Stefan Oelrich, head of Bayers pharmaceuticals department, admitted at the World Health Summit that the COVID-19 vaccine is gene therapy. He smugly stated that the drug companies knew people would reject the vaccine if they knew it was in fact a gene-altering injectable. They lied to us for profits as the vaccine certainly did not prevent anyone from contracting or transmitting the virus.

If we had surveyed two years ago if people were willing to take gene or cell therapy and inject it into your body we would have probably had a 95% refusal rate, Oelrich admits while forgetting many took the gene therapy through force.

Twitter has already flagged retweets of this video as misleading. The conspiracy theorists who were told they had no place in society were right as Big Pharma and governments worldwide used the public as guinea pigs for the largest gene therapy study in history. We still do not know the long-term health implications but have seen a variety of health issues and lingering side effects in the short-term. Revolutions have occurred over much less.

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Bayer Head Admits COVID-19 Vaccine is Gene Therapy

Summary: A new study reports that a new gene therapy shows promise in treating dry age-related macular degeneration. The gene therapy, ophNdi1, directly targets mitochondrial function that is malfunctioning in AMD.

Source: TCD

Researchers from Trinity College Dublin have developed a new gene therapy approach that shows promise for treating the dry form of age related macular degeneration (AMD)a progressive eye disease that affects up to 10% of adults over 65 years of age and is a leading cause of severe vision impairment and blindness in this age group.

Dry AMD cases represent about 8590% of all AMD cases and there are no treatments available to treat or preventdisease progression, underlining the need for developing treatment options for this debilitating disease. In adults, many diseases of aging have been found to have defects of mitochondrial function, including AMD.

The team, in Trinitys School of Genetics and Microbiology, have developed a new gene therapy (ophNdi1) that is the first of its kind to directly target mitochondrial function in cells that are malfunctioning in AMD.

Mitochondria are known as the powerhouses of the cell because they manage the production of energy but their performance dips greatly in dry AMD and this is linked to a deterioration in sight.

The new gene therapy cleverly uses a virus to access the cells that are suffering and deliver the code needed to give the failing mitochondria a lifeline, enabling them to generate extra energy and continue to function in supporting vision.

The therapy has shown benefit in multiple models of dry AMD, offering hope that it could one day progress to a treatment that could help millions across the globe.

Professor Jane Farrar, senior author, said, Critically, this study provides the first evidence in models that directly modulating bioenergetics in eye cells can provide benefit and improve visual function in dry AMD. In doing so, the study highlights the energy powerhouses of the cell, mitochondria, as key targets for dry AMD.

Dr. Sophia Millington-Ward, first author and research fellow in Trinitys School of Genetics and Microbiology, said, The novel gene therapy targeting cellular energy, or mitochondrial function, that we explored for dry AMD consistently provided benefit in the model systems tested.

Many retinal cells, essential for vision, require particularly high levels of energy compared to most other cells, which makes them particularly vulnerable tomitochondrial dysfunction.

The therapy we are developing directly targets mitochondrial function and increases energy production levels in the retina, which leads to better visual function in disease models of dry AMD.

Although there is further work to be done before this could be made available as a treatment for patients, the results give us hope that we are getting closer to a solution to this challenging, debilitating condition.

Author: Press OfficeSource: TCDContact: Press Office TCDImage: The image is in the public domain

Original Research: Open access.AAVmediated gene therapy improving mitochondrial function provides benefit in agerelated macular degeneration models by Sophia MillingtonWard et al. Clinical and Translational Medicine

Abstract

AAVmediated gene therapy improving mitochondrial function provides benefit in agerelated macular degeneration models

With an estimated 196 million people suffering from age-related macular degeneration (AMD) in 2020 and predicted to increase to 288 million by 2040,dry AMD, representing 70%90% of AMD cases, represents an enormous clinical need with no current therapies.

We have demonstrated that NDI1 and an optimised version of NDI1 (ophNdi1), a mitochondrial complex 1 equivalent fromSaccharomyces cerevisiae, provide functional and histological benefit in two murine models of dry AMD as well as benefit in two cellular models of dry AMD. There are no drugs on the market for dry AMD.

However, there are currently a small number of candidate gene therapies in clinical trial (clinicaltrials.gov).

To our knowledge, this is the first demonstration that a gene therapy directly targeting mitochondrial dysfunction provides functional benefit in in vivo models of dry AMD, making this a novel approach to treating this devastating condition.

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New Gene Therapy Shows Promise for Treating Age Related Macular ...

1. After $100M deal, J&J links gene therapy to improved vision in early-phase trial  FierceBiotech
2. Janssen Announces Late-Breaking Data from Two Gene Therapy Programs at the American Academy of Ophthalmology 2022 Annual Meeting  Johnson & Johnson
3. AAO: Janssen announces late-breaking data from a pair of gene therapy programs  Ophthalmology Times
4. #AAO22: J&J's first look at common eye disease portfolio pads the case for PhII of gene therapy  Endpoints News
5. Late-Breaking Phase 1/2 Data Demonstrates Safety Profile of Investigational Gene Therapy Botaretigene Sparoparvovec (AAV-RPGR) and Sustained Vision Improvement in Patients with X-Linked Retinitis Pigmentosa (XLRP)  GlobeNewswire
6. View Full Coverage on Google News

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After $100M deal, J&J links gene therapy to improved vision in early-phase trial - FierceBiotech

Dive Brief:

Roctavians road back to the FDA has been long, as BioMarin has had to gather additional data in support of its therapy. Delays and new safety concerns further hampered the companys resubmission plans.

The treatment is for people with severe hemophilia A, a genetic disease that leaves people with little or no clotting protein needed to prevent excessive bleeding.

While there are many treatments that work by replacing this protein artificially, Roctavian would be the first in the U.S. to address the diseases root cause by replacing the defective gene with a functional copy.

The FDA rejected BioMarins original approval application back in August 2020 and requested more data to prove the treatments benefit for patients over a longer period of time.

BioMarin successfully gathered that follow-up data, showing its gene therapy could restore protein levels to mildlevels and prevent bleeds. The company had expected to file for approval in June but pushed back to September after the FDA asked for further information.

Roctavian gained European approval last month, a few weeks before it was disclosed that a patient treated in clinical trials had developed leukemia. This was the second cancer case reported among patients taking Roctavian. Cases of cancer in clinical trials of gene therapies have been watched closely by U.S. regulators. However, in BioMarins case, genetic testing suggested the case may be naturally occurring, rather than stemming from the therapy. Trial monitors did not call for the study to be halted.

If approved in the U.S., Roctavian is expected to carry a high price tag, likely in the millions of dollars. In Europe, BioMarin is charging around 1.5 million euros, net of discounts. Other gene therapies cleared for the U.S. market have been priced at $2.1 million, $2.8 million and $3.0 million, respectively.

Pfizer and partner Sangamo Therapeutics as well as Spark Therapeutics now owned by Roche are also working on hemophilia gene therapies.

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BioMarin resubmits its hemophilia gene therapy to the FDA - BioPharma Dive

Massachusetts-based bluebird bio (BLUE Quick QuoteBLUE - Free Report) is a biotechnology company focused on developing gene therapies for severe genetic diseases and cancer.

bluebirds performance has been impressive after the company reported several positive news over the past two months.

In September, the company received an accelerated FDA approval for Syskona (elivaldogene autotemcel), also known as eli-cel, as a treatment for patients younger than 18 years with early, active cerebral adrenoleukodystrophy (CALD).

The company also announced that the clinical hold previously imposed by the FDA on the eli-cel development program in August 2021 has now been lifted on Sep 15, 2022, before the review of the Syskona biologics license application (BLA).

However, we remind investors that the continued approval of Syskona for CALD may depend upon verification and description of clinical benefit in a confirmatory study.

Thus, as a condition of Syskonas accelerated approval, bluebird will have to provide confirmatory long-term clinical data to the FDA from the ongoing long-term follow-up study (LTF-304), which follows patients treated in clinical trials for 15 years and from commercially treated patients.

With the accelerated approval in place, the company expects to launch Syskona commercially in the United States by the end of 2022 to enable patient access to gene therapy as soon as possible.

Syskona is the second ex-vivo lentiviral vector gene therapy to be approved by the FDA in the United States.

The first is bluebirds Zynteglo (betibeglogene autotemcel), also known as beti-cel, which the FDA approved in August as the first cell-based gene therapy. The therapy treats beta-thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions across all genotypes.

The FDA approvals boost bluebird bios growth prospects, but the successful commercialization of these therapies holds the key. Moreover, gene therapies are complex by nature.

bluebird will face stiff competition in the target market. CRISPR Therapeutics (CRSP Quick QuoteCRSP - Free Report) and its partner Vertex Pharmaceuticals (VRTX Quick QuoteVRTX - Free Report) announced that the FDA granted a rolling review to the gene therapy exagamglogene autotemcel (exa-cel), formerly known as CTX001. The companies are developing the therapy for treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

Vertex intends to submit its BLA for exa-cell for a rolling review by the FDA by the start of November and expects to complete the process by the end of first-quarter 2023.

These approvals have provided bluebird two priority review vouchers, which are transferable. These vouchers can help shorten the review process of a new drug application (NDA) from 10 months to six months. With the intent of exploring additional financing opportunities, bluebird intends to monetize these vouchers in the near term.

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bluebird's (BLUE) Gene Therapy Approvals to Drive the Top Line - Zacks Investment Research

Acquisition of select PACT assets enables AmplifyBio to expand beyond safety, efficacy, and toxicology services to enable "living medicine" developers to shorten timelines and mitigate risk when moving to the clinic

WEST JEFFERSON, Ohio and SOUTH SAN FRANCISCO, Calif., Oct. 3, 2022 /PRNewswire/ --AmplifyBio, a contract research organization (CRO) focused on accelerating innovation across pharmaceutical modalities; today announced the acquisition of select assets fromPACT Pharma, Inc., a privately held biopharmaceutical company developing neoantigen-specific T cell receptor cell therapies. The deal will provide AmplifyBio with advanced characterization platforms, bioinformatics capabilities, and 40 drug development experts to enhance their cell and gene therapy service offerings. AmplifyBio will also acquire the South San Francisco advanced laboratory space.

With the acquisition of these assets, AmplifyBio aims to provide an early, consistent characterization of a treatment's purity, potency, and viability throughout the life cycle of therapeutic development. Unlike small molecules, there is no single, consistent process for cell and gene therapy companies to research, develop, and test their therapeutics. The gap that exists in characterization between the discovery phase and preclinical testing leads to material changes in a therapeutic during development, which can in turn create manufacturing inconsistencies and safety concerns during scale-up.

"Many biologics developers have adopted the phrase 'the process is the product' to describe how their therapeutic is differentiated based on a unique development process," said AmplifyBio Chief Executive Officer (CEO) and President J. Kelly Ganjei. "Rather than create our own, individual technique, AmplifyBio aims to replace that saying with a new one: 'the product is the product. Our acquisition of these assets from PACT Pharma means that cell and gene therapies can now be differentiated based on safety and efficacy profiles and specific product characteristics, not development processes."

"This deal allows PACT to retain its core intellectual property and continue our mission of developing novel, neoantigen-targeted T-Cell Therapies," added Scott Garland, PACT Pharma's CEO. "At the same time, we're working with AmplifyBio to leverage our platforms to offer a unique combination of optimization, characterization, safety and efficacy services to a wider range of clients seeking to better understand the immunology of their adoptive cell therapies."

AmplifyBio was spun out in 2021 from Battelle, a not-for-profit organization that advances science and technology to have the greatest impact on our society and economy. Following today's acquisition of the South San Francisco facility, AmplifyBio plans to add a third site in New Albany, Ohio that consists of 350,000 square feet of multi-function lab spaces. There, AmplifyBio will build on its advanced therapy services by adding capabilities for complex genotypic and phenotypic characterization analysis for late-stage development. The company expects to add additional development platforms and partnerships to become a commercial accelerator delivering safe, effective, reproducible advanced therapies to patients.

About AmplifyBioAmplifyBio is a leading preclinical CRO focused on toxicology, safety, and pharmacology testing to advance therapeutics for the betterment of human health. Spun out of Battelle in May of 2021, AmplifyBio's mission is to continue to provide exceptional CRO study services in an agile environment better suited to commercial goals and expand analytic capabilities to serve the dynamic needs of advanced therapy development. Clients of AmplifyBio enjoy the peace of mind that comes from decades of experience in GLP and non-GLP study design and execution, combined with rapid investment in technology, expertise, and infrastructure that together provide the critical components of a reliable, agile partnership.

Media Contact AmplifyBio[emailprotected]For Inquiries to PACT[emailprotected]

SOURCE AmplifyBio

Originally posted here:
AMPLIFYBIO ACQUIRES PACT PHARMA ASSETS TO ENHANCE CELL AND GENE THERAPY CHARACTERIZATION CAPABILITIES - PR Newswire

Theres a handful of reasons why the Philadelphia region has been (perhaps unfortunately) dubbed Cellicon Valley in the last few years, and a new report from the Chamber of Commerce for Greater Philadelphia and economic consulting firm Econsult Solutions has IDd them all.

In the report, which looked at 14 large cell and gene therapy hubs in the US, Philadelphia ranked as the runner up, just behind Boston, as the top spot for research and innovation in this space. Other metro areas such as New York and San Francisco scored the third and fourth spots on the list. The report shouts out early local work, including the first FDA-approved gene (Luxturna) and cell (Kymriah) therapies developed here at Spark Therapeutics and the University of Pennsylvania, respectively.

The Philadelphia region is increasingly attracting new and expanding cell and gene therapy companies because it checks all the boxes, but its the regions research infrastructure as defined by NIH-funded cell and gene therapy research and its large number of research institutions that give it the edge, said Claire Marrazzo Greenwood, executive director and CEO of Council for Growth and SVP of economic competitiveness for the Chamber, in a statement.

The study compared cell and gene therapy hubs for their research infrastructure, human capital, innovation output, commercial activity and value proposition. Heres why Philly ranked high:

Because Philly is home to four Tier 1 universities, 93 higher ed institutions, and tons of hospitals and research institutions, it scored second in research infrastructure. The region scored first for most National Health Institute funding, and the report said 302 gene or cell therapy patents had been approved in the last decade. In 2021, the region was home to 15,400 jobs in pharmaceutical manufacturing, and it pulled in $4.2 billion in venture capital funding since 2018.

The talent coming from the high number of universities and colleges and more than 450,000 students in the region also ranked the region high for human capital. Of this, a whopping 54% stay in the region. R&D jobs in the field have also increased more than 100% in the last five years.

Philadelphia also scored high for its innovation output, meaning the region produces a large amount of intellectual property in the cell and gene therapy space. As the birthplace of the industry, the report says, the region is currently home to 302 granted patent and 130 clinical trials now underway.

The large amount of attention cell and gene therapy has gotten from investors in the last four years also ranked the region high in commercial activity. Within the past few years, two local cell and gene therapy companies Passage Bio and Cabaletta Bio have also completed IPOs, raising more than $260 million combined. Cell and gene therapy companies also make up a significant portion of Phillys commercial real estate, leasing about 12 million square feet, with about 9 million planned in construction projects.

And Philadelphias value proposition, or cost to do business, helped the region rank so highly, the report said. The region attracts families and talent with cultural institutions, culinary scene and schools. Plus, life sciences office space rentals (averaging about $58 per square foot) were very affordable next to cities like San Francisco (at $78 per square foot).

Greater Philadelphia is an extremely livable region, boasting some of the worlds best museums, top-notch restaurants, and large open spaces at a comparatively affordable price, Econsult said in its summary.

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Why Philly ranks #2 among best cell and gene therapy hubs in the US - Technical.ly