Gene delivery

Expanded Gene Therapy Portfolio Supports More Efficient Adeno-Associated Viral Manufacturing

Retrieved on: 
Wednesday, July 7, 2021
Branches of biology, Biology, Life sciences, Gene delivery, Genetic engineering, Applied genetics, Biotechnology, Thermo Fisher Scientific, Gene therapy

A new media panel, gene kit and advanced purification resins help reduce manufacturing costs while increasing the viability of gene therapies as treatment options for patients.

Key Points: 
  • A new media panel, gene kit and advanced purification resins help reduce manufacturing costs while increasing the viability of gene therapies as treatment options for patients.
  • "Despite the immense promise of gene therapy, robustness and yield continue to pose challenges in the manufacturing process," said Betty Woo, vice president and general manager, cell and gene therapy at Thermo Fisher Scientific.
  • "Growth media that maximizes viral titer and rapid analytical tests contribute to cost-effective and efficient AAV production.
  • Our expanding gene therapy portfolio is designed for a smooth ramp-up to clinical production and we have made focused investments to ensure we stay ahead of commercial demand.

Transine Therapeutics Appoints Industry Veteran Jan Thirkettle Ph.D. as CEO

Retrieved on: 
Monday, July 5, 2021
Life sciences, Biology, Articles, Biotechnology, Molecular biology, Gene delivery, Gene therapy, Biopharmaceutical

Dr Thirkettle has close to 25 years' experience of building cross-functional teams to drive delivery of innovative technologies.

Key Points: 
  • Dr Thirkettle has close to 25 years' experience of building cross-functional teams to drive delivery of innovative technologies.
  • Jan has built platforms and played a leadership role in the commercialisation of multiple therapeutics across small molecule, biopharmaceutical and gene therapy modalities.
  • Dr Thirkettle joins Transine from Freeline Therapeutics Holdings (NASDAQ: FRLN) where he was start-up CEO and more recently Chief Development Officer.
  • Combined with an outstanding team the Companyhas the opportunity to build an entire new class of therapeutic RNAs that can exquisitely modulate protein expression by interacting with the natural cellular translation machinery," said Dr Thirkettle, CEO of Transine Therapeutics.

Transine Therapeutics Appoints Industry Veteran Jan Thirkettle Ph.D. as CEO

Retrieved on: 
Monday, July 5, 2021
Life sciences, Biology, Articles, Biotechnology, Molecular biology, Gene delivery, Gene therapy, Biopharmaceutical

Dr Thirkettle has close to 25 years' experience of building cross-functional teams to drive delivery of innovative technologies.

Key Points: 
  • Dr Thirkettle has close to 25 years' experience of building cross-functional teams to drive delivery of innovative technologies.
  • Jan has built platforms and played a leadership role in the commercialisation of multiple therapeutics across small molecule, biopharmaceutical and gene therapy modalities.
  • Dr Thirkettle joins Transine from Freeline Therapeutics Holdings (NASDAQ: FRLN) where he was start-up CEO and more recently Chief Development Officer.
  • Combined with an outstanding team the Companyhas the opportunity to build an entire new class of therapeutic RNAs that can exquisitely modulate protein expression by interacting with the natural cellular translation machinery," said Dr Thirkettle, CEO of Transine Therapeutics.

uniQure Announces Presentations at Upcoming International Society on Thrombosis and Haemostasis (ISTH) Virtual Congress

Retrieved on: 
Friday, July 2, 2021
Branches of biology, Life sciences, Biology, Applied genetics, Gene delivery, Parvovirinae, Genetic engineering, Medical genetics, Adeno-associated virus, International Society on Thrombosis and Haemostasis, Therapy, Gene therapy

LEXINGTON, Mass.

Key Points: 
  • LEXINGTON, Mass.
  • and AMSTERDAM, The Netherlands, July 02, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that eight data presentations, of which three are oral presentations, will be delivered at the International Society on Thrombosis and Haemostasis (ISTH) Virtual Congress being held July 17-21, 2021.
  • His pre-recorded presentation, Rationale for Adeno-associated Virus (AAV) Mediated Gene Therapy in Patients with Pre-existing anti-AAV5 antibodies, will take place on Saturday, July 17 from 9:24 to 9:30 a.m.
    uniQure is delivering on the promise of gene therapy single treatments with potentially curative results.
  • www.uniQure.com

Everads Enters into Collaboration to Evaluate its Suprachoroidal Delivery Technology with a Clinical-Stage Gene Therapy Company

Retrieved on: 
Wednesday, June 30, 2021
Branches of biology, Biology, Articles, Emerging technologies, Gene delivery, Medical genetics, Molecular biology, Gene therapy, Genetic engineering, Retina

- Under new collaboration, partner given opt-in right to license specific retinal targets in exchange for pre-agreed license fees, development milestones and royalties

Key Points: 
  • - Under new collaboration, partner given opt-in right to license specific retinal targets in exchange for pre-agreed license fees, development milestones and royalties
    TEL AVIV, Israel, June 30, 2021 /PRNewswire/ -- Everads Therapy , a life sciences company developing a novel and differentiated suprachoroidal delivery technology for facilitating treatment of retinal diseases, announced today that a clinical-stage gene therapy company has entered into a collaboration to evaluate suprachoroidal delivery for its gene therapy pipeline programs using Everads' delivery platform.
  • This agreement is yet another indication of the attractiveness of our suprachoroidal delivery technology."
  • The technology was further developed and optimized via a collaboration with DALI Medical Devices , who specialize in injectable drug delivery solutions.
  • Everads Therapy is an ophthalmology-focused life sciences company whose goal is to restore vision of patients by enabling optimal treatment of debilitating retinal diseases via suprachoroidal delivery.

Neurogene Announces EMA Grants Orphan Drug Designation to CLN5 Batten Disease Gene Therapy 

Retrieved on: 
Tuesday, June 29, 2021
Biotechnology, FDA, Health, Genetics, Pharmaceutical, Branches of biology, Life sciences, Biology, Applied genetics, Gene delivery, Gene therapy, Emerging technologies, Medical genetics, Adeno-associated virus, Batten disease, Therapy, Gene therapy of the human retina, Orphan Drug Designation, CLN5, a subtype of Batten disease, Genetic Testing, Neurogene Inc., ORPHAN DRUG DESIGNATION, CLN5, A SUBTYPE OF BATTEN DISEASE, GENETIC TESTING, NEUROGENE INC.

Neurogene is collaborating with Batten disease experts, regulatory authorities, and caregivers to provide a safe and effective gene therapy for this disease as quickly as possible.

Key Points: 
  • Neurogene is collaborating with Batten disease experts, regulatory authorities, and caregivers to provide a safe and effective gene therapy for this disease as quickly as possible.
  • Applications for orphan designation are examined by the EMAs Committee for Orphan Medicinal Products (COMP), using the Committees network of experts.
  • 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.

Orchard Therapeutics Announces Regulatory and Clinical Updates for Lead Gene Therapy Programs

Retrieved on: 
Tuesday, June 29, 2021
Branches of biology, Life sciences, Health sciences, Bioethics, Gene delivery, Gene therapy, Genetic engineering, Therapy

ET

Key Points: 
  • ET
    BOSTONandLONDON, June 29, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics(Nasdaq: ORTX), a global gene therapy leader, today announced several program updates for the companys portfolio of approved and investigational hematopoietic stem cell (HSC) gene therapies.
  • ET to discuss recent regulatory and clinical updates for its lead programs.
  • Orchard Therapeuticsis a global gene therapy leader dedicated to transforming the lives of people affected by severe diseases through the development of innovative, potentially curative gene therapies.
  • In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy inMilan, Italy.

Calcium electroporation delivered with EndoVE to treat advanced Oesophageal Cancer

Retrieved on: 
Monday, June 28, 2021
Branches of biology, Biology, Natural sciences, Biotechnology, Gene delivery, Molecular biology, Calcium, Electroporation, Microbiology, Cancer, Calcium in biology

The trial aims to establish the safety and feasibility of treating patients with advanced oesophageal cancer in combination with local intratumoral injection of a calcium based solution.

Key Points: 
  • The trial aims to establish the safety and feasibility of treating patients with advanced oesophageal cancer in combination with local intratumoral injection of a calcium based solution.
  • Pulsed electrical fields generated by the ePORE and delivered directly to the tumour by the EndoVE endoscopic system results in electroporation of the cells and allows for passive diffusion of a locally injected calcium solution.
  • Compared to healthy cells, cancer cells are selectively less able to tolerate excessive intracellular calcium and results in their death.
  • Oesophageal cancer is the 7th most common cause of cancer morbidity and the 6th most common cause of cancer related death worldwide.

Adverum Announces Changes to Management Team

Retrieved on: 
Thursday, June 24, 2021
Branches of biology, Life sciences, Biology, Adverum Biotechnologies, Emerging technologies, Gene delivery, Medical genetics, Molecular biology, Therapy, Gene therapy

Dr. Fischer continued, Patient safety remains our top priority for everyone on the Adverum team.

Key Points: 
  • Dr. Fischer continued, Patient safety remains our top priority for everyone on the Adverum team.
  • Adverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases.
  • Risks and uncertainties facing Adverum are described more fully in Adverums Form 10-Q filed with theSEConMay 6, 2021under the heading Risk Factors.
  • Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

uniQure Announces Positive 52-Week Clinical Data from HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B and Provides Regulatory Update

Retrieved on: 
Tuesday, June 22, 2021
Branches of biology, Life sciences, Biology, Applied genetics, Gene delivery, Biotechnology, Bioethics, Gene therapy, Therapy, Adeno-associated virus, Spark Therapeutics, Jeanne Lusher

and AMSTERDAM, June 22, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive 52-week clinical data on all patients from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec , an investigational adeno-associated virus five ( AAV5 )-based gene therapy for the treatment of patients with severe and moderately severe hemophilia B.

Key Points: 
  • and AMSTERDAM, June 22, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive 52-week clinical data on all patients from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec , an investigational adeno-associated virus five ( AAV5 )-based gene therapy for the treatment of patients with severe and moderately severe hemophilia B.
  • These are the first clinical data to be reported from a Phase III gene therapy study in hemophilia B and, with 54 patients, the largest set of hemophilia B patients receiving a single gene therapy investigational product to date.
  • All patients in the HOPE-B pivotal study achieved steady-state FIX activity levels by 26-weeks after administration of etranacogene dezaparvovec.
  • The pivotal Phase III HOPE-B trial is a multinational, open-label, single-arm study to evaluate the safety and efficacy of etranacogene dezaparvovec.