Gene delivery

Celyad Oncology to Present Data from Phase 1 IMMUNICY-1 Trial of Non-Gene Edited Allogeneic CAR T Candidate CYAD-211 in Relapsed/Refractory Multiple Myeloma at the European Hematology Association Virtual Congress

Retrieved on: 
Wednesday, May 12, 2021
Gene delivery, Biotechnology

We are encouraged by the observed clinical activity at such a low dose level and the overall steady progress of the trial to date.

Key Points: 
  • We are encouraged by the observed clinical activity at such a low dose level and the overall steady progress of the trial to date.
  • This non-gene editing technology, which does not permanently alter the genome integrity, is intended to decrease the potential safety risk associated with \xe2\x80\x9coff-target\xe2\x80\x9d genome modifications.
  • The trial will evaluate multiple dose levels of CYAD-211: 30x106, 100x106 and 300x106 cells per infusion.
  • The Company has received funding from the Walloon Region (Belgium) to support the advancement of its CAR T cell therapy programs.

Akouos Presents Nonclinical Data Supporting Future Clinical Development of AK-OTOF and AK-antiVEGF at the American Society of Gene and Cell Therapy 24th Annual Meeting

Retrieved on: 
Tuesday, May 11, 2021
Molecular biology, Otoferlin, Gene delivery, Vector, XC2

In Otof knockout mice, AK-OTOF administration results in durable expression of human otoferlin protein sufficient for sustained restoration of auditory function.

Key Points: 
  • In Otof knockout mice, AK-OTOF administration results in durable expression of human otoferlin protein sufficient for sustained restoration of auditory function.
  • These IND-enabling nonclinical studies are promising and support future clinical development.
  • AK-OTOF is designed to deliver normal OTOF by utilizing a dual vector approach,\xc2\xa0which encodes the 5\xe2\x80\x99 and the 3\xe2\x80\x99 components of OTOF.
  • A one-to-one ratio of the AK-OTOF component vectors appears to be optimal for efficient reconstitution of full-length human otoferlin.

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.

Gracell Biotechnologies Appoints Seasoned Gene & Cell Therapy Executive Dr. Jenny (Yajin) Ni as Chief Technology Officer

Retrieved on: 
Monday, May 10, 2021
Branches of biology, Biology, Medical specialties, Molecular genetics, Gene delivery, Vaccination, Vaccine, Virology, Viral vector

Dr. Ni will lead the efforts to ensure the smooth technology transfer to Gracell\'s strategic collaborator, Lonza, for manufacturing of FasTCAR-enabled product candidates in the U.S.\nDr.

Key Points: 
  • Dr. Ni will lead the efforts to ensure the smooth technology transfer to Gracell\'s strategic collaborator, Lonza, for manufacturing of FasTCAR-enabled product candidates in the U.S.\nDr.
  • Ni brings over 25 years of experience in process and product development for gene & cell therapies and vaccines to Gracell.
  • While at VIRxSYS, Dr. Ni was instrumental in bringing the first-ever lentiviral vector-modified autologous T cell product for HIV infection into clinical development.
  • Gracell\'s comprehensive manufacturing strategy serves as a great foundation for its preclinical and clinical development initiatives," said Dr. Jenny (Yajin) Ni.

Clearside Biomedical Announces Multiple Poster Presentations at the Association for Research in Vision and Ophthalmology 2021 Virtual Meeting

Retrieved on: 
Monday, May 10, 2021
Branches of biology, Biology, Gene delivery, Medical genetics, Emerging technologies, Molecular biology, Genetic engineering, Viral vector, AAV, Gene therapy, MYO7A

\xe2\x80\x9cThe analyses demonstrate the utility of our proprietary suprachoroidal space (SCS\xc2\xae) delivery platform to administer small molecules and gene therapy.

Key Points: 
  • \xe2\x80\x9cThe analyses demonstrate the utility of our proprietary suprachoroidal space (SCS\xc2\xae) delivery platform to administer small molecules and gene therapy.
  • However, adeno-associated viral vectors (AAV) cannot accommodate large genes such as Myo7A, limiting investigational gene therapy for Usher syndrome.
  • Preclinical results from Clearside and independent investigators have shown pharmacodynamic effects with reduced growth of experimental neovascularization and decreased fluorescein leakage.
  • These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements.

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'

Akouos to Present at Bank of America 2021 Virtual Healthcare Conference

Retrieved on: 
Friday, May 7, 2021
Branches of biology, Biology, Life sciences, Otorhinolaryngology, Emerging technologies, Molecular biology, Gene delivery, Biotechnology, Sensorineural hearing loss, Otology, AAV, Gene therapy

b'BOSTON, May 07, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (\xe2\x80\x9cAkouos\xe2\x80\x9d) (NASDAQ: AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that Manny Simons, Ph.D., M.B.A., co-founder, president, and chief executive officer of Akouos, will participate in a fireside chat at the Bank of America 2021 Virtual Healthcare Conference on Wednesday, May 12 at 2:00 p.m. EDT.\nA live webcast of the fireside chat will be available on the investors section of the company\xe2\x80\x99s website at www.akouos.com .

Key Points: 
  • b'BOSTON, May 07, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (\xe2\x80\x9cAkouos\xe2\x80\x9d) (NASDAQ: AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that Manny Simons, Ph.D., M.B.A., co-founder, president, and chief executive officer of Akouos, will participate in a fireside chat at the Bank of America 2021 Virtual Healthcare Conference on Wednesday, May 12 at 2:00 p.m. EDT.\nA live webcast of the fireside chat will be available on the investors section of the company\xe2\x80\x99s website at www.akouos.com .
  • To access the webcast, please go to the Akouos website approximately 15 minutes prior to the start time to ensure adequate time for any software downloads that may be required.
  • Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss.
  • 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'