Category Archives: Gene therapy

As the cell and gene revolution heats up, contract manufacturer AGC Biologics is getting ahead of the curve with plans for its second commercial plant in Colorado.

Angling to bolster cell and gene production, AGC has clinched a deal for a commercial Novartis Gene Therapies factory in Longmont, Colorado. Located just 16 miles from AGCs 20,000-liter mammalian facility in Boulder, the new plant is expected to add significant additional capacity, AGC said in a release.

The move comes shortly after AGC charted an expansion at its cell and gene site in Milan, Italy, which it snared last July in its buyout of Italian CTG biotech Molecular Medicine (MolMed).

AGC hasnt divulged the Novartis plants price. The company didnt say how big it expects the Longmont workforce to be, but it will [aim] to hire a significant percentage of Novartis staff there.

RELATED:Novavax enlists AGC Biologics to manufacture adjuvant for COVID-19 shot

The 622,000-square-foot factory comes equipped with offices and production space across six buildings. It sits on a 229-acre campus located 40 miles north of Denver, AGC said.

Last June,AGC got its hands on its Boulder plant through similar means, picking up the commercial facility from AstraZeneca. That facility came equipped with two 20,000-liter stainless steel bioreactors, plusspace to add four more in the future. That same month, AGC tied up with Novavax to scale up and produce the Matrix-M adjuvant for its late-stage COVID-19 vaccine candidate,NVX-CoV2373.

Meanwhile, AGC has invested heavily in cell and gene therapiessince acquiring MolMed in 2020. With the addition of two MolMed commercial plants in Italy, AGC became one of the very few CDMOs to include both plasmid production and end-to-end cell and gene therapy services in its manufacturing repertoire, the company noted last year.

RELATED:AGC plots $194.5M, capacity-doubling upgrade to Copenhagen biologics site

At the time, AGC specifically highlighted MolMeds manufacturing know-how in genetically modified cells and viral vectors, or the engineered viruses used to deliver the cutting-edge medicines. That component, which is also used in AstraZeneca and Johnson & Johnsons recombinant COVID-19 vaccines, is already in shortage, with the bottleneck expected to tighten even more unless regulators, biopharmas and contractors move fast to address production shortfalls, GlobalDatasaid in a recent report.

And in March, AGC blueprinted an upgrade to its factory in Milan, sketching a capacity boost and the introduction of viral vector suspension capabilities. The expanded facilities should startfull operations in 2022, AGC has said.

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AGC Biologics expands further in Colorado with purchase of Novartis Gene Therapies plant - FiercePharma

Novartis Adakveo and Global Blood Therapeutics Oxbryta started their commercial life in 2019 as novel drugs for sickle cell disease. While physicians like their efficacy and safety profiles, formulary coverage is a hurdle, doctors told analysts in a survey. That's not to mention potential gene therapy threats.

For both Adakveo and Oxbryta, 64% of physicians said the drugs have unfavorable cost and coverage dynamics compared with traditional hydroxyurea therapies, analysts at JPMorgan wrote in summarizing survey responses from 25 U.S. physicians. Each of the doctors sees at least 25 sickle cell disease patients.

The unfavorable coverage profiles suggest the companies still have work to do to increase access, the JPMorgan team said in a recent note to investors. Currently, many payers are requiring patients try other meds before covering the newer drugs, the analysts wrote.

RELATED:New sickle cell drugs from Novartis, GBT need big discounts: ICER draft

The access problem comes in contrast to the fact that most doctors view the drugs as having a better or similar clinical profile compared with hydroxyurea.

Nevertheless, about half of physicians said their overall clinical experience with Oxbryta has been worse than that with hydroxyurea. Thatrate was 36% for Adakveo incomparison with the older drug. The JPMorgan team labeled the results as no surprise, given the Novartis and GBT drugs are relatively new.

Adakveo vs. Oxbryta

Between the two new offerings, doctors generally rated Adakveo higher in terms of itsability to reduce vaso-occlusive pain episodes,which is the most important product characteristic the doctors said they look for when making therapy choices. Oxbryta scored better with respect to hemoglobulin improvement or impact on anemia.

Adakveo is an antibody drug given by infusion once a month, whereas Oxbryta is a daily oral drug. Despite their difference, dosing and convenience dont weigh much on prescription behavior, the doctors said.

RELATED:Bluebird's Zynteglo trials set to resume, putting gene therapy back on flight path to FDA filing

Currently, physicians treat60% of sickle cell patients with single-agent drugs, the survey showed. Among those patients, only 6% are on Adakveo monotherapy, whileanother 6% take Oxbryta. Looking forward, the outlook appears to favor the Novartis drug. Over the next three years, the physicians expectsingle-agent use to rise to 18% for Adakveo but only 11% for Oxbryta, while combination utilization of the drugs with other therapies will remain relatively stable.

Butthe potential entry of gene therapies could become a key barrier to Oxbryta and Adakveo growth over the longer term. The surveyed doctors expect single-agent use of gene therapy to reach 24% of patients by 2028.

Overall, the physicians surveyed believe that a third of their sickle cell disease patients on average would be suitable for a gene therapy. Most physicians had a favorable view of the two clinical candidates, bluebird bios LentiGlobin and CRISPR Therapeutics and Vertexs CTX-001.

The physicians feedback highlights the potentialsqueeze fromfuture competition, the JPMorgan analysts said. All told, the team expects Adakveo to reach about $700 million in peak sales and Oxbryta to hit $950 million from the U.S. and EU.

COVID-19 slows launch

Like other drug launches in recent years, the pandemic has wreaked havoc on the sickle cell disease rollouts. A quarter of surveyed doctors said they'd seen a decrease in patient visits during the pandemic.In the first quarter, sales of Oxbryta reached $39 million in the U.S., compared with$37 million for Advakeo from the U.S. and EU.

RELATED:GBT chief blames COVID-19 for 'clear' slowdown in Oxbryta launch, but analysts are still impressed

Telemedicine has helped ease the negative effects from COVID-19, but doctors are less comfortable starting a new therapy without an in-person visit, GBTs chief commercial officer, David Johnson, said during an investors call in May.

As two doctors observed in their response to the JPMorgan survey, they have adopted telemedicine for very stable patients, or mostly for follow-ups and discussing side effects. Looking ahead, the physicians expect to increasingly shift back to on-site visits over the next six to 12 months.

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Novartis, GBT sickle cell drugs face coverage hurdles as gene therapy threats loom: survey - FiercePharma

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy in Oncology - Thematic Research" report has been added to ResearchAndMarkets.com's offering.

Gene therapy describes the treatment of various cancers with the use of in vivo treatments: viral and non-viral gene therapy products, therapeutic oligonucleotides, oncolytic viruses and genome editing therapies.

There are currently just 3 gene therapies marketed for oncology indications in the eight major pharmaceutical markets (8MM) (US, France, Germany, Italy, Spain, UK, Japan, and China). Oncolytic viruses lead the category with 2 products, followed by viral vector gene therapies with 1 approved drug.

Gene therapies are in development for melanoma and other various solid tumors. As of April 2021, there are 252 clinical trials investigating in vivo gene therapies across the 8MM with 81 drugs in development.

Sales of products that comprise the categories of in vivo gene therapy are forecast to reach over $7B by 2027. The therapeutic oligonucleotide market, which will be galvanized by the success of COVID-19 messenger ribonucleic acid (mRNA) vaccines, is forecast to reach $4.5B by 2027 globally.

Key Highlights

Key Questions Answered

Scope

Key Topics Covered:

1. Preface

2. Executive Summary

2.1. Key Findings

3. Gene Therapy Overview

3.1. What is Gene Therapy?

3.2. History of Gene Therapy Development in Cancer in the 8MM

3.3. Key Twitter Chat

4. Trends

4.1. Industry Trends - Gene Therapy Vectors

4.2. Industry Trends - Therapeutic Oligonucleotides

4.3. Industry Trends - Genome Editing

4.4. Industry Trends - Oncolytic Viruses

4.5. Regulatory Trends

5. Value Chain

5.1. Gene Therapy Value Chain

5.2. Gene Therapy Vectors

5.3. Therapeutic Oligonucleotides

5.4. Genome Editing

5.5. Oncolytic Viruses

5.6. Gene Therapy in Oncology Clinical Trials

6. Marketed Products

6.1. Marketed Gene Therapy Products for Cancer in the 8MM

6.2. Leading Gene Therapy Treatments in The 8MM

7. Pipeline Products

7.1. Gene Therapy Pipeline Products in the 8MM

7.2. Gene Therapy Pipeline Candidates

7.3. Late Stage Gene Therapy Candidates, 8MM

8. Market Analysis and Deals

8.1. Gene Therapy Market Analysis and Forecast by Class of Therapy

8.2. Top 10 Transaction Deals by Size during 2012-2021 in the Oncology Gene Therapy Space

8.3. Latest Transaction Deals in the Oncology Gene Therapy Space

8.4. Mergers and Acquisitions That Include Oncology Gene Therapy Assets: 2019 - 2021

8.5. Mergers and Acquisitions That Include Oncology Gene Therapy Assets: 2014 - 2018

9. Regulatory and Market Access

9.1. Gene Therapy in Clinical Trials

9.2. Regulatory - US

9.3. Market Access - US

9.4. Regulatory - Europe

9.5. Market Access - Europe

9.6. Regulation of Gene Therapy in the US and Europe

9.7. Regulatory and Market Access - Japan

9.8. Comparison of Early Access Schemes in the US, EU, and Japan

9.9. Regulatory and Market Access - China

10. Opportunities, Challenges, and Unmet Needs

10.1. Gene Therapy Vectors, Viral - Opportunities & Challenges

10.2. Gene Therapy Vectors, Non-viral - Opportunities & Challenges

10.3. Therapeutic Oligonucleotides - Opportunities & Challenges

10.4. Genome Editing - Opportunities & Challenges

10.5. Oncolytic Viruses - Opportunities & Challenges

10.6. Clinical Unmet Needs in Gene Therapy - Gap Analysis

10.7. Commercial Unmet Needs in Gene Therapy - Gap Analysis

10.8. Unmet Needs - KOLs Perspective

11. Companies

11.1. Drug Development Scorecard - Regenerative Medicine

11.2. Current Major Players

11.2.1. Amgen

11.2.2. SiBiono

11.2.3. Sunway Biotech

11.3. Future Players Based on Pipeline Strength

11.3.1. Candel

11.3.2. CG Oncology

11.3.3. Checkmate

11.3.4. Daiichi Sankyo

11.3.5. Ferring

11.3.6. FKD

11.3.7. Geron

11.3.8. Idera

11.3.9. Istari

11.3.10. VBL

12. Appendix

For more information about this report visit https://www.researchandmarkets.com/r/v0ma92

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Gene Therapy in Oncology Thematic Research Report 2021 - ResearchAndMarkets.com - Business Wire

A team of scientists and doctors from University College London Great Ormond Street Institute of Child Health (UCL GOS ICH) and Great Ormond Street Hospital (GOSH) have recreated and cured the condition using state-of-the-art laboratory and mouse models of the disease. They will soon apply for a clinical trial of the therapy.

The findings from the study have been published in Science Translational Medicine.

DTDS is a rare neurological condition that causes progressive dystoniaandparkinsonism, so called because of similarities to Parkinsons disease. It usually begins in infancy; however, some people may not develop symptoms until childhood or later. Infants with DTDS are rarely able to learn to walk or speak. Symptoms include slow movements, involuntary twisting postures of the arms and legs, and whole-body stiffness.

Currently, there are no effective treatments or a cure for the disorder and most children with DTDS sadly die before reaching adulthood, often from respiratory infections or other complications. Although the condition is rare, with around 50 children worldwide currently known to doctors, it has previously been mistaken for cerebral palsy and may continue to be undiagnosed.

Professor Manju Kurian discovered the faulty gene causing DTDS in 2009 and was subsequently granted seed funding worth just over 86,500 from Great Ormond Street Hospital Childrens Charity (GOSH Charity) to begin developing the treatment. Professor Kurians team and her collaborators at UCL have also spent the last decade working to better understand the mechanisms that underpin this disease, and this has enabled them to develop a new, precision gene therapy with the potential to treat this devastating disorder.

When developing the gene therapy, scientists took skin cells from children with DTDS and turned them into stem cells, which can grow into any type of cell to build or repair different parts of the body. Professor Kurians team, with work led by Dr Serena Barral, converted these stem cells into the exact brain cells (dopaminergic neurons) that carry the genetic fault responsible for DTDS.

Using this laboratory model made directly from the cells of children with this rare condition, scientists were able to test the experimental gene therapy for DTDS and show that it could relieve the disease-related defects in DTDS brain cells.

The team used fluorescence microscopy to see what was happening in the laboratory model. A seemingly random pattern of colours in the untreated cells demonstrated how the neurons and their communicating arms called neurites had not formed properly in cells with DTDS. The gene therapy treated cells formed a much more obvious cluster pattern for the neuron, with its red neurites, essentially showing the DTDS is cured in a laboratory model.

A further collaboration with UCLs Professor Simon Waddington and Dr Joanne Ng enabled the researchers to build on these results, studying DTDS in mice and testing gene therapy as a cure. The gene therapy injects a modified, harmless virus containing the healthy gene into the area of the brain where this gene is missing. The mice were successfully cured of their symptoms including involuntary and disordered movements, progressive parkinsonism, and weight loss. Based on the promising results of the laboratory tests, the next phase is to develop a clinical trial which would involve children diagnosed with DTDS.

Professor Manju Kurian, Consultant Paediatric Neurologist at GOSH and NIHR Research Professor at UCL Great Ormond Street Hospital Institute of Child Health, co-lead author on this study and the scientist behind the discovery of this disease, said: Our study provides real hope of an effective treatment for children who are living with this devastating, life-limiting brain disease, and it is hugely exciting to be at the stage of planning a clinical trial just ten years after discovering the gene that causes the condition.

We hope this pioneering gene therapy will prevent the progression of this rare but cruel disease with a single procedure, giving children the improved quality and length of life that they deserve. If we can use gene therapy to treat children with this condition early enough, there is great potential for improvement in their health.

Professor Simon Waddington, Professor of Gene Therapy at UCL, co-lead author on this study, said: Our whole working process has been guided by one principle: we want to find the answers for these children and how we can treat them.

The mice received the same carefully selected vector and delivery route that we plan to use in treating the children. This careful selection has allowed us to progress rapidly to design a protocol so we can start the clinical trial next year.

While DTDS is rare, we know that there are many other conditions we can model in this way, opening the door for a standardised approach to finding cures for these rare conditions.

Recommended Related Articles

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Gene therapy breakthrough could cure rare and fatal brain disease - Health Europa

CG01 is CombiGene's gene therapy for the treatment of drug-resistant focal epilepsy.

The newly released GMP plasmids were produced by Cobra in the early part of this year. It said the material had to be quality assured through a variety of analyses, a process that is now finalized.

The plasmid production itself was very successful and generated so much material that, according to current estimates, it will be enough for more productions of CG01 than originally planned, according to the parties.

The release of the plasmids produced by Cobra means that we will be able to start GMP production of CG01 later this year. In doing so, we are taking another important step towards the first in human study that we plan to start in 2022, saidJan Nilsson,CEO of CombiGene.

When asked why this production of plasmids is considered a landmark moment, and whether it was thought that the process was going to be more challenging than it turned out to be, Tony Hitchcock, technical director, Cobra, told BioPharma-Reporter:

This is a critical milestone for CombiGene as it provides the starting materials for the production of their clinical CGO1 therapy for the treatment of epilepsy. Cobra Biologics has been producing plasmid DNA for its customers for over 20 years, however the production of key plasmids for the production of AAV vectors such as CG01 can produce some challenges due to sequence elements and the size of the plasmids. There was a requirement to optimize processes to overcome these issues to generate plasmids of the required quality, and quantity, to support CombiGenes planned manufacturing campaigns.

Unlike many gene therapies, which are developed for the treatment of rare diseases, CG01 caters to a large population of patients, said CombiGene.

Epilepsy is a major global problem. Every year, approximately 47,000 drug-resistant patients with focal epilepsy are estimated to be added in the US, EU4, UK, Japan and China.CombiGene believes that it is realistic that 10-20% of these patients could be treated with the drug candidate, CG01.

Assuming, for example, that the therapy cost per patient is somewhere between $134,000 and $200,000, it provides sales between $750-$1,500 million annually, it added.

Cobra anticipates that there will be a long-term requirement to supply plasmid DNA to support the production of viral vectors, including AAV and Lenti viral vectors, for advanced therapy products, he added.

Whilst some manufacturers may adopt the use of stable cell lines for the production of certain vectors such as Lenti, it is likely that transfection approaches will be retained for some vectors such as AAV, where the generation of stable cell lines is more challenging, remarked Hitchcock.

What is next in terms of goals, both short term and long term, for Cobra Biologics?

Like many plasmid suppliers, Cobra is working to expand is plasmid production capabilities, both in terms of throughput and the numbers of High Quality (HQ) and GMP grade plasmid batches it can produce, whilst also scaling up its production processes to meet the needs for products entering later phase and commercial supply.

Furthermore, with Cobra Biologics becoming a Charles River company, alongside Cognate BioServices, and Vigene BioSciences, the goal is to continue to grow and develop our offering in the cell and gene therapy space, offering supply chain simplification and an end-to-end service offering for development, testing, and manufacturing, providing clients with an integrated solution from basic research and discovery through GMP production.

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Cobra flags successful production of gene therapy plasmids - BioPharma-Reporter.com

BioMarin has resubmitted a marketing authorisation application (MAA) for its haemophilia A gene therapy valoctocogene roxaparvovec to the European Medicines Agency (EMA), the company announced yesterday.

BioMarin initially withdrew the EU marketing application for the gene therapy also known as Roctavian last year, after stating that it was unable to provide the data needed to resolve a major objection raised by the EMAs Committee for Advanced Therapies.

The first EMA filing contained interim results from 32 adults with severe haemophilia A in a Phase III trial.

According to BioMarin, the EMA had requested the full 52-week results from all 134 patients enrolled in the study.

Now, BioMarin has that data in hand included in the new MAA is safety and efficacy data from the 134 patients enrolled in the study, all of whom have been followed for at least one year post-treatment with valoctocogene roxaparvovec.

"This is an important step to deliver the potential first gene therapy for people with haemophilia A.The data package for this submission includes the largest Phase III study in any gene therapy for haemophilia A supported by up to four years of observation in a Phase I/II study," said Hank Fuchs, president, worldwide research and development at BioMarin.

"We look forward to the EMA reviewing the robust data set. We believe that valoctocogene roxaparvovec represents a scientific innovation that may become an important treatment choice for those people who have unmet medical needs," he added.

BioMarin also announced that it is planning to submit two-year follow-up safety and efficacy data on all participants from the Phase III trial to support the benefit/risk profile of the gene therapy, which was previously requested by the US Food and Drug Administration (FDA).

Last August, BioMarin received a complete response letter from the FDA for valoctocogene roxaparvovec.

At the time, the FDA recommended that BioMarin complete the Phase III study and submit two-year follow-up safety and efficacy data on all study participants.

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BioMarin resubmits haemophilia A gene therapy to the EMA - PharmaTimes

The Pfizer/BioNTech and Moderna Covid-19 vaccines are not designed to alter your genes. (Photo by: ... [+] Daniel Garzon/VW Pics/Universal Images Group via Getty Images)

The Pfizer/BioNTech and Moderna Covid-19 vaccines are not genes in a bottle.

Yet, some on social media are now suggesting that such mRNA vaccines are actually gene therapy. For example, Alex Berenson, who has described himself as a former reporter for The New York Times and a current thriller novel author, mentioned the words gene therapy in a recent tweet. And presumably he wasnt referring to Gene Simmons therapy:

As you can see, Berensons tweet prompted a response and a correction from Seth Trueger, MD, MPH, an Assistant Professor of Emergency Medicine at Northwestern University.

Then theres the Its Gene Therapy, Not a Vaccine with Dr. David Martin podcast on the Weston A. Price Foundation website. This is the Foundation that once issued a press release entitled FDA and CDC Bias Against Raw Milk. Yes, the CDC has warned about the possibility of getting dangerous infections from raw milk, otherwise known as unpasteurized milk, as I covered previously from Forbes.

The Foundation website doesnt really describe Martins scientific background, simply saying that he received his Undergrad degree from Goshen College, Masters of Science from Ball State University and Doctorate from the University of Virginia, without specifying the actual subject matters of his degrees. So it isnt clear why they chose him to talk about Covid-19 versus someone else who happened to go to college and some kind of graduate school.

On the podcast, Martin said the following: The problem is that in the case of Moderna and Pfizer, this is not a vaccine. This is gene therapy. Its a chemotherapy agent that is gene therapy. It is not a vaccine. Chemotherapy? How did that word get in there? The National Cancer Institute (NCI) defines chemotherapy as treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Isnt mentioning chemotherapy when you are talking about a vaccine against a virus like mentioning spaghetti and hot dogs when talking about building a bear in a Build A Bear Workshop? Its mixing different things that dont seem to be related.

Martin then tried to explain, What is this doing? Its sending a strand of synthetic RNA into the human being and is invoking within the human being, the creation of the S1 spike protein, which is a pathogen. Its a toxin inside of human beings. This is not only not keeping you from getting sick, its making your body produce the thing that makes you sick.

No, no, no. Spike proteins line the surface of the severe acute respiratory syndrome coronavirus 2 (SARS-CoV2), which looks like a spiky massage ball but shouldnt be used as one. The spike protein helps the virus enter your cells. But the spike protein itself is not supposed to make you sick. Claiming that the spike protein alone is responsible for illness would be like claiming that a bleached blonde wig with frosted tips alone can cook like celebrity chef Guy Fieri.

The rest of the podcast didnt exactly scream science. It included the statement by Martin that Covid 19 is not a disease. It is a series of clinical symptoms. It is a giant umbrella of things associated with what used to be associated with influenza and with other febrile diseases. No. Covid-19 is not a giant umbrella. Where the ella, ella, eh, eh did that come from? Covid-19 is not the flu either. Repeat, having Covid-19 is not the same as having influenza.

Oh, and holy conspiracy theories, Batman, Martin asserted that Anthony Fauci, MD, Director of the National Institute of Allergy and Infectious Disease (NIAID) is manipulating this situation to force a vaccine on a population.

How much real evidence did Martin offer to support his claims? Well, the answer rhymes with not much. Its easy to say anything when the interviewer doesnt ask you to provide real concrete evidence behind what you are saying.

The Covid-19 mRNA vaccines are not gene therapy because they are not designed to alter or change your genes in any way. The U.S. National Library of Medicine (NLM) describes gene therapy as a technique that may allow doctors to treat a disorder by inserting a gene into a patients cells instead of using drugs or surgery. For example, doctors may be able to either inactivate or replace a mutated gene that isnt functioning properly or place a new gene in your body that will do something to combat a disease.

A gene consists of DNA and serves as the basic physical and functional unit of heredity, according to the NLM. Messenger RNA, known as mRNA for short, is different from DNA. RNA stands for ribonucleic acid. DNA stands for deoxyribonucleic acid. DNA serves as the library for instructions to produce different proteins. When a cell wants to produce a protein, it uses the DNA to produce a copy of mRNA. That mRNA then serves as a blueprint for the protein that is built by the ribosomes in your cells. The DNA is in the nucleus of the cell. The ribosomes are not. Thus, the mRNA from a Covid-19 vaccine will not go into the nucleus but instead will simply go to the ribosomes, which in turn will manufacture the spike protein.

This PBS News Hour video shows this process and emphasizes that mRNA should not alter your DNA:

The Pfizer/BioNTech and Moderna Covid-19 vaccines are not gene therapy. Repeat, these vaccines are not gene therapy. In fact, they dont really involve your genes, unless Gene Simmons or Gene Hackman happen to be getting the vaccines. Or you accidentally drops the vaccines on your jeans and then misspell the word jeans.

Full coverage and live updates on the Coronavirus

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Covid-19 mRNA Vaccines Are Not Gene Therapy, As Some Are ...

Vedere Bio II is fresh off a $77 million raise, and the follow-up act to the original Vedere Bio now has a big-name chief scientific officer in Gabor Veres, Ph.D.

The Cambridge, Massachusetts-based biotech is at work on next-gen eye disease gene therapies, aiming to restore sight, or help those keep it, with patients suffering from vision loss due to photoreceptor death.

To help in that mission, the company has poached Veres from rival biotech BioMarin, where he was vice president and head of gene therapy research focusing on AAV platform discovery and seeking out new indications. He also served stints at bluebird bio and Applied Genetic Technologies.

At VB II, he will provide strategic leadership to Vedere's research organization and will be responsible for driving and expanding the company's pipeline of ocular gene therapies, the biotech said in a statement.

The biotech has a history that while short, is very dense: Cyrus Mozayeni, M.D.s first go at vision-restoring gene therapies got snapped up as it was just getting started. Lots of suitors came knocking even when that company, Vedere Bio, was working under the radar, and Novartis eventually ponied up $150 million upfront to buy it last fall, with a promise of $130 million more.

Buoyed by that success but clearly still hungry for more, Mozayeni, its CEO and Atlas Venture entrepreneur in residence and his team last month nabbed $77 million to bankroll their second chapter: Vedere Bio II.

Although its technically a new entity, its working on gene therapies for vision loss with the same founders, management team and employees as its predecessor. Vederes focus is vision loss caused by the death of photoreceptors or light-sensing cells in the retina rather than blindness stemming from damage to the brain or optic nerve. With Veres, that team just got a little more diversified.

RELATED: Spark grabs FDA nod for Luxturna, a breakthrough gene therapy likely bearing a pioneering price

"Gabor has over 15 years of experience leading preclinical research and development for international pharmaceutical and biotechnology companies and holds 20 years of experience in the development of cell and gene therapy products, said Mozayeni.

His extensive track record designing and executing gene therapy research programs for a range of diseases, including conditions of the eye, will be an incredible asset to our team as we continue our work to restore vision to patients with both genetic and non-genetic causes of vision loss.

Gene therapies have long held much promise, but safety issues and manufacturing complexities have always dampened companies capacity to deliver. Gene therapies for eye disorders have been among the few to see R&D success translate into the market, most recently with Roche/Sparks Luxturna, though sales forecasts are fairly modest, and it requires specialist centers for administration.

Vedere's approach to vision restoration holds great potential to treat vision loss regardless of underlying genetic cause or disease stage, and I'm looking forward to helping build out and progress the company's unique platform and product pipeline, added Veres.

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BioMarin's gene therapy lead jumps ship to rival biotech Vedere Bio II - FierceBiotech

One year after receiving an experimental gene therapy developed by the Dutch drugmaker UniQure, patients with hemophilia B aren't having nearly as many bleeding issues as they used to have.

In hemophilia, genetic mutations prevent the body from making proteins needed to clot blood. People with the less common, "B" form of the disorder are missing a protein known as Factor IX. UniQure's medicine is meant to provide a working version of the gene so patients can generate their own clotting protein, and rely less or ideally, not at all on so-called replacement factor treatments.

UniQure said Tuesday that, a year after receiving the company's therapy, patients enrolled in the company's HOPE-B clinical trial were experiencing 80% fewer bleeding episodes that require treatment.

At the trial's start, these patients were having an average of three to four bleeding episodes annually. Now, according to UniQure, they're having less than one. The use of replacement factor therapy declined 96% during the 52-week period, as all but two of the 54 participants successfully discontinued their preventive infusions.

What's more, patients had almost 42% the amount of clotting protein typically observed in people who don't have hemophilia B. That's a slight improvement from six-month data presented in December, which showed protein levels at 39% of normal.

Yet, in spite of the positive data, UniQure shares dipped more than 5% in early afternoon trading Tuesday. CSL Behring, which spent $450 million to acquire the rights to UniQure's therapy, also saw its stock trade down.

Some of that stock market reaction may be related to an update Uniqure disclosed alongside the fresh data about the approval process for its therapy. According to UniQure, the Food and Drug Administration primarily wants to see evidence the therapy has a lasting effect, so the agency's focus will be on 52-week data. However, that time frame must start from when patients' protein levels stabilize, rather than from when they first received UniQure's therapy.

The company said all participants in its study achieved steady-state protein levels by week 26. As a result, UniQure is now measuring the study's success by annualized bleeding rates at week 78, with the expectation that patients to complete 78-week follow-up visits by the end of September. That should allow UniQure and CSL to file for approval in first quarter of 2022, months later than when the companies had estimated last year.

If approved, UniQure's gene therapy would become the first cleared to treat any kind of hemophilia. Another, for hemophilia A, was up for approval last year, but the FDA rejected it in in a major surprise. In issuing its rejection, the FDA asked for two years of follow-up data from the therapy's developer, BioMarin Pharmaceutical, a task that can't be completed until November 2021.

Estimates hold there are as many as 33,000 people living with hemophilia in the U.S., with the B form being about four times less common.

Though the FDA has approved around half a dozen drugs to treat hemophilia B, patients taking them can still experience bleeds. Additionally, they can develop a type of antibody called an inhibitor, which blocks the effects of replacement factor.

That's made gene therapy, with its potential as a one-time fix for the disease, an attractive option for some patients and physicians.

Still, concerns remain. UniQure's study was stopped late last year, for example, after researchers saw an unexpected case of liver carcinoma in one participant, reigniting long-held fears about gene therapy's potential to drive cancer.

UniQure investigated the case and recently concluded, along with an independent laboratory and outside experts, that its therapy was "highly unlikely" to have caused the cancer.

There are also worries that gene therapy won't work for everyone. Some people have pre-existing "neutralizing antibodies" that attack the viruses used to deliver functioning genetic material.

UniQure has found its therapy appears, for the most part, to bypass this issue of neutralizing antibodies. The company said Tuesday it hasn't seen any "significant correlation" in how patients who have these antibodies at least to a certain range are responding to its therapy.

UniQure said that range is high enough that it should encompass more than 93% of the general population. The company noted in December how one of the study participants who didn't respond its therapy had neutralizing antibody levels that were five times greater than anyone else in the group.

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A year after getting UniQure's gene therapy, hemophilia patients are still doing better - BioPharma Dive

As calls for mandatory COVID-19 vaccination grow around the world, its becoming ever more crucial to understand what these injections actually are. The mRNA vaccines created by Moderna and Pfizer are in fact gene therapies.

Interestingly enough, mainstream media, fact checkers and various industry front groups insist the gene therapy claim is bogus, even though every single detail about the vaccines shouts otherwise. Why are they spreading this disinformation? Why do they not want you to know what these injections actually are?

To start, lets take a look at some basic definitions of words. According to the U.S. Centers for Disease Control and Prevention, a vaccine is:1

Immunity, in turn, is defined as:

Thats the medical definition. The legal definition, in the few cases where it has been detailed, is equally unequivocal:

These definitions, both medical and legal, present problems for mRNA vaccines, since:

We should not be fooled by attempts to condition the public to accept redefined terms. As of February 2019, Merriam-Webster defined5 vaccine as a preparation of killed microorganisms, living attenuated organisms, or living fully virulent organisms that is administered to produce or artificially increase immunity to a particular disease. By February 26, 2021, they had updated the definition of vaccine to:6

A preparation that is administered (as by injection) to stimulate the bodys immune response against a specific infectious disease:

a: an antigenic preparation of a typically inactivated or attenuated pathogenic agent (such as a bacterium or virus) or one of its components or products (such as a protein or toxin)

b: a preparation of genetic material (such as a strand of synthesized messenger RNA) that is used by the cells of the body to produce an antigenic substance (such as a fragment of virus spike protein)

Lets be clear. Merriam-Webster does not dictate medical terminology. It can be used, however, to confuse people. For now, all medical dictionaries still show the traditional definition of vaccine,7 as Merriam-Webster did up until this year. That said, I would not be surprised if changes are made there as well, eventually, if the misrepresentation of COVID-19 mRNA vaccines is allowed to stand.

Theres also the issue of whether a gene therapy can be mandated, and this may hinge on it being accepted as a vaccine. The 1905 Supreme Court ruling in Jacobson v. Massachusetts8 essentially established that collective benefit supersedes individual benefit.

Since mRNA therapies do not render the immunized person immune, and do not inhibit transmission of the virus, they cannot qualify as a public health measure capable of providing collective benefit that supersedes individual risk, and therefore cannot be mandated.

Put another way, the ruling argues (although legal experts diverge on some of the finer details of its interpretation) that its acceptable for some individuals to be harmed by a public health directive as long as it benefits the collective. However, if vaccination is a public health measure meant to protect and benefit the collective, then it would need to accomplish two things:

Were now back to the original problem that mRNA therapies for COVID-19 do not accomplish either of these things. Since these gene therapies do not render the person immune, and do not inhibit transmission of the virus, they cannot qualify as a public health measure capable of providing collective benefit that supersedes individual risk.

On the contrary, the only one benefiting from an mRNA vaccine is the individual receiving the gene therapy, since all they are designed to do is lessen clinical symptoms associated with the S-1 spike protein.

In other words, they wont keep you from getting sick with SARS-CoV-2; they are only supposed to lessen your infection symptoms if or when you do get infected. So, getting vaccinated protects no one but yourself. Since youre the only one who will reap a benefit (less severe COVID-19 symptoms upon infection), the justification to accept the risks of the therapy for the greater good of your community is blatantly irrational.

Since mRNA vaccines do not meet the medical and/or legal definition of a vaccine, referring to them as vaccines, and marketing them as such, is a deceptive practice that violates9 15 U.S. Code Section 41 of the Federal Trade Commission Act,10 the law that governs advertising of medical practices.

The lack of completed human trials also puts these mRNA products at odds with 15 U.S. Code Section 41. Per this law,11,12 it is unlawful to advertise that a product or service can prevent, treat, or cure human disease unless you possess competent and reliable scientific evidence, including, when appropriate, well-controlled human clinical studies, substantiating that the claims are true at the time they are made.

Heres the problem: The primary end point in the COVID-19 vaccine trials is not an actual vaccine trial end point because, again, vaccine trial end points have to do with immunity and transmission reduction. Neither of those was measured.

Whats more, key secondary end points in Modernas trial include prevention of severe COVID-19 disease (defined as need for hospitalization) and prevention of infection by SARS-CoV-2, regardless of symptoms.13,14 However, Moderna did not actually measure rate of infection, stating that it was too impractical to do so.

That means theres no evidence of this gene therapy having an impact on infection, for better or worse. And, if you have no evidence, you cannot fulfill the U.S. Code requirement that states you must have competent and reliable scientific evidence substantiating that the claims are true.

Making matters worse, both Pfizer and Moderna are now eliminating their control groups by offering the real vaccine to any and all placebo recipients who want it.15 The studies are supposed to go on for a full two years, but by eliminating the control group, determining effectiveness and risks is going to be near impossible.

Alright. Lets move on to the definition of gene therapy. As detailed on MedlinePlus.govs What Is Gene Therapy page:16

Gene therapy is an experimental technique that uses genes to treat or prevent disease Researchers are testing several approaches to gene therapy, including: Introducing a new gene into the body to help fight a disease

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently being tested only for diseases that have no other cures.

Here, its worth noting that there are many different treatments that have been shown to be very effective against COVID-19, so it certainly does not qualify as a disease that has no cure. It makes sense that gene therapy should be restricted to incurable diseases, as this is the only time that taking drastic risks might be warranted. That said, heres how the U.S. Food and Drug Administration defines gene therapy:17

Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use. Gene therapy is a technique that modifies a persons genes to treat or cure disease. Gene therapies can work by several mechanisms:

Replacing a disease-causing gene with a healthy copy of the gene

Inactivating a disease-causing gene that is not functioning properly

Introducing a new or modified gene into the body to help treat a disease

November 17, 2020, the American Society of Gene + Cell Therapy (ASGCT) announced COVID-19 Vaccine Candidates Show Gene Therapy Is a Viable Strategy, noting that:18

Two COVID-19 vaccine trials, both of which use messenger RNA (or mRNA) technology to teach the body to fight the virus, have reported efficacy over 90 percent.

These findings, announced by Moderna on Nov. 16 and by Pfizer and its partner BioNTech on Nov. 9 demonstrate that gene therapy is a viable strategy for developing vaccines to combat COVID-19.

Both vaccine candidates use mRNA to program a persons cells to produce many copies of a fragment of the virus. The fragment then stimulates the immune system to attack if the real virus tries to invade the body.

As explained in the ASGCTs video above, mRNA are molecules that contain genetic instructions for making various proteins. mRNA vaccines deliver a synthetic version of mRNA into your cells that carry the instruction to produce the SARS-CoV-2 spike protein, the antigen, that then activates your immune system to produce antibodies. Then theres Modernas trial website,19 where they describe their technology thus:

Typical vaccines for viruses are made from a weakened or inactive virus, but mRNA-1273 is not made from the SARS-CoV-2 virus. It is made from messenger ribonucleic acid (mRNA), a genetic code that tells cells how to make protein, which help the bodys immune system make antibodies to fight the virus.

November 18, 2020, Wired magazine made a big deal about COVID-19 vaccines being genetic vaccines, noting:20

The active ingredient inside their shot is mRNA mobile strings of genetic code that contain the blueprints for proteins. Cells use mRNA to get those specs out of hard DNA storage and into their protein-making factories. The mRNA inside Pfizer and BioNTechs vaccine directs any cells it reaches to run a coronavirus spike-building program.

Importantly, as reported by David Martin, Ph.D.,21,22 Moderna describes its product not as a vaccine, but as gene therapy technology in SEC filings. This is because neither Moderna nor Pfizer make any claims about their products creating immunity or preventing transmission. Additionally, Modernas SEC filings specifically state that Currently, mRNA is considered a gene therapy product by the FDA, as well.23

In a February 2021 article, MIT Technology Review reviewed the history of mRNA technology in general, and Modernas in particular, stating:24

Vaccines were not their focus. At the companys founding in 2010, its leaders imagined they might be able to use RNA to replace the injected proteins that make up most of the biotech pharmacopoeia, essentially producing drugs inside the patients own cells from an RNA blueprint. We were asking, could we turn a human into a bioreactor? says Noubar Afeyan, the companys cofounder

Bloomberg, in August 2020, reported25 that the Moderna vaccine would seek to transform your body into a vaccine-making machine. The New York Times was more to the point. In May 2020, they reported26 that Researchers at two Harvard-affiliated hospitals are adapting a proven form of gene therapy to develop a coronavirus vaccine. Read it again A proven form of gene therapy.

So, to summarize: The definition of genetic is something relating to genes, and the definition of therapy is the medical treatment of a disease. The definition of gene therapy is the process of modifying or manipulating the expression of a gene, or altering the biological properties of living cells.

mRNA are snippets of genetic code that instructs cells to produce proteins. mRNA COVID-19 therapies deliver genetic instructions into your cells, thereby triggering your body to produce a fragment of the virus (the spike protein). So, mRNA vaccines ARE gene therapy. Theres simply no way around this. They fulfill all the definitions of gene therapy and none of the definitions for a vaccine.

Theres yet one more potential problem with the COVID-19 vaccine narrative as a whole, which Martin unpacked in a January 25, 2021, interview on the Wise Traditions podcast (above).27 In it, he explains:

COVID-19 is not a disease. It is a series of clinical symptoms. It is a giant umbrella of things associated with what used to be associated with influenza and with other febrile diseases.

The problem that we have is that in February [2020], the World Health Organization was clear in stating that there should not be a conflation between [SARS-CoV-2 and COVID-19]. One is a virus, in their definition, and one is a set of clinical symptoms. The illusion in February was that SARS-CoV-2 caused COVID-19.

The problem with that definition, and with the expectation, is that the majority of people who test positive using the RT-PCR method for testing, for fragments of what is associated with SARS-CoV-2, are not ill at all. The illusion that the virus causes a disease fell apart. Thats the reason why they invented the term asymptomatic carrier.

In short, SARS-CoV-2 has yet to be definitively proven to be the actual cause of COVID-19. So, a gene therapy that instructs your body to produce a SARS-CoV-2 antigen the viral spike protein cannot even be touted as a preventative against COVID-19, as the two have not been shown to be causally linked.

They have been willfully lying since the inception of this, Martin says in the interview. There is not a causal link between these things It has never even been close to established.

We have a situation where the illusion of the problem is that people say, I dont want to get COVID-19. What they mean is they dont want to get infected with a virus. The problem is those two things are not related to each other. A viral infection hasnt been documented in the majority of what is called cases.

There is no basis for that conflation other than the manipulation of the public. Thats the first half of the problem. The second half of the problem is that what is being touted as a vaccination is not a vaccine. This is gene therapy

What is this doing? Its sending a strand of synthetic RNA into the human being and is invoking within the human being, the creation of the S1 spike protein, which is a pathogen A vaccine is supposed to trigger immunity. Its not supposed to trigger you to make a toxin

Its not somewhat different. Its not the same at all Its not a prohibiting infection. Its not a prohibiting transmission device. Its a means by which your body is conscripted to make the toxin that then, allegedly, your body somehow gets used to dealing with, but unlike a vaccine which is to trigger the immune response this is to trigger the creation of the toxin.

As for why drug companies are misrepresenting this technology, Martin suspects its done exclusively so that they can get themselves under the umbrella of public health laws that exploit vaccination.

Experimental gene therapies do not have financial liability shielding from the government, but pandemic vaccines do, even in the experimental stage, as long as the emergency use authorization is in effect. This is indeed a major incentive to make sure this technology is perceived as a vaccine and nothing else.

So, by maintaining the illusion that COVID-19 is a state of emergency, when in reality it is not, government leaders are providing cover for these gene therapy companies so that they are insulated from any liability.

Ive written many articles detailing the potential and expected side effects of these gene therapy vaccines. If all of this is new to you, consider reviewing How COVID-19 Vaccine Can Destroy Your Immune System, Seniors Dying After COVID Vaccine Labeled as Natural Causes and Side Effects and Data Gaps Raise Questions on COVID Vaccine.

The take-home message here is that these injections are not vaccines. They do not prevent infection, they do not render you immune and they do not prevent transmission of the disease. Instead, they alter your genetic coding, turning you into a viral protein factory that has no off-switch. Whats happening here is a medical fraud of unprecedented magnitude, and it really needs to be stopped before its too late for a majority of people.

If you already got the vaccine and now regret it, you may be able to address your symptoms using the same strategies youd use to treat actual SARS-CoV-2 infection. I review these strategies at the end of Why COVID Vaccine Testing Is a Farce.

Last but not least, if you got the vaccine and are having side effects, please help raise public awareness by reporting it. The Childrens Health Defense is calling on all who have suffered a side effect from a COVID-19 vaccine to do these three things:28

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COVID-19 'Vaccines' Are Gene Therapy - Atlanta Business ...