Applied genetics

New adaptable nanoparticle platform enables enhanced delivery of gene therapies, RCSI research

Retrieved on: 
Wednesday, June 9, 2021
Branches of biology, Biology, Life sciences, Molecular biology, Medical genetics, Applied genetics, Biotechnology, Gene delivery, Gene therapy, Draft:Kevin Vance Morris

The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo.

Key Points: 
  • The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo.
  • A major challenge for gene therapies is preparing them in a way that can deliver the genetic information into the host cells.
  • The researchers developed a platform that produces bespoke star-shaped polypeptide nanoparticles, which effectively deliver a range of therapies, including gene therapies.
  • "With the success of the COVID-19 vaccines, the potential of gene therapies is becoming apparent, and advanced nanoparticle delivery systems are key to enabling their use clinically.

Bayer to Advance Two First-of-its-Kind Investigational Cell and Gene Therapies for Parkinson’s Disease

Retrieved on: 
Tuesday, June 8, 2021
Research, Genetics, Clinical trials, Biotechnology, Health, Pharmaceutical, General Health, Other Science, Science, Branches of biology, Life sciences, Biology, Applied genetics, Gene delivery, Emerging technologies, Medical genetics, Molecular biology, Therapy, Adeno-associated virus, Gene therapy, R. Jude Samulski, Bayer, AskBio, BlueRock Therapeutics, BAYER, ASKBIO, BLUEROCK THERAPEUTICS

In parallel a gene therapy investigational program also targeted at providing advanced therapies for Parkinsons disease is driven forward by Bayers wholly-owned clinical-stage adeno-associated virus (AAV) gene therapy company Asklepios BioPharmaceutical Inc. (AskBio).

Key Points: 
  • In parallel a gene therapy investigational program also targeted at providing advanced therapies for Parkinsons disease is driven forward by Bayers wholly-owned clinical-stage adeno-associated virus (AAV) gene therapy company Asklepios BioPharmaceutical Inc. (AskBio).
  • The potential of BlueRock and AskBios clinical candidates to treat Parkinsons disease and truly help patients with their high unmet medical need could be immense, said Wolfram Carius, Head of Cell and Gene Therapy at Bayer.
  • Parkinsons disease is the most common neurodegenerative movement disorder, impacting more than 10 million people worldwide.
  • By targeting the disease at its root cause, cell and gene therapies aim to go beyond symptomatic treatments.

FDA confirms NDA for Oleogel-S10 will not require an Advisory Committee Meeting

Retrieved on: 
Monday, June 7, 2021
Branches of biology, Biology, Life sciences, Applied genetics, Medical genetics, Biotechnology, Genetic disorder, Gene therapy

Amryts pre-clinical gene therapy platform, AP103, offers a potential treatment for patients with Dystrophic EB, and is also potentially relevant to other genetic disorders.

Key Points: 
  • Amryts pre-clinical gene therapy platform, AP103, offers a potential treatment for patients with Dystrophic EB, and is also potentially relevant to other genetic disorders.
  • Shore Capital (EdwardMansfield, Daniel Bush, John More) are NOMAD and Joint Broker to Amryt in the UK.
  • These statements are not guarantees of future performance or the ability to identify and consummate investments.
  • Such forward-looking statements reflect the Companys current beliefs and assumptions and are based on information currently available to management.

Genocea Presents Promising Long-term Results from GEN-009 Neoantigen Vaccine Phase 1 Trial at ASCO 2021

Retrieved on: 
Friday, June 4, 2021
Branches of biology, Medical specialties, Biology, Biotechnology, Vaccination, Applied genetics, Genomics, Medical genetics, Antigen, Vaccine, Individualized cancer immunotherapy, Mutanome

The poster is available for on-demand viewing on the ASCO website and also posted to the Scientific Resources section of the Genocea website here .

Key Points: 
  • The poster is available for on-demand viewing on the ASCO website and also posted to the Scientific Resources section of the Genocea website here .
  • Long-term results demonstrate that GEN-009 continues to generate broad immune responses against neoantigens that can lead to sustained clinical responses.
  • Six of the eight patients continue without recurrence with a median follow up of 25 months post start of the vaccination.
  • Notably, as previously reported, GEN-009 elicited T cell immune responses to 99% of the ATLAS-selected neoantigens, the highest seen across neoantigen vaccine programs.

Oyster Point Pharma Announces Preclinical Study Results and Pipeline Expansion with Enriched Tear Film (ETF™) Gene Therapy to Target Ocular Surface Diseases

Retrieved on: 
Thursday, June 3, 2021
Branches of biology, Biology, Life sciences, Applied genetics, Gene delivery, Medical genetics, Genetic engineering, Molecular biology, Adeno-associated virus, Gene therapy, Therapy, Nerve growth factor

ETF Gene Therapy is a proprietary adeno-associated virus (AAV) based gene therapy approach where a target gene is delivered to human lacrimal gland cells via intralacrimal gland injection.

Key Points: 
  • ETF Gene Therapy is a proprietary adeno-associated virus (AAV) based gene therapy approach where a target gene is delivered to human lacrimal gland cells via intralacrimal gland injection.
  • The goal for this target gene is to produce a selected gene product to change cell behavior and function on the ocular surface.
  • The goal for this target gene is to produce a gene product to change cell behavior and function on the ocular surface.
  • OC-101 (AAV-NGF) is Oyster Points investigational gene therapy in development as part of Oyster Points proprietary Enriched Tear Film (ETF) Gene Therapy pipeline expansion.

D. Wade Walke, Ph.D. Joins 23andMe as Vice President of Investor Relations

Retrieved on: 
Wednesday, June 2, 2021
Finance, Money, Applied genetics, 23andMe, Biotechnology companies, Investor relations, Ionis, Institutional Investor, IR

SUNNYVALE, Calif., June 2, 2021 /PRNewswire/ -- 23andMe Inc., a leading consumer genetics and research company, today announced that D. Wade Walke, Ph.D. will join the company as Vice President of Investor Relations.

Key Points: 
  • SUNNYVALE, Calif., June 2, 2021 /PRNewswire/ -- 23andMe Inc., a leading consumer genetics and research company, today announced that D. Wade Walke, Ph.D. will join the company as Vice President of Investor Relations.
  • He also brings a strong network of relationships with buy-side and sell-side healthcare analysts and institutional investors."
  • Prior to joining 23andMe, Walke spent nine years at Ionis Pharmaceuticals, a leading company in RNA-targeted drug discovery and development, where he most recently served as Vice President of Investor Relations.
  • From there, he pivoted to lead Communications and Investor Relations for the company, where he oversaw the implementation of a targeted program of IR activities.

LogicBio Therapeutics to Participate in Upcoming Investor Conferences

Retrieved on: 
Tuesday, June 1, 2021
Gene editing, Biology, Medical genetics, Emerging technologies, Molecular biology, Life sciences, Applied genetics, Articles, Biotechnology

LEXINGTON, Mass., June 1, 2021 /PRNewswire/ --LogicBio Therapeutics, Inc.(Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood, today announced that Frederic Chereau, chief executive officer of LogicBio, will participate in virtual fireside chats at the following upcoming investor conferences:

Key Points: 
  • LEXINGTON, Mass., June 1, 2021 /PRNewswire/ --LogicBio Therapeutics, Inc.(Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood, today announced that Frederic Chereau, chief executive officer of LogicBio, will participate in virtual fireside chats at the following upcoming investor conferences:
    Jefferies Virtual Healthcare Conference, Friday, June 4,at 9:30 AM ET.
  • JMP Securities Life Sciences Conference, Wednesday, June 16, at 2:00 PM ET.
  • LogicBio Therapeuticsis a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood.
  • LogicBio's cutting-edge sAAVy capsid development platform is designed to support development of treatments in a broad range of indications and tissues.

Scout Bio to Present at Stifel 2021 Virtual Jaws & Paws Conference

Retrieved on: 
Thursday, May 27, 2021
Life sciences, Biology, Branches of biology, Biotechnology, Medical genetics, Applied genetics, Gene delivery, Molecular biology, Gene therapy, Biopharmaceutical

PHILADELPHIA, May 27, 2021 (GLOBE NEWSWIRE) -- Scout Bio today announced that Mark Heffernan, Chief Executive Officer, will present at the upcoming Stifel 2021 Virtual Jaws & Paws Conference on Thursday, June 3, 2021 at 3:00 p.m.

Key Points: 
  • PHILADELPHIA, May 27, 2021 (GLOBE NEWSWIRE) -- Scout Bio today announced that Mark Heffernan, Chief Executive Officer, will present at the upcoming Stifel 2021 Virtual Jaws & Paws Conference on Thursday, June 3, 2021 at 3:00 p.m.
  • Scout Bio is a biotechnology company revolutionizing pet medicine by delivering a deep 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.

Generation Bio to Present at the Jefferies Virtual Healthcare Conference

Retrieved on: 
Thursday, May 27, 2021
Branches of biology, Biology, Life sciences, Biotechnology, Applied genetics, Gene delivery, Medical genetics, Gene therapy

CAMBRIDGE, Mass., May 27, 2021 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, announced today that Geoff McDonough, M.D., president and chief executive officer, will present at the Jefferies Virtual Healthcare Conference on Thursday, June 3, at 11:30 a.m.

Key Points: 
  • CAMBRIDGE, Mass., May 27, 2021 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, announced today that Geoff McDonough, M.D., president and chief executive officer, will present at the Jefferies Virtual Healthcare Conference on Thursday, June 3, at 11:30 a.m.
  • A live webcast of the presentation will be available on the investor section of the companys website at investors.generationbio.com .
  • Generation Biois an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases.
  • The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed.

Taysha Gene Therapies to Participate in Upcoming June Investor Healthcare Conferences

Retrieved on: 
Wednesday, May 26, 2021
Biotechnology, Pharmaceutical, Genetics, Health, Branches of biology, Life sciences, Biology, Biotechnology, Molecular biology, Applied genetics, Gene delivery, Emerging technologies, Therapy, Monogenic, Gene therapy

Webcasts for these conferences will be available in the Events & Media section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations .

Key Points: 
  • Webcasts for these conferences will be available in the Events & Media section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations .
  • Archived versions of the webcasts will be available on the website for 60 days.
  • Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease.
  • We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications.