Applied genetics

uniQure Presents HOPE-B Clinical Data Demonstrating Clinical Benefit in Hemophilia B Patients with Pre-existing Antibodies to AAV5 Vector

Retrieved on: 
Thursday, May 13, 2021
Branches of biology, Life sciences, Biology, CSL Behring, Applied genetics, Neutralizing antibody, Antibody, Gene therapy, Behring, Therapy

These data were previously presented at a medical meeting late last year.\n\xe2\x80\x9cThe ASGCT presentation highlights these initial HOPE-B data that demonstrate the successful treatment with an AAV5 gene therapy of patients with pre-existing NAbs,\xe2\x80\x9d stated Ricardo Dolmetsch , Ph.D., president of Research and Development at uniQure.

Key Points: 
  • These data were previously presented at a medical meeting late last year.\n\xe2\x80\x9cThe ASGCT presentation highlights these initial HOPE-B data that demonstrate the successful treatment with an AAV5 gene therapy of patients with pre-existing NAbs,\xe2\x80\x9d stated Ricardo Dolmetsch , Ph.D., president of Research and Development at uniQure.
  • \xe2\x80\x9cPatients in the trial who may not have been eligible for other gene therapies because of pre-existing neutralizing antibodies have achieved similar results with etranacogene dezaparvovec compared to those who did not have pre-existing NAbs.
  • Mean FIX activity at 26 weeks was 32.7 percent in participants with NAbs versus 41.3 percent in those without.
  • uniQure and CSL Behring have entered into a commercialization and license agreement providing CSL Behring exclusive global commercialization rights to etranacogene dezaparvovec.

Rocket Pharmaceuticals Presents Positive Clinical Data from Fanconi Anemia, Leukocyte Adhesion Deficiency-I, and Pyruvate Kinase Deficiency Programs at 24th Annual Meeting of the American Society of Gene and Cell Therapy

Retrieved on: 
Thursday, May 13, 2021
Biotechnology, Health, Genetics, Pharmaceutical, Clinical trials, Branches of biology, Biology, Applied genetics, Genetics, Gene delivery, Parvovirinae, Emerging technologies, Molecular biology, Adeno-associated virus, Danon disease, AAV, Gene therapy

All of these patients have been free of serious infections since hospital discharge following RP-L201 therapy.

Key Points: 
  • All of these patients have been free of serious infections since hospital discharge following RP-L201 therapy.
  • Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia.
  • Rocket\xe2\x80\x99s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Taysha Gene Therapies to Participate in Upcoming May Investor Healthcare Conferences

Retrieved on: 
Wednesday, May 12, 2021
Genetics, Health, Stem cells, Branches of biology, Life sciences, Biology, Biotechnology, Applied genetics, Genetic engineering, Gene delivery, Emerging technologies, Therapy, Gene therapy, Monogenic

b'Webcasts for these conferences will be available in the \xe2\x80\x9cEvents & Media\xe2\x80\x9d section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations .

Key Points: 
  • b'Webcasts for these conferences will be available in the \xe2\x80\x9cEvents & Media\xe2\x80\x9d 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.\nTaysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease.
  • With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside.
  • We have combined our team\xe2\x80\x99s 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.

Freeline to Participate at the 2021 RBC Capital Markets Global Healthcare Conference

Retrieved on: 
Wednesday, May 12, 2021
Applied genetics, Parvovirinae, Adeno-associated virus, Parvoviruses

b'LONDON, May 12, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the \xe2\x80\x9cCompany\xe2\x80\x9d or \xe2\x80\x9cFreeline\xe2\x80\x9d), a clinical-stage biotechnology company developing transformative AAV gene therapies for patients suffering from inherited systemic debilitating diseases, today announced that senior management will participate in a Fireside Chat at the virtual 2021 RBC Capital Markets Global Healthcare Conference on Tuesday, May 18, 2021 at 4:50 pm EDT.\nA webcast of the event will be available on the Investors section of the Freeline website.

Key Points: 
  • b'LONDON, May 12, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the \xe2\x80\x9cCompany\xe2\x80\x9d or \xe2\x80\x9cFreeline\xe2\x80\x9d), a clinical-stage biotechnology company developing transformative AAV gene therapies for patients suffering from inherited systemic debilitating diseases, today announced that senior management will participate in a Fireside Chat at the virtual 2021 RBC Capital Markets Global Healthcare Conference on Tuesday, May 18, 2021 at 4:50 pm EDT.\nA webcast of the event will be available on the Investors section of the Freeline website.
  • Senior management will also participate in virtual one-on-one meetings with investors at the conference.\nFreeline is a clinical-stage biotechnology company developing transformative adeno-associated virus (\xe2\x80\x9cAAV\xe2\x80\x9d) vector-mediated systemic gene therapies.
  • The Company is dedicated to improving patient lives through innovative, one-time treatments that provide functional cures for inherited systemic debilitating diseases.
  • The Company has clinical programs in Hemophilia\xc2\xa0B and Fabry disease, as well as preclinical programs in Gaucher disease and Hemophilia\xc2\xa0A.

Neurogene’s AAV Biodistribution Study Shows Route of Administration Essential Component in Optimizing Gene Therapy Treatment for Neurological Disease

Retrieved on: 
Tuesday, May 11, 2021
Biotechnology, Pharmaceutical, Genetics, Health, Branches of biology, Biology, Life sciences, Applied genetics, Gene delivery, Medical genetics, Gene therapy, Emerging technologies, Adeno-associated virus, Gene therapy of the human retina, Gene therapy for epilepsy

These results underscore that the route of delivery is an essential factor in developing effective treatments for genetic neurological diseases.

Key Points: 
  • These results underscore that the route of delivery is an essential factor in developing effective treatments for genetic neurological diseases.
  • The AAV9 product and the dose were controlled across the delivery groups.\nResults demonstrated that choice of delivery method has important implications for AAV9 biodistribution.
  • Our lead programs use adeno-associated virus (AAV) vector-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene.
  • Neurogene is also developing novel gene therapy technologies to advance treatments for complex neurological diseases that conventional gene therapy cannot successfully address.

Freeline Presents Data Highlighting Platform Technology at the American Society of Gene and Cell Therapy Annual Meeting 2021

Retrieved on: 
Tuesday, May 11, 2021
Branches of biology, Life sciences, Biology, Applied genetics, Gene delivery, Bioethics, Emerging technologies, Medical genetics, Gene therapy, Therapy

\xe2\x80\x9cCollectively, these posters demonstrate our potential to innovate across capsid, protein engineering, transduction efficiency, analytics and manufacturing, with the goal of delivering better gene therapy products for patients.

Key Points: 
  • \xe2\x80\x9cCollectively, these posters demonstrate our potential to innovate across capsid, protein engineering, transduction efficiency, analytics and manufacturing, with the goal of delivering better gene therapy products for patients.
  • The Company is dedicated to improving patient lives through innovative, one-time treatments that provide functional cures for inherited systemic debilitating diseases.
  • The Company\xe2\x80\x99s integrated gene therapy platform includes in-house capabilities in research, clinical development, manufacturing and commercialization.
  • For further information, please reference the Company\xe2\x80\x99s reports and documents filed with the U.S. Securities and Exchange Commission.

Orchard Therapeutics Announces New England Journal of Medicine Publication of HSC Gene Therapy Data for ADA-SCID

Retrieved on: 
Tuesday, May 11, 2021
Branches of biology, Biology, Life sciences, Gene delivery, Biotechnology, Medical genetics, Applied genetics, Emerging technologies, Gene therapy, Adenosine deaminase deficiency, Hematopoietic stem cell, Viral vector

Fifty (50) ADA-SCID patients were treated with investigational gene therapy composed of autologous CD34+ hematopoietic stem cells (HSCs) transduced ex\xc2\xa0vivo with a self-inactivating lentiviral vector (LVV) encoding the human ADA gene.

Key Points: 
  • Fifty (50) ADA-SCID patients were treated with investigational gene therapy composed of autologous CD34+ hematopoietic stem cells (HSCs) transduced ex\xc2\xa0vivo with a self-inactivating lentiviral vector (LVV) encoding the human ADA gene.
  • Results also showed sustained ADA gene expression, metabolic correction, and functional immune reconstitution in 48 out of the 50 patients.
  • Study patients in the UK received a very similar investigational HSC gene therapy product based on the same ADA LVV.
  • In 2018, Orchard acquired GSK\xe2\x80\x99s rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the\xc2\xa0San Raffaele Telethon Institute for Gene Therapy in Milan, Italy.

Vineti Introduces PTM Essentials™ to Accelerate Cell and Gene Therapy Clinical Trials with a New Digital Supply Chain Solution

Retrieved on: 
Monday, May 10, 2021
Branches of biology, Life sciences, Biology, Biotechnology, Gene delivery, Applied genetics, Bioethics, Medical genetics, Therapy, Gene therapy

The new clinical-phase solution, PTM Essentials\xe2\x84\xa2, delivers advanced therapy clinical studies quickly, safely, and simply with a turn-key supply chain management solution.\nThis pre-configured, pre-validated solution enables advanced therapy developers to rapidly initiate autologous clinical trials, automate data collection, and rely on a proven solution for next-generation patient safety.

Key Points: 
  • The new clinical-phase solution, PTM Essentials\xe2\x84\xa2, delivers advanced therapy clinical studies quickly, safely, and simply with a turn-key supply chain management solution.\nThis pre-configured, pre-validated solution enables advanced therapy developers to rapidly initiate autologous clinical trials, automate data collection, and rely on a proven solution for next-generation patient safety.
  • \xe2\x80\x9cOur new PTM Essentials\xe2\x84\xa2 solution will give cell and gene therapy developers the fast, reliable digital supply chain solution they require.
  • As the leader in cell and gene therapy supply chain orchestration technology, Vineti offers the only enterprise software platform supporting both clinical and commercial advanced therapies and knows first-hand what types of support these transformative clinical trials require.
  • The Vineti Personalized Therapy Management\xc2\xae (PTM) platform aligns and orchestrates the advanced therapy process and improves product performance overall.

Akouos Appoints Dr. Kathy Reape as Chief Development Officer

Retrieved on: 
Friday, May 7, 2021
Branches of biology, Biology, Life sciences, Emerging technologies, Gene delivery, Medical genetics, Molecular biology, Applied genetics, Actavis, Allergan, Gene therapy, Retina

b'BOSTON, May 07, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ: AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced the appointment of Dr. Kathy Reape as chief development officer.

Key Points: 
  • b'BOSTON, May 07, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ: AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced the appointment of Dr. Kathy Reape as chief development officer.
  • She also oversaw the development of Spark\xe2\x80\x99s pipeline of gene therapies addressing CNS disease, hemophilia, metabolic disorders, and inherited retinal dystrophies.
  • Prior to Spark, Dr. Reape was senior vice president of clinical development focusing on global brands research and development at Allergan and Actavis.
  • Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.\n'

REGENXBIO to Present at Upcoming Investor Conferences

Retrieved on: 
Thursday, May 6, 2021
Branches of biology, Biology, Applied genetics, Genetics, Medical genetics, Emerging technologies, Molecular biology, Adeno-associated virus, Parvoviruses, AAV, Gene therapy

b"ROCKVILLE, Md., May 6, 2021 /PRNewswire/ --REGENXBIO Inc. (Nasdaq: RGNX) today announcedit will present at the following May investor conferences, which will each be held in a virtual meeting format:\nA live webcast of each fireside chat can be accessed in the 'Investors' section of REGENXBIO's website at www.regenxbio.com .

Key Points: 
  • b"ROCKVILLE, Md., May 6, 2021 /PRNewswire/ --REGENXBIO Inc. (Nasdaq: RGNX) today announcedit will present at the following May investor conferences, which will each be held in a virtual meeting format:\nA live webcast of each fireside chat can be accessed in the 'Investors' section of REGENXBIO's website at www.regenxbio.com .
  • An archived replay of each webcast will be available on the same website for approximately 30 days following each fireside chat.\nREGENXBIOis 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.\n"