEWSWIRE) -- Outpace Bio , a company using protein design and cell engineering to create advanced cell therapies, today announced two key leadership appointments.
  • The appointments bolster Outpace’s mission to develop technologies that overcome the barriers standing in the way of effective and safe cell therapies for cancer.
  • “We launched Outpace to unleash the full power of cell therapies by combining the groundbreaking fields of de novo protein design and cell engineering, and I'm thrilled that Aaron and Eric have joined us on this mission,” said Marc Lajoie, PhD , CEO and co-founder of Outpace.
  • “Cell therapies have shown tremendous benefits for blood cancers, but similar results for solid tumors have lagged behind” said Dr. Foster.

ISCT Reaffirms Position on Addressing Ethical Implications of US Expanded Access Pathway

Retrieved on: 
Thursday, May 4, 2023
Stem Cells, Biotechnology, FDA, Other Health, Health, General Health, Pharmaceutical, Research, Genetics, Science, Clinical Trials, ISCT, Expanded access, History, Terminal, Committee, Ethics, Patient, JD, Cell, Gene therapy, Safety, Pharmaceutical industry

Firstly, expanded access can delay or halt clinical trials so that data on the safety and efficacy of interventions is never generated.

Key Points: 
  • Firstly, expanded access can delay or halt clinical trials so that data on the safety and efficacy of interventions is never generated.
  • Speaking on the significance of the report, Patricia J. Zettler, JD, (Chair, ISCT Expanded Access Working Group) JD said, “We have seen a substantial increase in patient interest in non-trial access, which has spurred controversy and contention around the ethics behind expanded access.
  • To address these sector-wide concerns, ISCT formed the Expanded Access Working Group to identify practical, ethical, and regulatory issues emerging from the use - and possible abuse - of the expanded access pathway.
  • To learn more about the ISCT Expanded Access Working Group visit our website here .

Apertura Gene Therapy Announces Scientific Founder Presentations on AAV Capsid Engineering Platforms at ASGCT 2023 Annual Meeting

Retrieved on: 
Wednesday, May 3, 2023
Science, Biotechnology, Research, Pharmaceutical, Health, Engineering, Genetics, Manufacturing, Vector, Heart, Broad Institute, Capsid, Gene, MIT, Tropism, Harvard University, AAV, Doctor of Philosophy, Cross, Cell, Society, Apertura and Clausura, Gene therapy, Vaccine, Target

Dr. Deverman and members of the Vector Engineering Lab of the Broad Institute will present findings on the founding platforms on which Apertura is based.

Key Points: 
  • Dr. Deverman and members of the Vector Engineering Lab of the Broad Institute will present findings on the founding platforms on which Apertura is based.
  • Both the Fit4Function AAV (adeno-associated virus) capsid engineering platform and a platform approach to engineering AAV capsids targeting known receptors leverage machine learning to accelerate capsid identification and selection.
  • Members of the Vector Engineering Lab will deliver five oral presentations and two poster presentations on AAV capsid engineering optimization strategies to enhance genetic medicine tropism and delivery efficiency.
  • Details of the presentations are as follows:

CANbridge Announces Financial Results and Corporate Updates for Twelve Months Ended December 31, 2022

Retrieved on: 
Friday, March 31, 2023
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, IPO, Glycogen storage disease type II, American Society of Gene and Cell Therapy, Congress, Investigational New Drug, Patient, MG, Cell, IBAT, Enzyme replacement therapy, Fabry disease, DSM-IV codes, University of Massachusetts, Gene, Food, GD, Glioblastoma, Paroxysmal nocturnal hemoglobinuria, ESGCT, Rare, FDA, IND, Safety, COVID-19, MPS II, Myasthenia gravis, MPS, AAV, PNH, Alexion, Alagille syndrome, Hunter syndrome, Gene therapy, SMA, Liver disease, Clinical trial, NDA, Part, Catholic Medical Mission Board, ERT, Mouse, European Society for Primary Immunodeficiencies, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Society, Humanitarian use licenses, European Society of Gene and Cell Therapy, Pharmaceutical industry, Cryptocurrency, Vaccine, Fabry, C5

CANbridge Pharmaceuticals, Inc. (“CANbridge,” stock code 1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies to treat rare diseases and oncology, today announced financial results for the twelve months ended 2022 and corporate update.

Key Points: 
  • CANbridge Pharmaceuticals, Inc. (“CANbridge,” stock code 1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies to treat rare diseases and oncology, today announced financial results for the twelve months ended 2022 and corporate update.
  • The adjusted loss for the year decreased by RMB124.6 million, or 21.4%, from RMB581.3 million, for the year ended December 31, 2021, to RMB456.7 million for the year ended December 31, 2022.
  • R&D expense excluding milestone payments increased by RMB37.8 million, from RMB213.9 million for the year ended December 31, 2021 to RMB251.7 million for the year ended December 31, 2022.
  • CANbridge completed the full technology transfer of these gene therapy products in late 2022.

Advanced Therapy Medicinal Products CDMO Market is expected to display a steady growth of 14% due to the rise in research & development activities | UnivDatos Market Insights

Retrieved on: 
Tuesday, March 21, 2023
SMA, Oncology, Advanced Therapy Medicinal Product, Central nervous system, Heart failure, Prevalence, Disability, Biotechnology, Stroke, COVID-19, Infection, Cancer, ATMP, Research, AGC, DSM-IV codes, Fujifilm, CDMO, Genetic disorder, Thermo Fisher Scientific, Cardiology, Education minister, Human musculoskeletal system, Conditional sentence, Growth, B cell, Catalent, Death, Gene therapy, Wuxi, Pharmaceutical industry, Health care, NOIDA

The advanced therapy medicinal products CDMO market report has been aggregated by collecting informative data on various dynamics such as market drivers, restraints, and opportunities.

Key Points: 
  • The advanced therapy medicinal products CDMO market report has been aggregated by collecting informative data on various dynamics such as market drivers, restraints, and opportunities.
  • This innovative report makes use of several analyses to get a closer outlook on the advanced therapy medicinal products CDMO market.
  • The advanced therapy medicinal products CDMO market report offers a detailed analysis of the latest industry developments and trending factors in the market that are influencing the market growth.
  • Furthermore, this statistical market research repository examines and estimates the advanced therapy medicinal products CDMO market at the global and regional levels.

Advanced Therapy Medicinal Products CDMO Market is expected to display a steady growth of 14% due to the rise in research & development activities | UnivDatos Market Insights

Retrieved on: 
Tuesday, March 21, 2023
SMA, Oncology, Advanced Therapy Medicinal Product, Central nervous system, Heart failure, Prevalence, Disability, Biotechnology, Stroke, COVID-19, Infection, Cancer, ATMP, Research, AGC, DSM-IV codes, Fujifilm, CDMO, Genetic disorder, Thermo Fisher Scientific, Cardiology, Education minister, Human musculoskeletal system, Conditional sentence, Growth, B cell, Catalent, Death, Gene therapy, Wuxi, Pharmaceutical industry, Health care, NOIDA

The advanced therapy medicinal products CDMO market report has been aggregated by collecting informative data on various dynamics such as market drivers, restraints, and opportunities.

Key Points: 
  • The advanced therapy medicinal products CDMO market report has been aggregated by collecting informative data on various dynamics such as market drivers, restraints, and opportunities.
  • This innovative report makes use of several analyses to get a closer outlook on the advanced therapy medicinal products CDMO market.
  • The advanced therapy medicinal products CDMO market report offers a detailed analysis of the latest industry developments and trending factors in the market that are influencing the market growth.
  • Furthermore, this statistical market research repository examines and estimates the advanced therapy medicinal products CDMO market at the global and regional levels.

QurAlis Reveals Newest Program Targeting UNC13A RNA Mis-splicing Incorporating its FlexASO™ Platform

Retrieved on: 
Monday, March 20, 2023
Asos, DNA, FRCP, Gene, Therapeutic index, Episcopal conference, RNA, Gene therapy, Cytoplasm, HIV disease progression rates, Patient, Muscular Dystrophy Association, CST, Frontotemporal dementia, Synapse, ASO, MDA, ALS, FTD, Disease, Vaccine, UNC13A

CAMBRIDGE, Mass., March 20, 2023 /PRNewswire/ -- QurAlis Corporation, a clinical-stage biotechnology company developing breakthrough precision medicines for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases with genetically validated targets, today announced the launch of its newest program that targets UNC13A mis-splicing, a critical genetic alteration in neurodegenerative diseases like ALS and frontotemporal dementia (FTD). Incorporating its proprietary FlexASO™ Splice Modulator Platform, QurAlis' antisense oligonucleotides (ASOs) correct this mis-splicing, restore UNC13A protein production, and reduce cryptic exons that may contribute to disease progression. An exon is a segment of a DNA or RNA molecule containing information coding for a protein or peptide sequence.

Key Points: 
  • Incorporating its proprietary FlexASO™ Splice Modulator Platform, QurAlis' antisense oligonucleotides (ASOs) correct this mis-splicing, restore UNC13A protein production, and reduce cryptic exons that may contribute to disease progression.
  • An exon is a segment of a DNA or RNA molecule containing information coding for a protein or peptide sequence.
  • "We are excited to bring our UNC13A program out of stealth.
  • UNC13A is the third QurAlis program focused on a genetic target for the development of much-needed precision medicines for the sporadic ALS and FTD populations," said Kasper Roet, Ph.D., chief executive officer of QurAlis.

Generation Bio to Present at the 43rd Annual Cowen Healthcare Conference 

Retrieved on: 
Monday, February 27, 2023
Gene, Gene therapy, EST, Conference, Pharmaceutical industry, Broadcasting

CAMBRIDGE, Mass., Feb. 27, 2023 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq:GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, announced that Geoff McDonough, M.D., president and chief executive officer, will participate in the Gene Therapy panel discussion at the 43rd Annual Cowen Healthcare Conference on Monday, March 6th at 10:30 a.m. EST in Boston, MA.

Key Points: 
  • CAMBRIDGE, Mass., Feb. 27, 2023 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq:GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, announced that Geoff McDonough, M.D., president and chief executive officer, will participate in the Gene Therapy panel discussion at the 43rd Annual Cowen Healthcare Conference on Monday, March 6th at 10:30 a.m. EST in Boston, MA.
  • A live webcast of the panel will be available on the investor section of the company’s website at investors.generationbio.com.
  • A replay will be available there for 30 days following the event.

DNA Methylation Market Report 2022: Increasing Adoption of DNA Methylation Technology in Oncology Research Bolsters Growth - ResearchAndMarkets.com

Retrieved on: 
Monday, January 23, 2023
Other Health, Research, Pharmaceutical, Oncology, Medical Devices, Genetics, Clinical Trials, Science, Medical Supplies, FDA, Other Science, Health, DNA, Biomarker, Biotechnology, Gene therapy, Fred Hutchinson Cancer Research Center, NGS, PCR, DNA methylation, Patient, Conditional sentence, ACS, Alzheimer's disease, USD, American Cancer Society, Cancer, Esophageal cancer, Gene, Growth, PubMed, Prevalence, Death, Medical device, Fine chemical

The global DNA methylation market size is expected to reach USD 4.22 billion by 2030.

Key Points: 
  • The global DNA methylation market size is expected to reach USD 4.22 billion by 2030.
  • The market is expected to register a CAGR of 14.67% from 2022 to 2030, due to the increased usage of DNA methylation in sequencing analysis.
  • The increased adoption of DNA methylation in the agrigenomics field remains a promising driver of this growth.
  • The growing number of product launches, FDA approvals, and the presence of key market players remains a promising driver of growth in this region
    Chapter 1 DNA Methylation Market: Methodology And Scope
    Chapter 2 DNA Methylation Market: Executive Summary
    Chapter 3 DNA Methylation Market: Industry Outlook
    Chapter 4 DNA Methylation Market: Competitive Landscape
    Chapter 5 DNA Methylation Market: Technology Business Analysis

Breaking Down Barriers in Support of Patients with Rare Diseases, Upcoming Webinar Hosted by Xtalks

Retrieved on: 
Wednesday, February 1, 2023
MBA, Gene therapy, Scottish Informatics and Computer Science Alliance, RN, RAPT, Patient, Clinical trial, Scott Richter, Research, Labcorp Drug Development, Labcorp, Rare, Disease, Growth, MPH, Critical Path Institute, CCRC, Doctor of Philosophy, Pharmaceutical industry, Nursing, Science, MD

TORONTO, Feb. 1, 2023 /PRNewswire-PRWeb/ -- There are over 7,000 rare diseases, and one in 10 individuals worldwide have a rare disease. Over 675 diseases have some research and development targeting supportive care, treatment or a cure for these patients, with approximately only 5% of indications having a treatment available. There is tremendous opportunity and an even larger patient need.

Key Points: 
  • The featured speakers will discuss early scientific networking in a rare disease study by partnering with patient advocacy groups.
  • The speakers will share insights about engagement and retention of patients in clinical trials, expanded access programs and patient registries.
  • The speakers will also discuss Labcorp's strategy to overcome critical barriers with innovative solutions to efficiently conduct rare disease studies.
  • Ways to successfully collaborate with patients, families and advocacy groups
    Register to learn about breaking down barriers in support of patients with rare diseases.