Adeno-associated virus

Cure Genetics and Frametact Reach a $60 Million Collaboration and Licensing Agreement for the Development of Gene Therapy for Familial Neurological Diseases

Retrieved on: 
Thursday, September 14, 2023
Huntington's disease, Boehringer Ingelheim, Adeno-associated virus, Genetics, Alzheimer's disease, Partnership, Parkinson's disease, Myenteric plexus, AAV, DSM-IV codes, Pharmaceutical industry

The partnership will leverage Cure Genetics' proprietary VELP™ platform to develop novel Adeno-Associated Virus (AAV) vectors for the treatment of familial neurological diseases.

Key Points: 
  • The partnership will leverage Cure Genetics' proprietary VELP™ platform to develop novel Adeno-Associated Virus (AAV) vectors for the treatment of familial neurological diseases.
  • According to the agreement, Cure Genetics will receive an upfront and milestone payment totaling $60 million.
  • This collaboration will leverage the extensive expertise of Frametact in the domain of neurological diseases, combined with Cure Genetics' leading technology in AAV vector development and efficient in vivo AAV screening.
  • Cure Genetics looks forward to working with Frametact to accelerate the development of gene therapy products for brain diseases.

Culture Biosciences Appoints Darcy Birse as Chief Commercial Officer

Retrieved on: 
Wednesday, September 13, 2023
San Francisco, Business development, Partnership, Advisory board, Stockholm University, Biotechnology, Business, Doctor of Philosophy, CCO, Marketing, Assistant, Bachelor of Science, AAV, Globally Executable MHP, EMBO, Dalhousie University, CST, Structural biology, Cell, Biophysics, Growth, Adeno-associated virus, Culture, SVP, Management, Fine chemical, Biochemistry, Chemistry, Physics

South San Francisco, Sept. 13, 2023 (GLOBE NEWSWIRE) -- Culture Biosciences , a leading provider of innovative, future-forward bioprocessing solutions, is pleased to announce the appointment of Darcy Birse as the company's new Chief Commercial Officer (CCO).

Key Points: 
  • South San Francisco, Sept. 13, 2023 (GLOBE NEWSWIRE) -- Culture Biosciences , a leading provider of innovative, future-forward bioprocessing solutions, is pleased to announce the appointment of Darcy Birse as the company's new Chief Commercial Officer (CCO).
  • Darcy will lead the company's commercial strategy and drive growth initiatives to further strengthen Culture's position as a leader in the bioprocessing industry.
  • With a career spanning over 30 years in the life sciences sector, Darcy brings a wealth of global experience in Commercial Leadership, Marketing, Sales and Business Development to Culture Biosciences.
  • "As we continue to expand our presence in the bioprocessing market and deliver novel solutions to our clients, we are thrilled to welcome Darcy Birse to our executive team," said Will Patrick, CEO and Co-founder of Culture Biosciences.

Global Viral Vector & Plasmid DNA Manufacturing Market Report 2022: Facility Expansion for Cell and Gene Therapies Presents Opportunities - ResearchAndMarkets.com

Retrieved on: 
Tuesday, September 20, 2022
Genetics, Health Technology, Health, Observation, Porter's five forces analysis, Immortalised cell line, COVID-19, RNA interference, Downstream processing, Risk, Global Viral, Adeno-associated virus, Geography, Adenoviridae, Growth, Ansoff Matrix, Industry, Market development, Sense (molecular biology), Bioprocess, USD, Workflow, Infection, Cell therapy, SWOT, Genetic disorder, CAGR, Vaccine, Transfection, Lentivirus, Application, Purification, Vaccine, Cancer

The Global Viral Vector & Plasmid DNA Manufacturing Market is segmented based on Cell Line, Type, Transfection, Indication, Workflow, Application, and Geography.

Key Points: 
  • The Global Viral Vector & Plasmid DNA Manufacturing Market is segmented based on Cell Line, Type, Transfection, Indication, Workflow, Application, and Geography.
  • The report presents a detailed Ansoff matrix analysis for the Global Viral Vector & Plasmid DNA Manufacturing Market.
  • The Global Viral Vector & Plasmid DNA Manufacturing Market is analyzed using the Ansoff Matrix to provide the best approaches a company can take to improve its market position.
  • The report offers a comprehensive evaluation of the Global Viral Vector & Plasmid DNA Manufacturing Market.

Oxford Biomedica Solutions signs agreement with new partner to deliver The Full Solution for their AAV programs

Retrieved on: 
Wednesday, September 14, 2022
Lentivirus, AAV, Adeno-associated virus, IND, OXB, Partnership, Patient, LSE, CEO, CTAS, Chair, Leadership, Solution, Oxford BioMedica, Biotechnology, 104th Street station (IND Fulton Street Line), Vaccine, Group

The agreement will provide The Full Solution from construct design to process development to IND support, including a proven platform for multiple constructs supporting its new partners gene therapy programs.

Key Points: 
  • The agreement will provide The Full Solution from construct design to process development to IND support, including a proven platform for multiple constructs supporting its new partners gene therapy programs.
  • Tim Kelly, Chair and CEO of Oxford Biomedica Solutions said We are excited to offer the full breadth of our capabilities and expertise to partner new and unique programs that have the potential to positively impact more patients lives.
  • This partnership demonstrates Oxford Biomedica Solutions leadership in the delivery of high-quality and high-titer AAV vectors for gene therapies.
  • Oxford Biomedica Solutions T: +1 215 680 4733 / E: [email protected]
    Oxford Biomedica Solutions offers a plug and play platform that is capable of achieving both high titer and high product quality vector for partners.

LEXEO Therapeutics Receives Rare Pediatric Disease Designation and Orphan Drug Designation for LX1004 for the Treatment of CLN2 Batten Disease

Retrieved on: 
Wednesday, July 28, 2021
Clinical medicine, Lipid storage disorders, Branches of biology, Rare diseases, Medicine, Batten disease, Lysosomal storage disease, Adeno-associated virus

Both designations granted to LX1004 underscore the critical importance and urgency to advance new treatment approaches for CLN2 Batten disease, a fatal genetic disorder affecting the central nervous system (CNS), said R. Nolan Townsend, Chief Executive Officer of LEXEO Therapeutics.

Key Points: 
  • Both designations granted to LX1004 underscore the critical importance and urgency to advance new treatment approaches for CLN2 Batten disease, a fatal genetic disorder affecting the central nervous system (CNS), said R. Nolan Townsend, Chief Executive Officer of LEXEO Therapeutics.
  • The FDA grants Rare Pediatric Disease designation for serious and life-threateningdiseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the U.S.
  • CLN2 Batten disease is an autosomal recessive lysosomal storage disease with approximately 1,000 cases worldwide.
  • LEXEOs current pipeline consists of adeno-associated virus (AAV)-mediated gene therapies in rare cardiac diseases, CLN2 Batten disease, and APOE4-associated Alzheimers disease.

Capsida Biotherapeutics Unveils Next-Generation Gene Therapy Manufacturing

Retrieved on: 
Thursday, July 22, 2021
Branches of biology, Parvovirinae, Adeno-associated virus, Parvoviruses, Gene therapy, AAV, Biology, Genetics, Oncolytic AAV

As a fully integrated gene therapy company, Capsida is unique in its end-to-end expertise which includes AAV engineering, cargo development and optimization, translational biology, early capsid manufacturability, clinical development, and state-of-the-art manufacturing.

Key Points: 
  • As a fully integrated gene therapy company, Capsida is unique in its end-to-end expertise which includes AAV engineering, cargo development and optimization, translational biology, early capsid manufacturability, clinical development, and state-of-the-art manufacturing.
  • "One of our key strategic priorities at Capsida is to create therapies that can be manufactured consistently and rapidly while maintaining the highest quality," said Rayne Waller, chief manufacturing officer of Capsida Biotherapeutics.
  • "Our new manufacturing facility eliminates the dependence on contract manufacturing which will allow us to bring products seamlessly from discovery into clinical studies faster.
  • Capsida Biotherapeutics Inc. is developing tissue-targeted gene therapies using its biologically driven, high-throughput adeno-associated virus (AAV) engineering and proprietary cargo development platform.

Global Vectorized Antibodies for In Vivo Expression Market - by DNA and mRNA - ResearchAndMarkets.com

Retrieved on: 
Tuesday, July 13, 2021
Health, Genetics, Branches of biology, Medical specialties, Biology, Antibodies, Immunology, Glycoproteins, Recombinant antibodies, Biotechnology, Molecular biology, Synthetic antibody, Adeno-associated virus

Sales of recombinant therapeutic antibodies in the year 2020 exceeded US$ 184 bln (+11% vs previous year).

Key Points: 
  • Sales of recombinant therapeutic antibodies in the year 2020 exceeded US$ 184 bln (+11% vs previous year).
  • Manufacturing of recombinant antibodies requires large volumes, costly production and complex protein characterization.
  • Each of the three different approaches (AAV DNA, plasmid DNA and mRNA) for in vivo expression of antibodies has its specific profile of advantages and disadvantages.
  • This report evaluates the industry landscape of antibody vectorization with optimized technologies for direct in vivo delivery of synthetic nucleic acid-encoded antibodies.

LogicBio Therapeutics to Participate in William Blair Biotech Focus Conference 2021

Retrieved on: 
Thursday, July 8, 2021
Branches of biology, Biology, Applied genetics, Adeno-associated virus, Parvovirinae, Parvoviruses, Emerging technologies, Jean Bennett, Gene therapy of the human retina

LEXINGTON, Mass., July 8, 2021 /PRNewswire/ --LogicBio Therapeutics, Inc.(Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood, today announced that Frederic Chereau, chief executive officer of LogicBio, will participate in a virtual panel discussion entitled "Delivery and Durability of Genetic Medicines" at the William Blair Biotech Focus Conference 2021 on Thursday, July 15, 2021 at 10:00 a.m.

Key Points: 
  • LEXINGTON, Mass., July 8, 2021 /PRNewswire/ --LogicBio Therapeutics, Inc.(Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood, today announced that Frederic Chereau, chief executive officer of LogicBio, will participate in a virtual panel discussion entitled "Delivery and Durability of Genetic Medicines" at the William Blair Biotech Focus Conference 2021 on Thursday, July 15, 2021 at 10:00 a.m.
  • LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering gene editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood.
  • The Company's second platform, sAAVy, is an adeno-associated virus (AAV) capsid engineering platform designed to optimize gene delivery for treatments in a broad range of indications and tissues.
  • For more information, visit www.logicbio.com , which does not form a part of this release.

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

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.