Gene delivery

Cytovance Biologics announces Plasmid DNA Manufacturing: An Optimized Platform Process Webinar

Retrieved on:

Monday, June 14, 2021

Life sciences, Branches of biology, Biology, Molecular biology, Pharmaceutical industry, Gene delivery, Biotechnology, DNA, Plasmid, Biopharmaceutical, Contract manufacturing organization, DNA supercoil

OKLAHOMA CITY, June 14, 2021 (GLOBE NEWSWIRE) -- Cytovance Biologics, a leading biopharmaceutical contract development and manufacturing organization (CDMO) of mammalian, microbial and gene therapy biologics, announces the next webinar of its 2021 series: pDNA Manufacturing: An Optimized Platform Process.

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OKLAHOMA CITY, June 14, 2021 (GLOBE NEWSWIRE) -- Cytovance Biologics, a leading biopharmaceutical contract development and manufacturing organization (CDMO) of mammalian, microbial and gene therapy biologics, announces the next webinar of its 2021 series: pDNA Manufacturing: An Optimized Platform Process.
Our featured speakers will discuss strain selection, plasmid optimization, media composition and fermentation conditions necessary to produce high quality supercoiled plasmid DNA.
The demand for large quantities of high-quality supercoiled plasmid DNA (Sc-pDNA) has increased over the years due to its primary role in gene therapy.
The platform purification process developed by Cytovance addresses the main challenges in large scale manufacturing of pDNA such as scalability, quantity and quality.

Codexis and Takeda Expand Strategic Collaboration and License Agreement to Discover Additional Gene Therapy for a Fourth Rare Genetic Disorder

Retrieved on:

Thursday, June 10, 2021

Branches of biology, Biology, Genetic engineering, Life sciences, Gene delivery, Biotechnology, Emerging technologies, Molecular biology, Gene therapy, Transgene

Takeda is combining these improved transgenes with its gene therapy capabilities to develop novel candidates for the treatment of rare genetic disorders.

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Takeda is combining these improved transgenes with its gene therapy capabilities to develop novel candidates for the treatment of rare genetic disorders.
We are thrilled to expand our collaboration with Takeda to advance novel gene therapies for the treatment of rare diseases.
Codexis and Takeda are excited about the prospect for each of these improved sequences to enable differentiated gene therapies for patients with rare genetic diseases, stated John Nicols, Codexis President and CEO.
Takeda is responsible for the pre-clinical and clinical development and commercialization of gene therapy products resulting from the collaboration programs.

Generation Bio to Present at the JMP Securities Life Sciences Conference

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Wednesday, June 9, 2021

Branches of biology, Articles, Biology, Biotechnology, Applied genetics, Medical genetics, Gene delivery, Emerging technologies, Gene therapy, Dosing

CAMBRIDGE, Mass., June 09, 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 JMP Securities Life Sciences Conference on Wednesday, June 16, at 11:30 a.m.

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CAMBRIDGE, Mass., June 09, 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 JMP Securities Life Sciences Conference on Wednesday, June 16, at 11:30 a.m.
A live webcast of the presentation will be available on the investor section of the companys website at investors.generationbio.com .
The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed.
The ctLNP is designed to deliver large genetic payloads, including multiple genes, to specific tissues to address a wide range of indications.

New adaptable nanoparticle platform enables enhanced delivery of gene therapies, RCSI research

Retrieved on:

Wednesday, June 9, 2021

Branches of biology, Biology, Life sciences, Molecular biology, Medical genetics, Applied genetics, Biotechnology, Gene delivery, Gene therapy, Draft:Kevin Vance Morris

The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo.

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The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo.
A major challenge for gene therapies is preparing them in a way that can deliver the genetic information into the host cells.
The researchers developed a platform that produces bespoke star-shaped polypeptide nanoparticles, which effectively deliver a range of therapies, including gene therapies.
"With the success of the COVID-19 vaccines, the potential of gene therapies is becoming apparent, and advanced nanoparticle delivery systems are key to enabling their use clinically.

New adaptable nanoparticle platform enables enhanced delivery of gene therapies, RCSI research

Retrieved on:

Wednesday, June 9, 2021

Branches of biology, Biology, Life sciences, Molecular biology, Medical genetics, Applied genetics, Biotechnology, Gene delivery, Gene therapy, Draft:Kevin Vance Morris

The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo.

Key Points:

The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo.
A major challenge for gene therapies is preparing them in a way that can deliver the genetic information into the host cells.
The researchers developed a platform that produces bespoke star-shaped polypeptide nanoparticles, which effectively deliver a range of therapies, including gene therapies.
"With the success of the COVID-19 vaccines, the potential of gene therapies is becoming apparent, and advanced nanoparticle delivery systems are key to enabling their use clinically.

Transgene Strengthens Global IP Protection for its Novel Invir.IO™ Oncolytic Viruses Encoding Immune Checkpoint Inhibitors (ICIs)

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Tuesday, June 8, 2021

Oncology, Health, Other Health, Clinical trials, Pharmaceutical, Biotechnology, Branches of biology, Virotherapy, Medicine, Life sciences, Gene delivery, Experimental cancer treatments, Oncolytic virus, Biotechnology, Virus, Vector, Viral vector, Pexastimogene devacirepvec

Candidates based on this viral design aim to stimulate an immune response locally in the tumor and to optimize the safety profile of the virus.

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Candidates based on this viral design aim to stimulate an immune response locally in the tumor and to optimize the safety profile of the virus.
Transgene has already obtained patent grants for this new technology in the US, Europe, Australia, Hong Kong, Israel, and Russia.
By protecting the vectorization of ICIs in our VVcopTK-RR- oncolytic viruses, we are extending our Invir.IO IP beyond the original viral backbone protection.
With its proprietary platform Invir.IO, Transgene is building on its viral vector engineering expertise to design a new generation of multifunctional oncolytic viruses.

Bayer to Advance Two First-of-its-Kind Investigational Cell and Gene Therapies for Parkinson’s Disease

Retrieved on:

Tuesday, June 8, 2021

In parallel a gene therapy investigational program also targeted at providing advanced therapies for Parkinsons disease is driven forward by Bayers wholly-owned clinical-stage adeno-associated virus (AAV) gene therapy company Asklepios BioPharmaceutical Inc. (AskBio).

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In parallel a gene therapy investigational program also targeted at providing advanced therapies for Parkinsons disease is driven forward by Bayers wholly-owned clinical-stage adeno-associated virus (AAV) gene therapy company Asklepios BioPharmaceutical Inc. (AskBio).
The potential of BlueRock and AskBios clinical candidates to treat Parkinsons disease and truly help patients with their high unmet medical need could be immense, said Wolfram Carius, Head of Cell and Gene Therapy at Bayer.
Parkinsons disease is the most common neurodegenerative movement disorder, impacting more than 10 million people worldwide.
By targeting the disease at its root cause, cell and gene therapies aim to go beyond symptomatic treatments.

Krystal Biotech to Participate in Upcoming Investor Conferences

Retrieved on:

Thursday, June 3, 2021

Branches of biology, Life sciences, Health sciences, Bioethics, Gene delivery, Gene therapy, Molecular biology, Krystal, Forward-looking statement, Therapy

Krystal Biotech, Inc. (NASDAQ:KRYS) is a pivotal-stage gene therapy company leveraging its novel, redosable gene therapy platform and in-house manufacturing capabilities to develop therapies to treat serious rare diseases.

Key Points:

Krystal Biotech, Inc. (NASDAQ:KRYS) is a pivotal-stage gene therapy company leveraging its novel, redosable gene therapy platform and in-house manufacturing capabilities to develop therapies to treat serious rare diseases.
In addition, the forward-looking statements included in this press release represent Krystals views as of the date of this release.
Krystal anticipates that subsequent events and developments will cause its views to change.
However, while Krystal may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so.

Oyster Point Pharma Announces Preclinical Study Results and Pipeline Expansion with Enriched Tear Film (ETF™) Gene Therapy to Target Ocular Surface Diseases

Retrieved on:

Thursday, June 3, 2021

Branches of biology, Biology, Life sciences, Applied genetics, Gene delivery, Medical genetics, Genetic engineering, Molecular biology, Adeno-associated virus, Gene therapy, Therapy, Nerve growth factor

ETF Gene Therapy is a proprietary adeno-associated virus (AAV) based gene therapy approach where a target gene is delivered to human lacrimal gland cells via intralacrimal gland injection.

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ETF Gene Therapy is a proprietary adeno-associated virus (AAV) based gene therapy approach where a target gene is delivered to human lacrimal gland cells via intralacrimal gland injection.
The goal for this target gene is to produce a selected gene product to change cell behavior and function on the ocular surface.
The goal for this target gene is to produce a gene product to change cell behavior and function on the ocular surface.
OC-101 (AAV-NGF) is Oyster Points investigational gene therapy in development as part of Oyster Points proprietary Enriched Tear Film (ETF) Gene Therapy pipeline expansion.

Adverum Appoints CMO and CSO

Retrieved on:

Tuesday, June 1, 2021

Branches of biology, Life sciences, Biology, Genetic engineering, Emerging technologies, Gene delivery, Medical genetics, Molecular biology, Adverum Biotechnologies, Gene therapy, Therapy

Dr. Riley added, AAV gene therapy represents the forefront of genomic medicines and Adverum has unique capabilities and world-class scientific minds that enable us to advance the science.

Key Points:

Dr. Riley added, AAV gene therapy represents the forefront of genomic medicines and Adverum has unique capabilities and world-class scientific minds that enable us to advance the science.
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.