Gene delivery

Gyroscope Therapeutics Postpones Initial Public Offering

Retrieved on: 
Friday, May 7, 2021

b'Gyroscope Therapeutics Holdings plc (\xe2\x80\x9cGyroscope\xe2\x80\x9d), a clinical-stage gene therapy company focused on treating diseases of the eye, today announced it has postponed plans for its initial public offering.\n"In light of market conditions, we have decided to postpone our planned initial public offering," said Khurem Farooq, Chief Executive Officer.

Key Points: 
  • b'Gyroscope Therapeutics Holdings plc (\xe2\x80\x9cGyroscope\xe2\x80\x9d), a clinical-stage gene therapy company focused on treating diseases of the eye, today announced it has postponed plans for its initial public offering.\n"In light of market conditions, we have decided to postpone our planned initial public offering," said Khurem Farooq, Chief Executive Officer.
  • In the meantime, we are continuing to advance our clinical program for our investigational gene therapy, GT005, as well as our earlier stage pipeline.
  • "\nGyroscope Therapeutics is a clinical-stage gene therapy company developing gene therapy beyond rare disease to treat diseases of the eye that cause vision loss and blindness.
  • These securities may not be sold nor may offers to buy be accepted prior to the time the registration statement becomes effective.

Catalent Acquires Additional Facility at its Gosselies, Belgium Campus for Commercial-Scale Plasmid DNA Manufacturing

Retrieved on: 
Thursday, May 6, 2021

This new facility is located on Catalent\'s existing campus in Gosselies, adjacent to the Delphi Genetics building.

Key Points: 
  • This new facility is located on Catalent\'s existing campus in Gosselies, adjacent to the Delphi Genetics building.
  • This investment allows Catalent to support these programs from development through to CGMP commercial production, offering the full horizontal supply chain," said Manja Boerman, Ph.D., President, Catalent Cell & Gene Therapy.
  • She added, "The Center of Excellence we have established in Belgium brings critical experience and expertise together on one campus, allowing for key synergies to best support our customers.
  • With deep expertise in viral vector development, scale-up and manufacturing for gene therapies, Catalent is a full-service partner for plasmid DNA, adeno-associated viral (AAV), lentiviral and other viral vectors, oncolytic viruses, live virus vaccines and cell therapies.

Locanabio Announces Upcoming Preclinical Data Presentations at American Society of Gene and Cell Therapy Annual Meeting

Retrieved on: 
Wednesday, May 5, 2021

b'SAN DIEGO, May 5, 2021 /PRNewswire/ --Locanabio, Inc., an RNA-targeting gene therapy company focused on developing life-changing therapies for patients with severe neurodegenerative, neuromuscular and retinal diseases, today announced that new data from its preclinical research will be highlighted in oral and poster presentations at the 24th Annual American Society of Gene and Cell Therapy (ASGCT) Meeting, taking place virtually from May 11-14, 2021.

Key Points: 
  • b'SAN DIEGO, May 5, 2021 /PRNewswire/ --Locanabio, Inc., an RNA-targeting gene therapy company focused on developing life-changing therapies for patients with severe neurodegenerative, neuromuscular and retinal diseases, today announced that new data from its preclinical research will be highlighted in oral and poster presentations at the 24th Annual American Society of Gene and Cell Therapy (ASGCT) Meeting, taking place virtually from May 11-14, 2021.
  • Our unique and multidimensional approach uses gene therapy to deliver RNA binding protein-based systems to correct the message of disease-causing RNA and thereby change the lives of patients with devastating genetic diseases.
  • These broad capabilities delivered via gene therapy enable Locanabio to potentially address a wide range of severe diseases with a single administration.
  • The company is currently advancing programs in neurodegenerative, neuromuscular and retinal diseases.

Carmine Therapeutics appoints Don Haut as CEO

Retrieved on: 
Wednesday, May 5, 2021

b'CAMBRIDGE, Mass., May 5, 2021 /PRNewswire/ -- Carmine Therapeutics, Inc., an emerging leader in gene therapy, today announced that Don Haut, PhD has been appointed as its Chief Executive Officer, and member of the Board of Directors.\nFounded and incubated by EVX Ventures, Carmine is developing next-generation, non-viral gene therapies for a broad spectrum of diseases.

Key Points: 
  • b'CAMBRIDGE, Mass., May 5, 2021 /PRNewswire/ -- Carmine Therapeutics, Inc., an emerging leader in gene therapy, today announced that Don Haut, PhD has been appointed as its Chief Executive Officer, and member of the Board of Directors.\nFounded and incubated by EVX Ventures, Carmine is developing next-generation, non-viral gene therapies for a broad spectrum of diseases.
  • As such, Carmine believes its gene therapies have the potential for repeat dosing, large and multiple payload capacity, and improved manufacturing economics.\n"We are thrilled to welcome Don to the Carmine team.
  • Carmine has made tremendous progress in advancing the research and development of our platforms, and is nowmovingan extensive pipeline into the clinic.
  • Headquartered inCambridge, MAwith a research presence inSingapore, Carmine is pioneering next generation gene therapy based on red blood cell extracellular vesicles.\nTo date, Carmine has raised close to US$20M in financing.

Shape Therapeutics Announces Presentations at the Upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

Retrieved on: 
Tuesday, May 4, 2021

ShapeTX gene therapy platform comprises RNAskipTM, RNAfixTM, RNAswitch 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.

Key Points: 
  • ShapeTX gene therapy platform comprises RNAskipTM, RNAfixTM, RNAswitch 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 analytics platform, where data drives decisions today to enable tomorrow's gene therapies.
  • ShapeTX is committed to data-driven scientific advancement, passionate people, and a mission of providing lifelong cures to patients.
  • Shape Life!\n"

AVROBIO Provides Regulatory Update on Investigational AVR-RD-01 for Fabry Disease

Retrieved on: 
Monday, May 3, 2021

b'AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today provided an update on its regulatory plans for AVR-RD-01, the first investigational lentiviral gene therapy for Fabry disease.

Key Points: 
  • b'AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today provided an update on its regulatory plans for AVR-RD-01, the first investigational lentiviral gene therapy for Fabry disease.
  • In addition, the conversion of Fabrazyme to full approval limits the accelerated approval pathways available for new therapies to treat Fabry disease.
  • Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease.
  • AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.\nView source version on businesswire.com: https://www.businesswire.com/news/home/20210503005315/en/\n'

Affinia Therapeutics Closes $110 Million Series B Financing

Retrieved on: 
Monday, May 3, 2021

b'WALTHAM, Mass., May 03, 2021 (GLOBE NEWSWIRE) -- Affinia Therapeutics, an innovative gene therapy company with a proprietary platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies for rare and non-rare diseases, today announced it has closed an oversubscribed $110 million Series B financing with a premier syndicate of life science investors, co-led by EcoR1 Capital and Farallon Capital Management.

Key Points: 
  • b'WALTHAM, Mass., May 03, 2021 (GLOBE NEWSWIRE) -- Affinia Therapeutics, an innovative gene therapy company with a proprietary platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies for rare and non-rare diseases, today announced it has closed an oversubscribed $110 million Series B financing with a premier syndicate of life science investors, co-led by EcoR1 Capital and Farallon Capital Management.
  • Existing investors Atlas Venture, F-Prime Capital, Lonza, Mass General Brigham Ventures, and New Enterprise Associates (NEA) also participated in the round.
  • \xe2\x80\x9cWe are excited to partner with this syndicate of top investors to expand our platform to regulatory elements and work toward the next generation of one-time, potentially curative medicines for broader use.\xe2\x80\x9d\nIn 2020, Affinia Therapeutics announced a Series A financing and a multi-year research collaboration with Vertex Pharmaceuticals.
  • In connection with the Series B financing, Caroline Stout, partner at EcoR1 Capital, has been appointed to Affinia Therapeutics\xe2\x80\x99 board of directors.\n\xe2\x80\x9cGene therapies with novel vectors and regulatory elements have the potential to benefit a broad range of both rare and non-rare diseases,\xe2\x80\x9d said Ms. Stout.

Horizon Therapeutics plc Selects AGC Biologics to Further Supply KRYSTEXXA® (pegloticase injection) at AGC’s Copenhagen Facility

Retrieved on: 
Friday, April 30, 2021

We provide world-class development and manufacture of mammalian and microbial-based therapeutic proteins, plasmid DNA (pDNA), viral vectors and genetically engineered cells.

Key Points: 
  • We provide world-class development and manufacture of mammalian and microbial-based therapeutic proteins, plasmid DNA (pDNA), viral vectors and genetically engineered cells.
  • Our global network spans the U.S., Europe and Asia, with cGMP-compliant facilities in Seattle, Washington; Boulder, Colorado; Copenhagen, Denmark; Heidelberg, Germany; Milan, Italy; and Chiba, Japan and we currently employ more than 1,700 employees worldwide.
  • Our commitment to continuous innovation fosters the technical creativity to solve our clients\xe2\x80\x99 most complex challenges, including specialization in fast-track projects and rare diseases.
  • AGC Biologics is the partner of choice.

Forge Biologics Announces Closing of $120 Million Series B Financing

Retrieved on: 
Thursday, April 29, 2021

b'Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced the closing of a $120 million Series B financing.

Key Points: 
  • b'Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced the closing of a $120 million Series B financing.
  • The financing was led by RA Capital Management with participation from Perceptive Advisors and related affiliates, Surveyor Capital (a Citadel company), Octagon Capital, and Marshall Wace.
  • \xe2\x80\x9cWe have built Forge into a focused gene therapy development engine with a firm commitment to helping our clients provide potentially life-saving gene therapies to patients.
  • By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most.\nView source version on businesswire.com: https://www.businesswire.com/news/home/20210429005365/en/\n'

Sio Gene Therapies Announces Four Upcoming Oral Presentations at the 24th Annual Meeting of the American Society of Gene and Cell Therapy

Retrieved on: 
Wednesday, April 28, 2021

The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well.

Key Points: 
  • The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well.
  • We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinson\xe2\x80\x99s disease, which affects millions of patients globally.
  • All forward-looking statements are based on estimates and assumptions by Sio\xe2\x80\x99s management that, although Sio believes to be reasonable, are inherently uncertain.
  • All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected.