Gene delivery

bluebird bio Receives EC Approval for SKYSONA™ (elivaldogene autotemcel, Lenti-D™) Gene Therapy for Patients Less Than 18 Years of Age With Early Cerebral Adrenoleukodystrophy (CALD) Without Matched Sibling Donor

Retrieved on: 
Wednesday, July 21, 2021

bluebird bio, Inc. (Nasdaq: BLUE) today announced that the European Commission (EC) has granted marketing authorization of SKYSONA™ (elivaldogene autotemcel, Lenti-D™), a one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA)-matched sibling hematopoietic (blood) stem cell (HSC) donor is not available. SKYSONA is the first one-time gene therapy approved in the European Union (EU) to treat CALD, a rare neurodegenerative disease that occurs in childhood and can rapidly lead to progressive, irreversible loss of neurologic function, and death.

Key Points: 
  • SKYSONA is a one-time gene therapy custom-designed to treat the underlying cause of the neurologic condition CALD.
  • SKYSONA uses ex vivo transduction with the Lenti-D lentiviral vector (LVV) to add functional copies of the ABCD1 gene into a patients own HSCs.
  • The addition of the functional ABCD1 gene allows patients to produce the ALD protein (ALDP), which is thought to facilitate the breakdown of VLCFAs.
  • The marketing authorization of SKYSONA is supported by efficacy and safety data from the Phase 2/3 Starbeam study (ALD-102).

Shape Therapeutics Secures $112 Million in Series B Financing to Advance Breakthrough RNA Technology Platforms to Democratize Gene Therapy

Retrieved on: 
Thursday, July 15, 2021

ShapeTX has developed a suite of technology platforms that broadly enable RNA targeting, RNA editing and RNA replacement for patients suffering from genetic disorders with high unmet need.

Key Points: 
  • ShapeTX has developed a suite of technology platforms that broadly enable RNA targeting, RNA editing and RNA replacement for patients suffering from genetic disorders with high unmet need.
  • Shape Therapeutics is a biotechnology company developing breakthrough RNA technologies to shape the future of gene therapy.
  • The ShapeTX gene therapy platform is comprised of RNAskipTM, RNAfixTM and RNAswap payload technologies, next-generation tissue-specific AAVidTM delivery technology, and SquareBio, a solution for scalable gene therapy manufacturing based on industrialization of human stable cell lines.
  • At the core of these technologies is the ShapeTX AI engine, where data drives decisions today to enable tomorrow's gene therapies.

GentiBio and Forge Biologics Announce Viral Vector Contract Development and GMP Manufacturing Partnership

Retrieved on: 
Thursday, July 15, 2021
Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20210715005550/en/
    Forge will provide adeno-associated viral (AAV) vector process and analytical development, scale-up engineering and GMP manufacturing services for GentiBios engineered Tregs therapeutic candidates.
  • All development and GMP activities will occur at The Hearth, Forges 175,000 ft2 gene therapy GMP production facility in Columbus, Ohio.
  • Forge offers a broad array of services ranging from process development to GMP manufacturing, making them a strong partner for GentiBios cell therapies with capacity to support GentiBio as we advance programs through clinical development.
  • To learn more, visit https://www.gentibio.com
    Forge Biologics is a hybrid gene therapy contract manufacturing and therapeutics development company.

Genespire appoints Philippe Mauberna as Chief Financial Officer

Retrieved on: 
Tuesday, July 13, 2021

Former Nanobiotix Chief Financial Officer brings extensive experience in international life sciences finance, successful fundraisings and operations

Key Points: 
  • Former Nanobiotix Chief Financial Officer brings extensive experience in international life sciences finance, successful fundraisings and operations
    Italy, Milan, 13 July 2021: Genespire, a next generation gene therapy company developing first-in-class transformative therapies with advanced lentiviral vectors, today announces the appointment of Philippe Mauberna, as Chief Financial Officer (CFO), effective immediately.
  • Mr. Mauberna joins a growing management team at Genespire and will lead financial operations as the company progresses the development of its novel gene therapies towards the clinic.
  • Mr. Mauberna is a highly experienced finance professional having worked in the life sciences industry for over 20 years in key financial and operational roles.
  • Philippe Mauberna, Chief Financial Officer of Genespire added: Genespire is one of the most promising and differentiated companies within the gene therapy space and I am delighted to be joining the company at such an exciting time.

All that Glitters is not Gold - A Discussion on the Stark Differences in "cGMP" Plasmid Manufacturing, Quality, and Regulations, Upcoming Webinar Hosted by Xtalks

Retrieved on: 
Monday, July 12, 2021

The regulatory guidelines are likely to become stricter as more products are approved.

Key Points: 
  • The regulatory guidelines are likely to become stricter as more products are approved.
  • The discussion will highlight the importance of the quality incorporated from early process development to a validated commercial production process.
  • Register for the webinar and be armed with critical information needed for your next plasmid DNA outsourcing decision.
  • For more information, or to register for this event, visit All that Glitters is not Gold A Discussion on the Stark Differences in "cGMP" Plasmid Manufacturing, Quality, and Regulations.

Atsena Therapeutics Appoints Jennifer Wellman to Board of Directors

Retrieved on: 
Monday, July 12, 2021

DURHAM, N.C., July 12, 2021 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced the appointment of Jennifer Wellman to its board of directors.

Key Points: 
  • DURHAM, N.C., July 12, 2021 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced the appointment of Jennifer Wellman to its board of directors.
  • Ms. Wellman, a biotech executive with more than 20 years of gene therapy development experience, will serve as an independent director.
  • Were thrilled to welcome Jen to Atsenas board of directors and look forward to leveraging her strategic insights.
  • Benjamin Yerxa, PhD, Chief Executive Officer of Foundation Fighting Blindness, has moved from Atsenas board of directors to its scientific advisory board.

Renovacor Announces the Appointment of Jiwen Zhang, Ph.D., as Senior Vice President, Regulatory Affairs and Quality Assurance

Retrieved on: 
Monday, July 12, 2021

I look forward to working with my new colleagues to advance REN-001 down its regulatory path and am thrilled to be part of the Renovacor team.

Key Points: 
  • I look forward to working with my new colleagues to advance REN-001 down its regulatory path and am thrilled to be part of the Renovacor team.
  • Dr. Zhang brings over 20 years of experience in regulatory affairs to Renovacor, and has over a decade of experience working specifically in the cell and gene therapy space.
  • She most recently served as vice president, head of regulatory affairs at Passage Bio, Inc., an AAV-based gene therapy company.
  • Neither Renovacor nor CHAQ gives any assurance that either Renovacor or CHAQ will achieve its expectations.

Generation Bio to Present at William Blair Biotech Focus Conference

Retrieved on: 
Thursday, July 8, 2021

CAMBRIDGE, Mass., July 08, 2021 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, announced today that Geoff McDonough, M.D., president and chief executive officer, will present at the William Blair Biotech Focus Conference 2021 on Thursday, July 15, at 9:00 a.m.

Key Points: 
  • CAMBRIDGE, Mass., July 08, 2021 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, announced today that Geoff McDonough, M.D., president and chief executive officer, will present at the William Blair Biotech Focus Conference 2021 on Thursday, July 15, at 9:00 a.m.
  • A live webcast of the presentation will be available on the investor section of the companys website at investors.generationbio.com .
  • Generation Biois an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases.
  • The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed.

Taysha Gene Therapies to Participate in Upcoming July Investor Healthcare Conference

Retrieved on: 
Thursday, July 8, 2021

A live webcast of this conference will be available in the Events & Media section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations .

Key Points: 
  • A live webcast of this conference will be available in the Events & Media section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations .
  • An archived version of the webcast will be available on the website for 60 days.
  • Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease.
  • We have combined our teams 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.

PharmaJet Partner Zydus Cadila Seeks EUA on World’s First Plasmid DNA COVID-19 Vaccine

Retrieved on: 
Wednesday, July 7, 2021

PharmaJet , the maker of innovative, needle-free injection technology, today announced that its partner Zydus Cadila has applied for Emergency Use Authorization (EUA) to the office of the Drug Controller General of India (DCGI) for its plasmid DNA Vaccine against COVID-19.

Key Points: 
  • PharmaJet , the maker of innovative, needle-free injection technology, today announced that its partner Zydus Cadila has applied for Emergency Use Authorization (EUA) to the office of the Drug Controller General of India (DCGI) for its plasmid DNA Vaccine against COVID-19.
  • The ZyCoV-D vaccine is the first ever plasmid DNA vaccine for human use and represents a key milestone in scientific innovation and technology advancement.
  • Chris Cappello, President and CEO, PharmaJet, said, We are pleased to be partnering with Zydus Cadila as they move forward with plans to commercialize the first ever DNA vaccine for COVID-19.
  • The PharmaJet Needle-free Systems provide increased vaccine effectiveness, a preferred patient and caregiver experience, and a proven path to commercialization.