Parvovirinae

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.

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

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.

Capsida Appoints Swati Tole, M.D., as Chief Medical Officer

Retrieved on: 
Wednesday, May 12, 2021
Parvovirinae, Adeno-associated virus, Parvoviruses, University of California, San Francisco, Genentech, AAV, Companies, Life sciences, California

b'THOUSAND OAKS, Calif., May 12, 2021 /PRNewswire/ --Capsida Biotherapeutics Inc., a biotechnology company creating a new class of targeted gene therapies using its next-generation adeno-associated virus (AAV) engineering and cargo development platform, today announced the appointment of Swati Tole, M.D., as the Company\'s first chief medical officer.

Key Points: 
  • b'THOUSAND OAKS, Calif., May 12, 2021 /PRNewswire/ --Capsida Biotherapeutics Inc., a biotechnology company creating a new class of targeted gene therapies using its next-generation adeno-associated virus (AAV) engineering and cargo development platform, today announced the appointment of Swati Tole, M.D., as the Company\'s first chief medical officer.
  • Tole comes to Capsida from Genentech where she spent 13 years in roles of increasing responsibility, most recently as vice president of clinical development.
  • She earned her medical degree from the University of California, San Francisco (UCSF) and an M.S.
  • Tole said, "I\'m thrilled to join Capsida and to be a part of the growth of such a transformative company.

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.

AskBio Announces Presentations at Upcoming American Society of Gene and Cell Therapy's 24th Annual Meeting

Retrieved on: 
Thursday, May 6, 2021
Gene therapy, Parvovirinae

"\nDetails on AskBio\'s presentations and presence at ASGCT include:\nTim Kelly, AskBio\'s President of Manufacturing, will participate in a panel discussion, "Considerations for the Clinical Development of Cell & Gene Therapies."

Key Points: 
  • "\nDetails on AskBio\'s presentations and presence at ASGCT include:\nTim Kelly, AskBio\'s President of Manufacturing, will participate in a panel discussion, "Considerations for the Clinical Development of Cell & Gene Therapies."
  • The panel discussion among industry leaders will take place on Tuesday, May 11, 2021 from 6:00 p.m.
  • AskBio\'s gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library.
  • AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.\nView original content to download multimedia: http://www.prnewswire.com/news-releases/askbio-announces-presentations-a...\n'

LEXEO Therapeutics Announces FDA Fast Track Designation Granted to LX1001 for the Treatment of APOE4 Associated Alzheimer’s Disease

Retrieved on: 
Tuesday, April 20, 2021
Apolipoproteins, Parvovirinae, Adeno-associated virus, Parvoviruses, Apolipoprotein E, Branches of biology, AAV

b"NEW YORK, April 20, 2021 (GLOBE NEWSWIRE) -- LEXEO Therapeutics , a clinical-stage gene therapy company, today announced that the U.S. Food and Drug Administration\xc2\xa0(FDA) has granted Fast Track designation to LX1001, the company\xe2\x80\x99s adeno-associated virus (AAV) mediated gene therapy program, for the potential treatment of apolipoprotein E4 (APOE4) associated Alzheimer\xe2\x80\x99s disease.

Key Points: 
  • b"NEW YORK, April 20, 2021 (GLOBE NEWSWIRE) -- LEXEO Therapeutics , a clinical-stage gene therapy company, today announced that the U.S. Food and Drug Administration\xc2\xa0(FDA) has granted Fast Track designation to LX1001, the company\xe2\x80\x99s adeno-associated virus (AAV) mediated gene therapy program, for the potential treatment of apolipoprotein E4 (APOE4) associated Alzheimer\xe2\x80\x99s disease.
  • APOE is the major transporter of cholesterol in the brain with prevailing evidence suggesting that it is a major genetic risk factor in the pathogenesis of the disease.
  • The study will assess the safety and toxicity of LX1001, and establish a maximum tolerable dose.
  • LEXEO Therapeutics\xe2\x80\x99 pipeline consists of adeno-associated virus (AAV)-mediated therapies primarily developed at Weill Cornell Medicine\xe2\x80\x99s Department of Genetic Medicine.

REGENXBIO To Present at the Chardan 5th Annual Genetic Medicines Manufacturing Summit

Retrieved on: 
Wednesday, April 21, 2021
Adeno-associated virus, Parvovirinae, Parvoviruses, AAV

b"ROCKVILLE, Md., April 21, 2021 /PRNewswire/ --REGENXBIO Inc. (Nasdaq: RGNX) today announced that Curran M. Simpson, Chief Operations and Technology Officer, will present at the Chardan 5th Annual Genetic Medicines Manufacturing Summit on Tuesday, April 27, 2021 at 3:15 p.m.

Key Points: 
  • b"ROCKVILLE, Md., April 21, 2021 /PRNewswire/ --REGENXBIO Inc. (Nasdaq: RGNX) today announced that Curran M. Simpson, Chief Operations and Technology Officer, will present at the Chardan 5th Annual Genetic Medicines Manufacturing Summit on Tuesday, April 27, 2021 at 3:15 p.m.
  • The conference will be held in a virtual meeting format.\nA webcast of the fireside chat can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com .
  • REGENXBIO's NAV 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.
  • REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.\n"

Rocket Pharmaceuticals Reports Positive Long-Term Clinical Data from RP-L201 Trial for the Treatment of Leukocyte Adhesion Deficiency-I at the Clinical Immunology Society 2021 Annual Meeting

Retrieved on: 
Wednesday, April 14, 2021
Health, Genetics, Clinical trials, Research, Science, Pharmaceutical, Biotechnology, Rare diseases, Parvovirinae, Branches of biology, Adeno-associated virus, Parvoviruses, Danon disease, AAV, Clinical medicine, Applied sciences, Leukocyte Adhesion Deficiency-I, Rocket Pharmaceuticals, Inc.

LAD-I leads to recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics, require frequent hospitalizations and are ultimately fatal.

Key Points: 
  • LAD-I leads to recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics, require frequent hospitalizations and are ultimately fatal.
  • In all patients treated, CD18 expression has substantially exceeded the 4-10% threshold associated with survival into adulthood, with consistent peripheral blood vector copy number levels.
  • 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.