By delivering a viral payload of gene silencers directly to the heart, scientists have developed a new strategy for regenerating cardiac muscle after damage from a heart attack. Described in a paper published Wednesday in Science Translational Medicine, the approach led to new cell growth and improved heart function in pigs.

The findings, though preliminary, indicate its possible to prod cardiac muscle cells into regenerating, at least long enough to stave off some of the worst after-effects of a heart attack. If the gene therapy bears out as safe and effective in further testing, it might someday be used to address the root cause of heart failure one of the leading causes of death in the U.S.

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The facility will equip AGC Biologics with significant additional capacity: adding 622,000 square feet of operations and office space within six buildings. It is 16 miles from AGC Biologics large-scale stainless steel mammalian facility in Boulder.

AGC Biologics says the new facility will help it expand its end-to-end Cell and Gene Therapy (C>) offering, ensuring security of supply for current and future C> customers.It adds that it plans to hire a 'significant percentage' of the current staff employed by Novartis Gene Therapies at the site

Parent company AGC acquired Italys MolMed in 2020 and has since boosted investment in cell and gene therapy technologies: presenting itself as one of a few CDMOs in the world offering end-to-end services in the space.

In February, AGC Biologics also announced an expansion at the Center of Excellence for Cell and Gene Therapy in Milan, Italy: with increased capacities and viral vector suspension capabilities.

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AGC Biologics to acquire cell and gene therapy manufacturing facility in Longmont, Colorado - BioPharma-Reporter.com

CRISPR gene editing has had a big decade. The technology, which earned two of its discovers a Nobel Prize in 2020, can target and edit genes more easily than its predecessors. Still, as tantalizing (and controversial) as the technologys been over the years, its mostly been developed in the lab.

Thats changing now as a growing number of clinical trials are beginning to test gene therapies in humans.

Early CRISPR trials have focused on hereditary blindness and diseases of the blood, including cancer, sickle cell anemia, and beta thalassemia.

Although cutting-edge, the therapies can be costly and intense. In one trial for sickle cell anemia, doctors remove cells from the body, edit them in a dish, and then infuse them back into the patient. In another trial, practitioners inject the gene editing system directly into target tissues in the eye.

Such approaches wont work as readily for other diseases. So researchers and doctors are looking for a general delivery method, like any other medication. Now, a clinical trial from University College London (UCL) has taken a step in that direction.

Participants in the trial suffer from a condition called hereditary transthyretin amyloidosis, in which a mutated gene produces a malformed protein (transthyretin) that builds up in and damages the heart and nerves. The disease is eventually fatal.

Patients received a single infusion of a CRISPR-based therapy into their bloodstream. Blood carried the therapy to the liver, where it switched off the mutated gene and curtailed production of the errant protein. Though the Phase 1 trial was small, the approach had strong results relative to existing options. And it hints at the possibility other genetic diseases may be treated in a similar fashion in the future.

The University of California, Berkeleys Jennifer Doudna, who shared the Nobel Prize for CRISPR, cofounded Intellia, the company that, alongside fellow biotech company Regeneron, developed the treatment (NTLA-2001) used in the UCL trial.

This is a major milestone for patients, Doudna said. While these are early data, they show us that we can overcome one of the biggest challenges with applying CRISPR clinically so far, which is being able to deliver it systemically and get it to the right place.

The therapy is made up of three parts. A tiny bubble of fat, called a lipid nanoparticle, carries a payload of CRISPR machinery: a strand of guide RNA and a sequence of mRNA coding for the Cas9 protein.

Billions of these CRISPR-carrying nanoparticles are infused into the bloodstream, making their way to the liver, the source of the dysfunctional protein. The mRNA instructs the cells to produce the Cas9 protein (CRISPRs genetic scissors) which then links up with the guide RNA, seeks out the target gene, and snips it.

The cell repairs the DNA at the site of the break, but imperfectly, switching the gene off and shutting down production of the problematic protein.

Interim trial results, reported in the New England Journal of Medicine last weekend, were very encouraging. The trials six patients, who received either a low or high dose, reported no serious side effects. Meanwhile, production of the target protein declined by up to 96 percent (and an average of 87 percent) in those given the high dose.

The disease, which affects some 50,000 people worldwide, was untreatable until recently.

Existing drugs, approved by the FDA in 2018, silence the mRNA that produces the malformed thyretin protein, instead of altering its gene. They reduce protein production some 80 percent and keep people alive longer, but dont work for everyone and require ongoing treatment.

The CRISPR approach, if successful, would be a one-time treatment. That is, by targeting the genes themselves, the protein is permanently silenced.

Patrick Doherty, a trial participant, told NPR he jumped at the opportunity.

Doherty, an avid trekker and hiker, was diagnosed with transthyretin amyloidosiswhich had killed his fatherafter noticing symptoms, like tingling fingers and toes and breathlessness on walks.

Its [a] terrible prognosis, he said. This is a condition that deteriorates very rapidly. Its just dreadful.

Doherty started feeling better a few weeks after the treatment and said improvements have continued.

A one-hit wonder, he called it. A two-hour process, and thats it for the rest of your life.

Although the results are promising, theres reason to temper expectations.

The trial, as noted was small, and focused on safety. Future work will further test safety and efficacy in larger groups, whichas is apparent from recent experience with covidcan reveal rare side effects or prove disappointing despite early success.

Researchers will likely also look out for off-target snips in the liver or other cells. A benefit of this approach, however, is the cells break down the mRNA after theyve made the Cas9 protein. In other words, the gene-editing system doesnt persist long.

It also remains to be seen whether the approach would work as well in other diseases. The liver was a prime target for the trial because it greedily soaks up foreign substances. Other organs and tissues may not be as amenable to a general infusion of the therapy.

Finally, the treatment may come with a hefty price tag, perhaps running into the hundreds of thousands of dollars according to Bloomergs Sam Fazeli.

Not one person in my field is doing a victory lap, even around their laboratory bench,Fyodor Urnov, a University of California, Berkeley gene editing expert, told US Today. Were all slightly blue because were all holding our breath.

If the trial does prove successful, however, researchers will want to know if they can reach any organ or target tissue with a general infusion. And can genes also be edited in vivo? Instead of merely knocking out a faulty gene, can we safely correct it?

In the future, when the kinks have been worked out and the science more thoroughly proven, Urnov said, gene editing could help millions of people around the world with genetic conditions. And this trial, it seems, is a notable step in that direction.

Image Credit: NIH

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Breakthrough CRISPR Gene Therapy Could Be a 'One and Done' Injection - Singularity Hub

Viralgen, the gene therapy CDMO coinedby Bayers Asklepios BioPharmaceutical, is wading into commercial waters.

The Spanish contractor on Wednesday said itcut the ribbon on the first $83 million piece of itsnew factory in the Basque Country, Spain. Viralgen, which specializes in adeno-associated virus (AAV) vectorsthe engineered viruses used to deliver gene therapieshas been helping customers with preclinical through phase 2 projects since 2018.

With the new plant, located in the same San Sebastian technology park as Viralgens existing facility, the company will now be able to tackle projects from pre-clinical stages all the way through commercial medicines, it said.

Thanks to the new plant, Viralgen has expanded its capacity fivefold. The company expects the site will release its first commercial-grade 2,000-liter batches by the middle of next year. Throughout 2021, the company will work to validate its equipment and score certifications.

RELATED:With $71M investment, French CDMO Yposkesi set to double capacity and become a viral vector force

Viralgen has invested more than 70 million($83 million) for the first phase of its manufacturing upgrade. Itplans to drop a total of 120million ($142 million) as it works to buildtwo more buildings there. The company will recruit130 workers to staff thefacility and expects its headcount at the site to surpass 250 by 2022.

Viral vectors are needed for many next-gen drugs, from CAR-T cell therapies and gene therapies to certain COVID-19 vaccinesand thats causing big problems for the industry. The vector shortage is already upon us, and its set to worsen in the coming years unless regulators, biopharmas and contract manufacturers move fast to address shortfalls, GlobalData said in a recent report.

RELATED:CDMO Vibalogics, speeding toward U.S. commercial plant, pumps $50M into viral vector capacity

Answering the call, Viralgen'splant will boastthe distinction of being one of the "largest" AAV factories in the world once it's complete, Viralgen says on its website. The company says it will use the facility to help "democratize" access to gene therapies.

Columbus Venture Partners and Asklepios, also known as AskBio, founded Viralgen in 2017 in a bid to tackle mounting production demands in the gene therapy arena. As of last fall, both Viralgen and AskBio now fly the Bayer banner.

In October, Bayer struck a deal to acquire AskBioand Viralgen by extensionfor $2 billion upfront, plus another $2 billion in milestones. The deal will allow Bayer to tap into AskBios AAV gene therapy platform and a pipeline of clinical-phase treatments for Parkinsons disease, Pompe disease and congestive heart failure.

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Bayer's gene therapy CDMO Viralgen cuts ribbon on first phase of $142M viral vector expansion - FiercePharma

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