Category Archives: Gene therapy

The NHS has reported treating its first patient with Novartis Luxturna (voretigene neparvovec) a revolutionary new gene therapy that can restore eyesight, as part of its NHS Long Term Plan.

The therapy is for those born with an inherited retinal disorder - Lebers Congenital Amaurosis (LCA) - who have poor sight which swiftly deteriorates, with many ultimately losing their vision completely in childhood.

The life-changing treatment for children and adults is the first in a new generation of gene therapies that can be directly administered to patients, in this case through an injection. Many patients in the trials have recovered their night time vision with the treatment.

Jake Ternet, patient at Moorfields Eye Hospital was the first in the UK to receive the treatment.

Professor Stephen Powis, NHS medical director, said:Loss of vision can have devastating effects, particularly for children and young people, but this truly life changing treatment offers hope to people with this rare and distressing condition.

Once again, the NHS is at the forefront of the genomic revolution with patients in England among the first to benefit from this new form of treatment a modern day miracle as part of the Long Term Plan.

Back in September last year, The National Institute for Health and Care Excellence (NICE) recommended the use of Luxturna on the NHS for certain patients with RPE65-mediated inherited retinal dystrophies in those with vision loss.

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First patient undergoes Luxturna gene therapy on NHS - PharmaTimes

February 21, 2020

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Enrollment is complete in the two highest dose groups in a phase 1/2 clinical trial evaluating an adeno-associated virus-based gene therapy for the treatment of X-linked retinitis pigmentosa, according to a press release from Applied Genetic Technologies Corporation.

The open-label, dose escalation trial is evaluating the safety and efficacy of AGTC-501 in patients with XLRP due to a mutation in the RPGR gene.

We expect that the information that will be obtained from the two additional dose groups will help to reinforce the data generated to date and previously reported in September 2019 and January 2020, Sue Washer, president and CEO of AGTC, said in the release. We plan to report interim data from these two new dose groups and to report top-line 12-month data for the first four dose groups in the second half of 2020 and intend to initiate a pivotal trial by the end of the year.

In January, the company announced 6-month preliminary data showing a favorable safety profile in all 25 patients treated with AGTC-501.

Enrollment is complete in the two highest dose groups in a phase 1/2 clinical trial evaluating an adeno-associated virus-based gene therapy for the treatment of X-linked retinitis pigmentosa, according to a press release from Applied Genetic Technologies Corporation.

The open-label, dose escalation trial is evaluating the safety and efficacy of AGTC-501 in patients with XLRP due to a mutation in the RPGR gene.

We expect that the information that will be obtained from the two additional dose groups will help to reinforce the data generated to date and previously reported in September 2019 and January 2020, Sue Washer, president and CEO of AGTC, said in the release. We plan to report interim data from these two new dose groups and to report top-line 12-month data for the first four dose groups in the second half of 2020 and intend to initiate a pivotal trial by the end of the year.

In January, the company announced 6-month preliminary data showing a favorable safety profile in all 25 patients treated with AGTC-501.

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Enrollment complete in two more cohorts in X-linked retinitis pigmentosa gene therapy trial - Healio

Moorfields Eye Hospital has become the first NHS trust to offer Luxturna gene therapy for RPE65 retinal dystrophy.

NHS England has agreed a discounted price for the therapy with manufacturer Novartis. The therapy costs around 600,000 privately.

Moorfields patient Jake Ternent, 23, became the first patient to receive Luxturna on the NHS.

The Durham resident described the treatment as a golden opportunity.

I hope the treatment can improve my night vision, and even possibly my day vision, which would be incredible. I feel lucky and privileged to get this on the NHS, Mr Ternent said.

Moorfields Eye Hospital consultant ophthalmologist, Professor James Bainbridge, highlighted that, the ability to provide gene therapy is a major landmark in NHS care.

This is the first of a whole new generation of treatments for sight impairment made available by the NHS with the expectation of changing lives, he emphasised.

Luxturna has been approved by The National Institute for Health and Care Excellence. It is estimated that just under 90 people in England will be eligible for the treatment.

Image credit: Pixabay/Jason Shivers

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The ability to provide gene therapy is a major landmark in NHS care - AOP

John Hopkins University will use Orgenesis point-of-care processing technology for cell and gene therapy research in a collaboration announced this week.

The US research University has licensed access to Orgenesiss point-of-care (PoC) platform which as the name suggests is used to develop and process cell therapies in the clinic

Orgenesis CEO Vered Caplan said, Our POCare platform is designed to provide unique cell and gene therapy solutions in a cost effective, high quality and scalable manner, using closed systems and other advanced cell processing technologies at the point of care.

Image: Tingtingou/creativecommons

Caplan added the aim is to support John Hopkins Universitys growing development and processing needs in order to advance and accelerate cell and gene based clinical therapeutic research.

News of the collaboration comes weeks after Orgenesis sold Masthercell, its third-party cell and gene therapy business, to Catalent for $315 million (285 million).

At the time Orgenesis said it expects to use the net proceeds from the sale of Masthercell to grow its point-of-care cell therapy business

Caplan said, We decided it was the right time to sell Masthercell to maximize value for our shareholders, and focus our efforts around our POCare solutions, which we believe represent a major paradigm shift and will play a major role in the future of the cell and gene therapy market.

She explained that by helping healthcare providers switch from costly, centralized manufacturing models to a localized point-of-care model it can reduce costs and accelerate cell therapy development.

The point of care platform is designed to collect, process and supply cells within the patient care setting for various therapeutic treatments.

The aim is to reduce cost and complexity of supplying cell and gene therapies, as well as elevate quality standards by integrating automated processing.

John Hopkins University is the third institution to license use of the technology.

In January, Orgenesis announced the University of California, Davis will use the platform to develop, commercialize and supply cell and gene products and therapies.

Prior to that Orgenesis joint venture with Theracell signed an agreement with Greece-based Hygeia Group covering use of the platform at three hospitals owned by the latter organization.

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Orgenesis teams with John Hopkins' in PoC deal - Bioprocess Insider - BioProcess Insider

Xtalks Life Science Webinars

TORONTO (PRWEB) February 21, 2020

Join Forrest Brown, Engineering Services Manager Northeast Region and Jarred Christofaro, Engineering Services Manager Southeast Region of Sequence, Inc. for an informative session on Wednesday, February 26, 2020 at 1pm EST to learn about:

Cell and Gene Therapy production is one of fastest growing areas in biotechnology. There is increased demand to bring manufacturing spaces online quickly and with enough flexibility to accommodate process changes, especially for contract manufacturers. While this is becoming easier to accomplish through single-use technology and plug-and-play vendor packages, there are certainly pitfalls to navigate to ensure projects are delivered on-time and within budget.

By paying special attention to these 3 items, projects can be delivered more quickly and in a compliant fashion, greatly reducing business risk and ultimately leading to a more profitable operation. Topics of discussion will stem from both direct project experience and industry guidance from organizations such as ISPE. The use of a risk-based approach will be highlighted, as this is one of the most key elements to reducing cost and adhering to schedule. While this oftentimes means early involvement from various groups, establishing risks to the product/process early in the project lifecycle reduce costly design changes and allow for a compliant, hyper-focused C&Q plan which can meet tight schedule constraints. More attention to this at the beginning of a project ultimately saves time at the end when adherence to schedule is much more critical.

Cell and Gene Therapy projects are especially challenging. The cutting-edge nature oftentimes makes for tight timelines, but also requires a lot of flexibility considering how rapidly technology is changing. This impacts all areas of the project lifecycle, and this webinar will discuss some of the areas where key improvements can be made to save time and money.

For more information or to register for this event, visit Cell and Gene Therapy: Taking a New Facility from Construction to Production.

ABOUT XTALKS

Xtalks, powered by Honeycomb Worldwide Inc., is a leading provider of educational webinars to the global life science, food and medical device community. Every year thousands of industry practitioners (from life science, food and medical device companies, private & academic research institutions, healthcare centers, etc.) turn to Xtalks for access to quality content. Xtalks helps Life Science professionals stay current with industry developments, trends and regulations. Xtalks webinars also provide perspectives on key issues from top industry thought leaders and service providers.

To learn more about Xtalks visit http://xtalks.com

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Cell and Gene Therapy: Taking a New Facility from Construction to Production, Upcoming Webinar Hosted by Xtalks - PR Web

Image caption Matthew Wood hopes the gene therapy will help him keep his remaining vision

A new gene therapy has been used to treat patients with a rare inherited eye disorder which causes blindness.

It's hoped the NHS treatment will halt sight loss and even improve vision.

Matthew Wood, 48, one of the first patients to receive the injection, told the BBC: "I value the remaining sight I have so if I can hold on to that it would be a big thing for me."

The treatment costs around 600,000 but NHS England has agreed a discounted price with the manufacturer Novartis.

Luxturna (voretigene neparvovec), has been approved by The National Institute for Health and Care Excellence (NICE), which estimates that just under 90 people in England will be eligible for the treatment.

The gene therapy is for patients who have retinal dystrophy as a result of inheriting a faulty copy of the RPE65 gene from both parents. The gene is important for providing the pigment that light sensitive cells need to absorb light. Initially this affects night vision but eventually, as the cells die, it can lead to complete blindness.

An injection is made into the back of the eye - this delivers working copies of the RPE65 gene. These are contained inside a harmless virus, which enables them to penetrate the retinal cells. Once inside the nucleus, the gene provides the instructions to make the RPE65 protein, which is essential for healthy vision.

Matthew Wood started losing his sight as a child, and is now registered blind. However, he does have some peripheral vision and can detect large objects and bright lights. He told the BBC: "Since I was a child I was continually told there was no treatment for this condition, so it's amazing to receive this gene therapy."

Mr Wood, from London, had his right eye treated during an hour-long operation at the John Radcliffe Hospital in Oxford.

His left eye will be injected in a few weeks. The surgery was carried out by Prof Robert MacLaren, who has pioneered research into gene therapies for preventing blindness.

He told the BBC: "This is very exciting - this is the first approved NHS gene therapy for an eye disease, but there are opportunities to use gene therapy to treat other diseases in future, not only in the eye."

The treatment is only suitable for patients who have some remaining vision. It should bring the biggest benefits to children with RPE65 retinal dystrophy, as it could halt sight loss before permanent damage is done.

It is not known how long the benefits of the treatment will last, but it's thought it could be several decades.

Jake Ternent, 23, from Durham, had his gene therapy at Moorfields Eye Hospital in London.

Like Matthew Wood, he is registered blind, but has some limited sight. He told the BBC: "I hope the treatment could improve my night vision, and possibly even my day vision, which would be incredible. I feel lucky and privileged to get this on the NHS."

Prof James Bainbridge - from Moorfields Eye Hospital - who treated Jake, told the BBC: "To be at the point now where we are able to offer this treatment on the NHS, is truly remarkable. This is the first example of what's anticipated to be a whole new generation of treatments."

It will take a month or two before Matthew and Jake know what changes the gene therapy has made to their vision. But even if it simply prevents further sight loss, both say they will be delighted.

Professor Stephen Powis, NHS medical director, said: "Loss of vision can have a devastating effect, particularly for children and young people, but this truly life-changing treatment offers hope to people with this rare and distressing condition."

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Gene therapy to halt rare form of sight loss - BBC News

The incidence of cancer has increased tremendously in the past few years. With advancements in science and technology, the treatment for cancer has become efficient and affordable. However, there are some rare and complicated forms of cancer that require better treatments to increase success rates. Recently, cancer gene therapy has showed promising results in preclinical trials. This is a promising trend for the global cancer gene therapy market.

Gene therapy is a method of adding or replacing a gene in an organisms DNA. In some forms of cancer, there is a defective or malfunctioning gene. Nucleic acid is administered to an individual like a drug to correct the gene sequence. Globally, there is a huge thrust on gene therapy research, and it is boosting the growth in the cancer gene therapy market.

Key driving factors of the global cancer gene therapy market

Chemotherapy and radiation, along with surgery are some of the prominent treatments available for cancer today. In cases of early detection, doctors recommend surgery along with chemotherapy or radiation. But, if the cancer has metastasized (spread to other organs), then surgery is difficult. This calls for advanced treatments, a positive factor for the global cancer gene therapy market

While chemotherapy has its own advantages, it has several side effects on the patients. It causes weight loss, vomiting, hair loss, nausea, and other complications in the body. That is the reason why doctors are better treatment options. This is a boost for the global cancer gene therapy market

Radiation also has severe impact on the body. It may cause hair loss, nausea, vomiting, fatigue and skin problems depending on the area of radiation. It also results in lung and heart problems. These factors are favorable for the global cancer gene therapy market

The survival rate of some cancers like lung, pancreatic, and liver is very less. Their chemotherapy treatments have severe side effects. Researchers feel that cancer gene therapy can improve the quality of the treatment. These findings are pushing the demand in the global cancer gene therapy market

With promising in vivo (laboratory) results, many pharmaceutical companies have embarked upon research to develop cancer gene therapy. Investments in R&D have soared in the past few years, a welcoming trend for the global cancer gene therapy market

Cancer has become more prevalent than ever today. With rising number of people seeking treatment for this disease, the demand for quality treatment is expected to grow in the coming years. This is a prime growth factor for the global cancer gene therapy market

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Global Cancer Gene Therapy Market: Key Segments

Based on type, the cancer gene therapy market is segmented into gene transfer immunotherapy and oncolytic virotherapy. Immunotherapy uses genetically modified cells and viral particles to stimulate the immune system to destroy cancer cells. Immunotherapy include treatment with either cytokine gene delivery or tumor antigen gene delivery.

Oncolytic virotherapy uses viral particles, which replicate within the cancer cell causing the death of the cell. It is an emerging treatment modality that is expected to shows great promise, particularly in metastatic cancer treatment. It includes treatment with adenovirus, retrovirus, lentivirus, herpes simplex virus, adeno-associated virus, simian virus, alphavirus, and vaccinia virus.

Gene transfer is the newest treatment modality that is expected to introduce new modified genes into cancerous cell or associated tissue for destruction of cell or to slow down cancer growth. This technique is flexible as a wide variety of vectors and genes are used for clinical trials with positive outcomes. As gene therapy advance, they could be used alone or in combination with other treatments to control the disease. Gene transfer or gene replacement is performed using naked/plasmid vectors, electroporation, sonoporation, magnetofection, and gene gun.

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Global Cancer Gene Therapy Market: Regional Analysis

Based on region, the global cancer gene therapy market is segmented into North America, Europe, Asia Pacific, Latin America and Middle East & Africa. North America is anticipated to hold the largest market share. The U.S. dominates the cancer gene therapy market owing to its increase in funding for research & development and other government initiatives.

Key players in the biotech industry are engaging in research & development of gene therapy products. Moreover, rising demand for DNA vaccines and growing interest of venture capitalists to investment in commercialization of gene-based cancer therapies are likely to propel the market. The cancer gene therapy market in Asia Pacific is anticipated to expand at a rapid pace as in China cancer gene therapy is anticipated to attribute for largest revenue, due to the recent launch of Gendicine and rising healthcare expenditure with strong R&D facilities.

Global Cancer Gene Therapy Market: Key Players

Key players operating in the global cancer gene therapy market are Adaptimmune, ZioPharm Oncology Altor Bioscience, MolMed, bluebird bio, Shanghai Sunway Biotech Company limited, MultiVir, Shenzhen SiBiono GeneTech, and Corporation.

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Promising In Vitro Results to Fillip Global Cancer Gene Therapy Market - BioSpace

Bill Gates

Microsoft founder Bill Gates thinks artificial intelligence and gene therapy are the two technologies with the greatest power to change lives. In a speech Friday at the American Association for the Advancement of Science, Gates said AI can "make sense of complex biological systems," while gene-based tools have the potential to cure AIDS.

The potential of AI is only just being realized now, the billionaire philanthropistsaid, with computational power doubling every three and a half months. Along with improvements in handling data, Gates said it's enabling "the ability to synthesize, analyze, see patterns, gain insights and make predictions across many, many more dimensions than a human can comprehend."

Gates said the most exciting part of AI "is how it can help us make sense of complex biological systems and accelerate the discovery of therapeutics to improve health in the poorest countries."

Gene-editing technologies will meanwhile help with vaccines, diagnostics and therapeutics, Gates said. "[It] has the potential to improve health -- not only for rare genetic disorders, but also for diseases that predominately afflict people in poor countries."

Gates also addressed the deadly coronavirus, saying these two technologies could help with diagnostic tests, treatment and vaccine development.

"Our foundation has committed up to $100 million to address this new coronavirus because we believe it poses a serious threat to global health," Gates said. "This money will support efforts to detect, isolate and treat confirmed cases, help countries in sub-Saharan Africa and South Asia take steps to prepare for the epidemic and protect their most vulnerable citizens, and accelerate the development of vaccines, treatments and diagnostics."

Read more from the original source:
Bill Gates: AI and gene therapy have the power to save lives - CNET

A University of Delaware professor is receiving grant funding to develop a new therapy for people with low blood platelet counts and other blood disorders.

When patients dont have enough blood platelets, they cant form clots and this can sometimes be a lethal condition. These patients rely on platelet donations as a treatment, but those are often in short supply since they cannot be frozen and are only good for a few days.

A few years ago, UDs professor of Chemical and Biomolecular Engineering Terry Papoutsakis discovered a way to get patients to produce more blood platelets in their own bloodstream by introducing microparticles made from cultured stem cells.

These microparticles can be frozen to be used on a large scale. And Papoutsakis says they could also be used to treat genetic blood disorders like primary immune deficiencies.

To be able to use those particles for applications in gene therapy for a variety of blood diseasesa lot of genetic diseases or acquired diseases for patients, said Papoutsakis. So thats a difficult problem to deal with and we think this has great promise.

Papoutsakis is being awarded $250,000 through a partnership between University City Science Center and CSL Behring meant to find new biotherapies. He says the award took a few years to secure as a result of contract negotiations around intellectual property rights.

It took a little while to do it, but that is to be understood that it would, because of the potential that this might develop into something quite bigger than we currently see, he said.

Papoutsakis says he has already had some success testing his discovery on mice. He says the award will fund a continuation of those tests, moving the therapy closer to clinical trials on human patients.

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UD professor working to use gene therapy to treat blood disorders - Delaware First Media

In 2018, Pfizer initiated a Phase Ib clinical trial of its gene therapy treatment for Duchenne muscular dystrophy (DMD). The AAV9 gene therapy, PF-06939926, is designed to provide DMD patients with a form of the dystrophin gene at the root of the condition, thereby countering the progressive muscle degeneration and weakness that characterizes the disease.

Pfizer planned to dose 15 patients with the gene therapy, which must be stored at -70C. In light of those factors, Pfizer opted for a patient-led supply model that would only ship product once a subject was enrolled and ready for treatment.

The model made the lag between a site requesting and receiving product critical, as during that time patients with a progressive disease would be waiting for a potentially beneficial treatment. Pfizers goal was to package and ship the gene therapy to patients within two weeks.

That goal created challenges. Typically, the lag between the ordering and shipment of trial materials is six to eight weeks. Almac had already reduced that lead time by applying LEAN packaging and labelling principles but needed to shave a further two weeks off to meet Pfizers demands.

To do so, Almacs clinical services unit developed a dedicated packaging and labelling process. The process, which Almac executed at its US facility in Souderton, Pennsylvania, resulted in a 12-day lead time for the first patient enrolled in the trial.

Almac provided the packaging specification within two business days. Pfizer granted approval in one working day. Packaging and labelling took place four to five days after receipt of the initial request.

The case study presented by Almac covers the shipment of a gene therapy to a single patient. Yet, the process it describes has broader relevance for drug developers and contract packagers in an era defined by therapies targeting small patient populations.

In the four years preceding late 2019, the number of clinical trials of advanced therapy medicinal products (ATMP), such as cell and gene therapies, increased by two thirds, according to data tracked by the Alliance for Regenerative Medicine. Many ATMPs place new pressures on supply chains, which are adapting to quickly get medicines to patients.

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Almac and Pfizer slash gene therapy labeling times - OutSourcing-Pharma.com