Applied genetics

DTx Pharma to Present at Credit Suisse 2021 Healthcare Innovators Series and Viva Biotech Partnership Summit

Retrieved on: 
Thursday, February 25, 2021
Nucleic acids, Oligonucleotide, Biotechnology, Biology, Life sciences, Applied genetics, Molecular biology, Academic disciplines

DTx Pharma, Inc. is a privately-held biotechnology companycreating novel RNA-based therapeutics to treat the genetic drivers of disease.

Key Points: 
  • DTx Pharma, Inc. is a privately-held biotechnology companycreating novel RNA-based therapeutics to treat the genetic drivers of disease.
  • The company's proprietary delivery technology platform, FALCON (Fatty Acid Long Chain OligoNucleotide), utilizes fatty acids as targeting ligands to enable the delivery of oligonucleotide therapies to tissues and cell types throughout the body.
  • In preclinical studies, DTx has demonstrated cellular uptake and broad activity of oligonucleotides in the retina, muscle, heart, neurons, T cells and many other specialized cell types.
  • To learn more about DTx Pharma,please visit www.dtxpharma.com and follow DTx on Twitter and LinkedIn @DTxPharma.

Selecta Biosciences Announces Science Advances Publication Highlighting Potential Potency and Durability Benefits of ImmTOR™ in Gene Therapy

Retrieved on: 
Wednesday, February 24, 2021
Branches of biology, Life sciences, Health sciences, Applied genetics, Gene delivery, Genetic engineering, Immunology, Medicinal chemistry, Therapy, Gene therapy, Immunogenicity, AAV

These findings underscore the promise of Selectas ImmTOR platform to address current limitations, notably immunogenicity, safety and durability, of gene therapy.

Key Points: 
  • These findings underscore the promise of Selectas ImmTOR platform to address current limitations, notably immunogenicity, safety and durability, of gene therapy.
  • The results in this publication continue to expand our knowledge of and validate the benefits of co-administering ImmTOR with AAV gene therapies, said Carsten Brunn, Ph.D., president and chief executive officer of Selecta.
  • We look forward to continuing to investigate the benefits of adding ImmTOR to gene therapy in clinical studies this year.
  • Selecta Biosciences Inc. (NASDAQ: SELB) is leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses.

REGENXBIO to Participate in Upcoming Investor Conferences

Retrieved on: 
Wednesday, February 24, 2021
Branches of biology, Biology, Applied genetics, Genetics, Gene delivery, Medical genetics, Emerging technologies, Molecular biology, Adeno-associated virus, AAV, Gene therapy

ROCKVILLE, Md., Feb. 24, 2021 /PRNewswire/ --REGENXBIO Inc. (Nasdaq: RGNX) today announcedit will participate in the following March investor conferences, which will each be held in a virtual meeting format:

Key Points: 
  • ROCKVILLE, Md., Feb. 24, 2021 /PRNewswire/ --REGENXBIO Inc. (Nasdaq: RGNX) today announcedit will participate in the following March investor conferences, which will each be held in a virtual meeting format:
    Live webcasts of the Raymond James and Barclays fireside chats can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com .
  • An archived replay of the webcasts will be available on the same website for approximately 30 days following the presentation.
  • REGENXBIOis a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy.REGENXBIO'sNAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10.REGENXBIOand its third-party NAVTechnology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

SIRION Biotech Announces Collaboration with Sanofi to Innovate Gene Therapy Treatments with Improved Adeno-Associated Virus Capsids

Retrieved on: 
Tuesday, February 23, 2021
Research, Other Health, General Health, Professional services, Pharmaceutical, Venture capital, Genetics, Science, Biotechnology, Stem cells, FDA, Health, Other Science, Branches of biology, Biology, Genetics, Applied genetics, Gene delivery, Genetic engineering, Biotechnology, Medical genetics, Viral vector, Adeno-associated virus, Gene therapy, Oncolytic AAV

SIRION Biotech GmbH , a world leader in viral vector-based gene delivery technologies for gene & cell therapy, announced today that it signed a license and collaboration agreement with Sanofi, a global biopharmaceutical company, to develop improved tissue-selective adeno-associated virus (AAV) vectors to realize effective gene therapy treatments for disorders affecting major human organs.

Key Points: 
  • SIRION Biotech GmbH , a world leader in viral vector-based gene delivery technologies for gene & cell therapy, announced today that it signed a license and collaboration agreement with Sanofi, a global biopharmaceutical company, to develop improved tissue-selective adeno-associated virus (AAV) vectors to realize effective gene therapy treatments for disorders affecting major human organs.
  • AAV vectors are a promising and clinically validated gene delivery platform for the potential treatment of a variety of human diseases.
  • The goal of this collaboration is to develop new and modified AAV capsids that exhibit a safe product profile with improved specificity and higher gene delivery efficiency.
  • SIRION Biotech was founded in 2005 with the goal to spark a new generation of viral vector technologies for gene and cell therapy as well as vaccine development.

Catalent to Acquire Delphi Genetics and Launch US Plasmid Manufacturing Site to Establish Global pDNA Development and Manufacturing Capabilities

Retrieved on: 
Tuesday, February 23, 2021
Branches of biology, Biology, Life sciences, Gene delivery, Molecular biology, Biotechnology, Catalent, Applied genetics, Bioproduction, Plasmid, Gene therapy

Catalent is also announcing the launch of pDNA development and manufacturing services at its Rockville, Maryland facility.

Key Points: 
  • Catalent is also announcing the launch of pDNA development and manufacturing services at its Rockville, Maryland facility.
  • Together, the addition of pDNA technology and production capabilities marks a significant milestone for Catalent Cell & Gene Therapy.
  • Plasmid DNA is a key component in most gene therapy and gene-enabled cell therapy production processes.
  • Founded in 2001 as a spin off from the Universit libre de Bruxelles (ULB), Delphi Genetics is a leading bioproduction CDMO with capabilities in handling the entire plasmid DNA development and CGMP manufacturing process.

Bionano Publishes Method for Identifying Genes Likely to Cause Neurological Diseases Based on a Model Developed by Lineagen to Improve Interpretation of Variants of Unknown Significance

Retrieved on: 
Monday, February 22, 2021
Branches of biology, Genetics, Applied genetics, Cancer screening, Genes, Genetic epidemiology, Variant of uncertain significance, Genetic counseling

The resulting list of high scoring genes enhances Lineagens clinical interpretation capabilities allowing their genetic counsellors to better interpret variants of unknown significance (VUS), which can improve diagnostic abilities and provide for more precise counseling to patients and families.

Key Points: 
  • The resulting list of high scoring genes enhances Lineagens clinical interpretation capabilities allowing their genetic counsellors to better interpret variants of unknown significance (VUS), which can improve diagnostic abilities and provide for more precise counseling to patients and families.
  • The gene scoring model can also be integrated into Bionanos analysis software for Saphyr data, allowing scientists and clinicians to quickly identify and filter structural variants based on their increased risk of causing CNS diseases.
  • Armed with NeuroSCORE, scientists could discover novel genes not previously associated with CNS diseases, potentially enabling clinicians to more accurately diagnose patients with previously uncharacterized neurodevelopmental disease.
  • Despite this progress, many thousands of genes are poorly characterized and their function unknown.

Scout Bio to Present at the BofA Securities 2021 Animal Health Summit

Retrieved on: 
Thursday, February 18, 2021
Life sciences, Health sciences, Biology, Genetic engineering, Applied genetics, Biopharmaceutical, Pharmaceutical industry, Pharmacy, Gene therapy, Biotechnology, Medicine, Therapy

PHILADELPHIA, Feb. 18, 2021 (GLOBE NEWSWIRE) -- Scout Bio,a biotechnology company revolutionizing pet medicine by delivering a pipeline of one-time gene therapies for major chronic pet health conditions, today announced that Mark Heffernan, Scout Bios Chief Executive Officer, will present at the upcoming BofA Securities 2021 Animal Health Summit on Thursday, February 25, 2021 at 2:35 p.m.

Key Points: 
  • PHILADELPHIA, Feb. 18, 2021 (GLOBE NEWSWIRE) -- Scout Bio,a biotechnology company revolutionizing pet medicine by delivering a pipeline of one-time gene therapies for major chronic pet health conditions, today announced that Mark Heffernan, Scout Bios Chief Executive Officer, will present at the upcoming BofA Securities 2021 Animal Health Summit on Thursday, February 25, 2021 at 2:35 p.m.
  • Scout Bio is a biotechnology company revolutionizing pet medicine by delivering a pipeline of one-time gene therapies for major chronic pet health conditions.
  • Scout Bios therapeutics are designed to induce long-term expression of therapeutic proteins in pet patients using AAV vector technology.
  • Scout Bio has a research and development collaboration with the University of Pennsylvanias Gene Therapy Program, a leader in the field of genetic medicine research.

Selecta Biosciences and AskBio Initiate First-in-Human Dose-Escalation Study to Evaluate ImmTOR™ in Gene Therapy

Retrieved on: 
Wednesday, February 17, 2021
Branches of biology, Life sciences, Biology, Applied genetics, Molecular biology, Health policy, Medicinal chemistry, Therapy, Gene therapy

The trial aims to determine the optimal dose of ImmTOR to mitigate the formation of antibodies to AAV8 capsids used in gene therapies.

Key Points: 
  • The trial aims to determine the optimal dose of ImmTOR to mitigate the formation of antibodies to AAV8 capsids used in gene therapies.
  • Data from this study will inform the design of future clinical trials in patients as we seek to unlock the full potential of gene therapy.
  • The dose-escalation trial of SEL-399 is designed to evaluate the safety and preliminary efficacy of ImmTOR in gene therapy.
  • Selecta Biosciences Inc. (NASDAQ: SELB) is leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses.

Forge Biologics Receives FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Designations for FBX-101 Gene Therapy for Patients with Krabbe Disease

Retrieved on: 
Tuesday, February 16, 2021
Life sciences, Branches of biology, Health sciences, Biotechnology, Gene delivery, Rare diseases, Applied genetics, Lipid storage disorders, Krabbe disease, Gene therapy, Therapy, Biopharmaceutical

Forge is now actively recruiting patients for enrollment in the RESKUE phase 1/2 clinical trial of FBX-101, a novel, first-in-human AAV gene therapy for the disease.

Key Points: 
  • Forge is now actively recruiting patients for enrollment in the RESKUE phase 1/2 clinical trial of FBX-101, a novel, first-in-human AAV gene therapy for the disease.
  • FBX-101 is the first intraveniousgene therapy program for patients with Krabbe disease and marks a major step forward in building out the company's hybrid model as a gene therapy manufacturing and development engine.
  • "FDA's decision to grant these designations to our first-in-human investigational gene therapy highlights the urgency of developing a treatment for Krabbe patients," said Timothy J. Miller, Ph.D., CEO, President and Co-Founder of Forge Biologics.
  • "Krabbe is a devastating disease, and it is imperative to develop treatment options like FBX-101 that may address all manifestations of the disease."

Worldwide Biobanking Industry to 2027 - High Growth Potential in Developing Countries

Retrieved on: 
Monday, February 15, 2021
Biological databases, Applied genetics, Biorepository, Branches of biology, Applied sciences, Biobanks, Academic disciplines, Tumor Bank, Biological specimen

Biobank is a biorepository meant to collect and preserve biological materials that are used for diagnosis, biodiversity studies, and research.

Key Points: 
  • Biobank is a biorepository meant to collect and preserve biological materials that are used for diagnosis, biodiversity studies, and research.
  • The rise in funding by private & government organizations for biobanking and increase in application areas of bio banked samples are the major factors that drive the growth of the global biobanking market.
  • However, ethical issues related to biobanking and lack of awareness about it are expected to impede the market growth.
  • Furthermore, government is taking multiple initiatives to support regenerative medicine research, which is expected to boost the market growth.