Archive for the ‘Gene Therapy Research’ Category

AMSTERDAM--(BUSINESS WIRE)--VectorY Therapeutics, a biotech company focusing on the development of innovative gene therapy approaches for the treatment of muscular and neurodegenerative disorders through vectorized antibodies, today announces a collaboration with Wageningen University to develop novel baculovirus-based technologies for the production of safe and affordable AAV gene therapies.

One of the most important challenges for the gene therapy industry is to develop robust and scalable manufacturing processes that yield safe therapies. VectorY, together with its partner Wageningen University, is making an important strategic investment in these therapies of the future, by developing a production platform capable of successfully manufacturing safe products at a significant lower COGS. The collaboration represents an important step to enable the medical and economical feasibility of gene therapy for diseases that affect larger patient populations.

Under the terms of the collaboration, the Bioprocess Engineering and Virology Groups at Wageningen University and VectorY Therapeutics will work together on two projects. One will utilize the molecular toolbox to generate innovative stable baculovirus genome seeds for AAV production at large scale. The second will focus on the design and evaluation of an intensified and scalable baculovirus production process in bioreactors using state of the art bioprocess technologies.

We are very pleased to be collaborating with VectorY to develop proprietary, next generation AAV expression systems and industry-leading bioprocessing capabilities, said Monique van Oers, Professor of Virology, Wageningen University.

We are honored to partner with Wageningen University, a significant pioneer in the Baculovirology and Bioprocessing field. This partnership will further strengthen VectorYs proprietary AAV production technologies for the development of Next Generation Gene Therapies, added Alexander Vos, CEO VectorY Therapeutics.

ENDS

Notes to Editors

About VectorY

VectorY combines the therapeutic potential of antibodies and gene therapy to develop long-lasting therapeutic solutions for muscular and neurodegenerative diseases with high unmet medical need. Founded in October 2020, and based in the Amsterdam Science Park, VectorY is a fully integrated gene therapy company focused on the development of innovative therapeutics based on a novel AAV gene therapy platform andantibody- based targeted degradation technologies, and proprietary manufacturing technology. VectorY develops proprietary & partnered programs and product candidates are based on new technologies that will enable the next generation of highly scalable manufacturing processes within VectorYs own manufacturing facilities. VectorYs manufacturing capabilities will include a state-of-the-art multi-product GMP facility in the Netherlands, with the capability to deliver suspension based AAV viral vector manufacturing of up to 2000L for both clinical and commercial supply.

For more information, see http://www.vectorytx.com

Wageningen University & Research

The mission of Wageningen University and Research is To explore the potential of nature to improve the quality of life. Under the banner Wageningen University & Research, Wageningen University and the specialised research institutes of the Wageningen Research Foundation have joined forces in contributing to finding solutions to important questions in the domain of healthy food and living environment. With its roughly 30 branches, 6.800 employees and 12.900 students, Wageningen University & Research is one of the leading organisations in its domain. An integrated approach to problems and the cooperation between various disciplines are at the heart of Wageningens unique approach. As such, the Laboratory of Virology and The Bioprocess Engineering group work closely together in a number of biotechnological projects aimed at the further development of the baculovirus expression system for vaccine production and gene therapy applications. By combining our research efforts we can optimally profit from knowledge gained in fundamental virology and new technological developments in bioprocess engineering.

For more information, see https://www.wur.nl/en.htm

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VectorY and Wageningen University Sign Strategic Collaboration for the Development of Novel Baculovirus-based AAV Production Technologies - Business...

The human heart, the size of a fist, located just behind and slightly left of the breastbone, tirelessly beats an average of 100,000 times a day. However, conditions that stop the heart from pumping blood efficiently can cause serious problems and ultimately require a heart transplantation.

In a study published in the journal 'Science Translational Medicine', researchers from Osaka University showed that a previously unknown mutation can lead to a condition called dilated cardiomyopathy, which is one of the main causes of heart failure.

Heart failure refers to an incurable condition where the heart is no longer able to meet the body's demands in terms of blood supply. It is one of the most common causes of death and it affects almost 40 million people worldwide, representing a huge public health problem. One of the main factors leading to heart failure is a disease called dilated cardiomyopathy (or DCM). DCM is characterized by dilation of the heart's chambers and a pumping disfunction. Previous research has shown that DCM is often inherited and has a genetic basis. However, for up to 80 per cent of the familial DCM cases, the genetic mutation causing the disease has still not been known.

ALSO READ: Can Omicron cause heart damage? Here's what experts say

The research team identified a gene called BAG5 as a novel causative gene for DCM. First, they studied patients from different families, highlighting a correlation between loss of function mutations in the BAG5 gene and DCM. The researchers found that this mutation has a complete penetrance, meaning that 100 per cent of the individuals presenting it will develop the disease. They then found in a mouse model of dilated cardiomyopathy that mice without BAG5 exhibited the same symptoms of human DCM, such as dilatation of the heart's chambers and irregular heart rhythm. This indicated that mutations that erase the function of BAG5 can cause cardiomyopathy.

"Here we showed that loss of BAG5 perturbs calcium handling in mouse cardiomyocytes," said Dr. Hideyuki Hakui, lead author of the study. BAG5 is important for calcium handling in the heart muscle cells, and calcium is essential for a regular rhythm and overall health of the cardiac muscle, explaining why a loss of BAG5 leads to cardiomyopathy.

"After demonstrating that BAG5 mutations led to loss of functional BAG5 protein," continued Dr. Yoshihiro Asano, senior author of the study, "we also showed that administration of an AAV9-BAG5 vector in a murine model could restore cardiac function. This finding suggests that gene therapy with adeno-associated viruses (AAV) should be further investigated as a possible treatment alternative to heart transplantation for patients who are BAG5 deficient." AAV gene therapy refers to an innovative form of therapy aimed at fixing mutated genes in diseases that have a genetic cause like DCM. Therefore, these findings have paved the way for a potential precision medicine treatment based on gene therapy.

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Not just bones, calcium is vital for heart too: Study - Hindustan Times

New Jersey, United States,-The research study presented in this report offers a complete and intelligent analysis of the competition, segmentation, dynamics, and geographical advancement of the Gene Therapy Cell Culture Media Market. It takes into account the CAGR, value, volume, revenue, production, consumption, sales, manufacturing cost, prices, and other key factors related to the Gene Therapy Cell Culture Media market. The authors of the report have segmented the Gene Therapy Cell Culture Media market as per product, application, and region. Segments of the Gene Therapy Cell Culture Media market are analyzed on the basis of market share, production, consumption, revenue, CAGR, market size, and more factors. The analysts have profiled leading players of the Gene Therapy Cell Culture Media market, keeping in view their recent developments, market share, sales, revenue, areas covered, product portfolios, and other aspects.

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The report includes company profiling of almost all important players of the Gene Therapy Cell Culture Media market. The company profiling section offers valuable analysis on strengths and weaknesses, business developments, recent advancements, mergers and acquisitions, expansion plans, global footprint, market presence, and product portfolios of leading market players. This information can be used by players and other market participants to maximize their profitability and streamline their business strategies. Our competitive analysis also includes key information to help new entrants to identify market entry barriers and measure the level of competitiveness in the Gene Therapy Cell Culture Media market.

Key Players Mentioned in the Gene Therapy Cell Culture Media Market Research Report:

Fujifilm Holdings Corporation, HiMedia Laboratories Pvt., Ltd, Lonza Group Ltd, Sartorius AG, Thermo Fisher Scientific Inc., Merck KGaA, Danaher Corporation, Takara Holdings Inc., Novartis International AG, Bio-Techne Corporation.

Gene Therapy Cell Culture MediaMarket Segmentation:

Gene Therapy Cell Culture Media Market, By Media Type

Serum-containing Media Serum-free Media Stem Cell Media Specialty Media Chemically Defined Media

Gene Therapy Cell Culture Media Market, By End User

Biotechnology & Pharmaceutical Industry Academic Institute Research Laboratory

The Gene Therapy Cell Culture Media market is segmented as per the type of product, application, and geography. All of the segments of the Gene Therapy Cell Culture Media market are carefully analyzed based on their market share, CAGR, value and volume growth, and other important factors. The report also provides accurate estimations about the CAGR, revenue, production, sales, and other calculations for the Gene Therapy Cell Culture Media market. Each regional market is extensively studied in the report to explain why some regions are progressing at a high rate while others at a low rate. We have also provided Porters Five Forces and PESTLE analysis for a deeper study on the Gene Therapy Cell Culture Media market

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Gene Therapy Cell Culture Media Market Report Scope

Geographic Segment Covered in the Report:

TheGene Therapy Cell Culture Mediareport provides information about the market area, which is further subdivided into sub-regions and countries/regions. In addition to the market share in each country and sub-region, this chapter of this report also contains information on profit opportunities. This chapter of the report mentions the market share and growth rate of each region, country and sub-region during the estimated period.

North America (USA and Canada) Europe (UK, Germany, France and the rest of Europe) Asia Pacific (China, Japan, India, and the rest of the Asia Pacific region) Latin America (Brazil, Mexico, and the rest of Latin America) Middle East and Africa (GCC and rest of the Middle East and Africa)

Key questions answered in the report:

1. Which are the five top players of the Gene Therapy Cell Culture Media market?

2. How will the Gene Therapy Cell Culture Media market change in the next five years?

3. Which product and application will take a lions share of the Gene Therapy Cell Culture Media market?

4. What are the drivers and restraints of the Gene Therapy Cell Culture Media market?

5. Which regional market will show the highest growth?

6. What will be the CAGR and size of the Gene Therapy Cell Culture Media market throughout the forecast period?

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Gene Therapy Cell Culture Media Market Size | Global Industry Trends, Segmentation, Business Opportunities And Forecast To 2029 The Oxford Spokesman...

Four years ago, a small Philadelphia biotech company won U.S. approval for the first gene therapy to treat an inherited disease, a landmark after decades of research aimed at finding ways to correct errors in DNA.

Since then, most of the world's largest pharmaceutical companies have invested in gene therapy, as well as cell therapies that rely on genetic modification. Dozens of new biotech companies have launched, while scientists have taken forward breakthroughs in gene editing science to open up new treatment possibilities.

But the confidence brought on by such advances has also been tempered by safety setbacks and clinical trial results that fell short of expectations. In 2022, the outlook for the field remains bright, but companies face critical questions that could shape whether, and how soon, new genetic medicines reach patients. Here are five:

Food and Drug Administration approval of Spark Therapeutics' blindness treatment Luxturna a first in the U.S. came in 2017. A year and a half later, Novartis' spinal muscular atrophy therapy Zolgensma won a landmark OK.

But none have reached market since, with treatments from BioMarin Pharmaceutical and Bluebird bio unexpectedly derailed or delayed.

That could change in 2022. Two of Bluebird's treatments, for the blood disease beta thalassemia and a rare brain disorder, are now under review by the FDA, with target decision dates in May and June. BioMarin, after obtaining more data for its hemophilia A gene therapy, plans to soon approach the FDA about resubmitting an application for approval.

Others, such as CSL Behring and PTC Therapeutics, are also currently planning to file their experimental gene therapies with the FDA in 2022.

Approvals, should they come, could provide important validation for their makers and expand the number of patients for whom genetic medicines are an option. In biotech, though, approvals aren't the end of the road, but rather the mark of a sometimes challenging transition from research to commercial operations. With price tags expected to be high, and still outstanding questions around safety and long-term benefit, new gene therapies may prove difficult to sell.

A record $20 billion flowed into gene and cell therapy developers in 2020, significantly eclipsing the previous high-water mark set in 2018.

Last year, the bar was set higher still, with a total of $23 billion invested in the sector, according to figures compiled by the Alliance for Regenerative Medicine. About half of that funding went toward gene therapy developers specifically, with a similar share going to cell-based immunotherapy makers.

Driving the jump was a sharp increase in the amount of venture funding, which rose 73% to total nearly $10 billion, per ARM. Initial public offerings also helped, with sixteen new startups raising at least $50 million on U.S. markets.

Entering 2022, the question facing the field is whether those record numbers will continue. Biotech as a whole slumped into the end of last year, with shares of many companies falling amid a broader investment pullback. Gene therapy developers, a number of which had notable safety concerns crop up over 2021, were hit particularly hard.

Moreover, many startups that jumped to public markets hadn't yet begun clinical trials roughly half of the 29 gene and cell therapy companies that IPO'd over the past two years were preclinical, according to data compiled by BioPharma Dive. That can set high expectations companies will be hard pressed to meet.

Generation Bio, for example, raised $200 million in June 2020 with a pipeline of preclinical gene therapies for rare diseases of the liver and eye. Unexpected findings in animal studies, however, sank company shares by nearly 60% last December.

Still, the pace of progress in gene and cell therapy is fast. The potential is vast, too, which could continue to support high levels of investment.

"I think fundamentally, investment in this sector is driven by scientific advances, and clinical events and milestones," said Janet Lambert, ARM's CEO, in an interview. "And I think we see those in 2022."

The potential of replacing or editing faulty genes has been clear for decades. How to do so safely has been much less certain, and concerns on that front have set back the field several times.

"Safety, safety and safety are the first three top-of-mind risks," said Luca Issi, an analyst at RBC Capital Markets, in an interview.

Researchers have spent years making the technology that underpins gene therapy safer and now have a much better understanding of the tools at their disposal. But as dozens of companies push into clinical trials, a number of them have run into safety problems that raise crucial questions for investigators.

In trials run by Audentes Therapeutics and by Pfizer (in separate diseases), study volunteers have tragically died for reasons that aren't fully understood. UniQure, Bluebird bio and, most recently, Allogene Therapeutics have reported cases of cancer or worrisome genetic abnormalities that triggered study halts and investigations.

While the treatments being tested were later cleared in the three latter cases, the FDA was sufficiently alarmed to convene a panel of outside experts to review potential safety risks last fall. (Bluebird recently disclosed a new hold in a study of its sickle cell gene therapy due to a patient developing chronic anemia.)

The meeting was welcomed by some in the industry, who hope to work with the FDA to better detail known risks and how to avoid them in testing.

"[There's] nothing better than getting people together and talking about your struggles, and having FDA participate in that," said Ken Mills, CEO of gene therapy developer Regenxbio, in an interview. "The biggest benefit probably is for the new and emerging teams and people and companies that are coming into this space."

Safety scares and setbacks are likely to happen again, as more companies launch additional clinical trials. The FDA, as the recent meeting and clinical holds have shown, appears to be carefully weighing the potential risks to patients.

But, notably, there hasn't been a pullback from pursuing further research, as has happened in the past. Different technologies and diseases present different risks, which regulators, companies and the patient community are recognizing.

"We're by definition pushing the scientific envelope, and patients that we seek to treat often have few or no other treatment options," said ARM's Lambert.

Last June, Intellia Therapeutics disclosed early results from a study that offered the first clinical evidence CRISPR gene editing could be done safely and effectively inside the body.

The data were a major milestone for a technology that's dramatically expanded the possibility for editing DNA to treat disease. But the first glimpse left many important questions unanswered, not least of which are how long the reported effects might last and whether they'll drive the kind of dramatic clinical benefit gene editing promises.

Intellia is set to give an update on the study this quarter, which will start to give a better sense of how patients are faring. Later in the year the company is expecting to have preliminary data from an early study of another "in vivo" gene editing treatment.

In vivo gene editing is seen as a simpler approach that could work in more diseases than treatments that rely on stem cells extracted from each patient. But it's also potentially riskier, with the editing of DNA taking place inside the body rather than in a laboratory.

Areas like the eye, which is protected from some of the body's immune responses, have been a common first in vivo target by companies like Editas Medicine. But Intellia and others are targeting other tissues like the liver, muscle and lungs.

Later this year, Verve Therapeutics, a company that uses a more precise form of gene editing called base editing, plans to treat the first patient with an in vivo treatment for heart disease (which targets a gene expressed in the liver.)

"The future of gene editing is in vivo," said RBC's Issi. His view seems to be shared by Pfizer, which on Monday announced a $300 million research deal with Beam Therapeutics to pursue in vivo gene editing targets in the liver, muscle and central nervous system.

With more and more cell and gene therapy companies launching, the pipeline of would-be therapies has grown rapidly, as has the number of clinical trials being launched.

Yet, many companies are exploring similar approaches for the same diseases, resulting in drug pipelines that mirror each other. A September 2021 report from investment bank Piper Sandler found 21 gene therapy programs aimed at hemophilia A, 19 targeting Duchenne muscular dystrophy and 18 going after sickle cell disease.

In gene editing, Intellia, Editas, Beam and CRISPR Therapeutics are all developing treatments for sickle cell disease, with CRISPR the furthest along.

As programs advance and begin to deliver more clinical data, companies may be forced into making hard choices.

"[W]e think investors will place greater scrutiny as programs enter the clinic and certain rare diseases are disproportionately pursued," analysts at Stifel wrote in a recent note to investors, citing Fabry disease and hemophilia in particular.

This January, for example, Cambridge, Massachusetts-based Avrobio stopped work on a treatment for Fabry that was, until that point, the company's lead candidate. The decision was triggered by unexpected findings that looked different than earlier study results, but Avrobio also cited "multiple challenging regulatory and market dynamics."

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5 questions facing gene therapy in 2022 - BioPharma Dive

DUBLIN, Jan. 14, 2022 /PRNewswire/ -- The "Cell and Gene Therapy Business Outlook" report has been added to ResearchAndMarkets.com's offering.

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The Twice-Monthly Publication Cell and Gene Therapy Business Outlook will offer the following:

Market Sizing and Forecasting of CAGT Markets: Each issue sizes up the market opportunity and projects the future revenues for a given therapeutic segment.

Keeping an Eye on Financing: With billions of investment dollars announced each year, Cell and Gene Therapy Business Outlook tracks who is getting financed (and the companies behind the financing) each issue. On a regular basis we will analyze trends in that financing.

News Briefs and Analysis of the Science That will Shape Tomorrow's Business: Cell and Gene Therapy Business Outlook is designed to provide the most relevant news. With a focus on what the recent news of the day means for business, our curated news and news analysis means that you and your organization can be confident you won't miss an important development in cell and gene therapy.

Deals Between CAGT Companies Tracked: Each issue's "Recent Deals Table" tracks the important deals between stem cell companies as well as the deals they engage in (tech transfers, partnerships, mergers, distribution and other activities) with companies outside the industry.

Cell and Gene Therapy Tools: This newsletter will also report on developments, product launches and deals relating to the makers of cell and gene therapy manufacturing equipment and supplies.

Target Audience Includes:

Director, Vice President or Manager of Market Research

Director, Vice President or Manager of Marketing

Director, Vice President or Manager of Research and Development

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Director of Business Development

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"GT and CT have been a hot topic! We are tasked to know this area and need a resource like this"-Consulting Firm

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"The charts and tables are a good idea to keep up with the amount of new announcements in CGT. We appreciated the scientific discussion in this issue and your recent webinar." -Cell and Gene Therapy Startup

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Cell and Gene Therapy Business Outlook Service - Yahoo Finance

BioMarin Pharmaceutical plans to return to the Food and Drug Administration later this year with clinical trial results it says prove its gene therapy for hemophilia can prevent bleeding for years after treatment.

The data from BioMarin's study, disclosed Sunday ahead of the J.P. Morgan Healthcare Conference, are meant to meet requirements laid out by the agency when it rejected the company's previous approval application a year and a half ago.

They show the gene therapy restored blood clotting protein levels to a range consistent with mild hemophilia and, while those levels waned over time, that trial participants experienced very few, if any, bleeds across the two years most were studied. A handful of volunteers in the Phase 3 trial, the largest to date of a hemophilia gene therapy, were followed for three years and had similar results.

"I believe that these results will answer, quantitatively, quite a lot of the questions that agencies have had," said Hank Fuchs, BioMarin's head of research and development, on a conference call Sunday.

Regulators in Europe have already begun evaluating an application from BioMarin and are expected to make a decision in the first half of this year. In the U.S., BioMarin aims to quickly review the results with the FDA and, should the agency agree, potentially resubmit the therapy in the second quarter.

BioMarin's gene therapy, called Roctavian, is the product of years of research by the California biotech and builds on more than a decade of work by other scientists to develop a treatment for hemophilia's genetic cause. It is designed to deliver into the body a functional copy of the gene that's mutated in people with the "A" form of hemophilia, who are left with little or no clotting protein to stem bleeding.

People with severe hemophilia A, who make up about half of all those with disease, must take regular, preventive infusions of "replacement" clotting protein, also known as Factor VIII. Roctavian, which is meant for these individuals, would in theory allow them to stop, freeing them from chronic treatment while more effectively preventing bleeding.

Results from an earlier, much smaller trial showed such promise and, by mid-2020, BioMarin had come close to replicating those findings in the first group of volunteers enrolled in its Phase 3 study. But the FDA unexpectedly, according to the company sought more information to prove that benefit could last two years.

Last January, BioMarin revealed one-year results from all participants in the trial and, on Sunday, disclosed data from its two-year analysis. Treatment decreased the number of bleeds per year by 85%, from an average of nearly five among the 112 volunteers who were studied for at least six months before infusion to less than one at year two.

Among 17 participants who were given Roctavian three years before the analysis was conducted, the average annual bleeding rate remained below one as well.

"Our clinical outcome here is unassailably great," said Fuchs in a separate interview. "It almost makes the application, honestly, bulletproof."

But levels of Factor VIII activity, which had risen sharply to an average of 43 international units per deciliter of blood at one year, declined to 23 IU/dL by year two and, for those 17 participants, 17 IU/dL by year three. BioMarin reported these values using a lab test known as a chromogenic assay, which it says is more conservative than another one also used.

People with severe hemophilia typically have less than one IU/dL of Factor VIII in their blood, while mild hemophilia is typically considered to be between 5 IU/dL and 40 IU/dL.

The decline has been a source of doubt, causing concerns that Roctavian's ability to prevent bleeds might wane over time as well. At least for the first few years, Sunday's results show that isn't happening yet. BioMarin also points to data from an earlier study, in which annualized bleeding rates remained below one through five years, despite reduced Factor VIII activity.

"A small amount of Factor VIII is going to go a long way towards hemostatic efficacy," said Fuchs on Sunday's call, "and it gives us confidence that what we've seen so far in the Phase 2 study is gonna read through to the Phase 3 study when we get there."

Extrapolating efficacy puts BioMarin on somewhat uncertain ground, however, as it is the first company to advance this far with a gene therapy for hemophilia A. Jean-Jacques Bienaime, BioMarin's CEO, argues the data so far for Roctavian indicate treatment should result in at least five years of bleeding control and perhaps even eight or longer.

"With the Phase 2, we have demonstrated at least five years already. Predicting eight years, I don't think, is a big stretch," he said in an interview.

How the FDA will view BioMarin's data is unclear, although analysts on Wall Street predicted the latest results would be enough to merit an approval. The agency could convene a panel of outside experts to review a resubmitted application from the company, a possibility Fuchs acknowledged on the conference call.

Also uncertain is how Roctavian would be perceived by hemophilia patients and by insurers, should it eventually secure an approval. BioMarin has previously suggested a price as high as between $2 million and $3 million, but that might be viewed as high if Roctavian's benefit isn't lifelong. (ICER, a looked-to drug cost watchdog, previously found Roctavian could be cost effective at a price of even $2.5 million.)

Fuchs said the company plans to present more data at a medical meeting, likely this year, that should help clarify the relationship between Factor VIII activity and expected durability of benefit.

Importantly for Roctavian's future, Sunday's data, while relatively sparse, indicated no new safety issues had emerged in testing. There were no cases of "inhibitors," or antibodies that work against clotting protein, developing following treatment, nor were there any cases of cancer or blood clot blockages.

The former two are both newly of interest following reports of cancers developing in other gene therapy trials, and data showing higher-than-normal levels of clotting factor in a trial of another hemophilia gene therapy being developed by Pfizer and Sangamo Therapeutics.

Note: This story has been updated to include mention of the assay used by BioMarin to measure Factor VIII activity, and of ICER's analysis.

Read more:
BioMarin plans return to FDA with updated data on hemophilia gene therapy - BioPharma Dive

NEW YORK, Jan. 12, 2022 /PRNewswire/ -- Polaris Market Research recently published a research report on "Gene Delivery Technologies Market Share, Size, Trends, Industry Analysis Report, By Mode (Biological [Adenovirus, Retrovirus, AAV, Lentivirus, Other Viruses, Non-viral], Chemical, Physical); By Application (Gene Therapy, Cell Therapy, Vaccines, Research); By Method; By Regions; Segment Forecast, 2021 2028" in its online research storage.

According to [127+ Pages] research report published by Polaris Market Research, the global Gene Delivery Technologies Market size & share expected to reach to USD 7.86 Billion by 2028 from USD 2.64 Billion in 2020, at a compound annual growth rate (CAGR) of 15.0% during forecast period 2021 to 2028.

What is Gene Delivery Technology? How big is Gene Delivery Technology Industry?

Gene delivery technology is widely used in gene therapies, which involves transferring of genetic and hereditary disorders. These therapies have also performed an important role in shaping the entire pharma landscape. Around 27 gene therapies were revealed in the marketplace and over 990 companies emphasized the research & development, and commercialization of innovative therapies by 2020. The constantly changing market environment for advanced therapies is reportedly driving the market for gene delivery technologies.

The operating market players are building various business strategies to boost the market for gene delivery technologies, while the developing gene delivery technologies are creating openings for several new players in the market. Different research settings offer market applications for various gene delivery technologies. However, due to technical challenges related to each modal type, the clinical settings produce very few applications. Within the clinical settings, physical technologies require a breakthrough in their use.

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Key Aspects Covered By Report:

Top Market Companies Profiles Covered:

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Industrial Gene Delivery Technology Market: Growth Factors

The adoption of gene therapies and subsequent increase in clinical research activities around the globe has fueled the market growth. Also, the growing acceptance of gene therapy products and services has supported the gene delivery technologies market growth prospects. Other key driving factors of the market involve technological advancements in viral vectors, a rising pipeline of advanced therapies, and a growing number of regulatory approvals for advanced therapy products.

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Global Gene Delivery Technology Market: Key Segmentation

Insight by Mode

In 2020, the biological vectors market segment secured the largest revenue share of the gene delivery technologies industry due to the high success rate of Kymriah and Yescarta. Following the acceptance of vectors-based therapy products, the above-mentioned vectors have experienced greater attention.

The chemical delivery method market segment is expected to secure a lucrative growth rate over the study period. Clinical challenges are observed in viral systems which propelled the chemical methods' use. Chemical delivery systems have replaced viral delivery systems because of their capability in combating challenges.

The physical delivery methods market segment has lower transfection efficiency than biological or chemical modes. One other drawback called low cell viability in electroporation-based physical methods enables other market players to gain more share. It helps them to address a high focus on transfection and cell viability issues.

Also Read, Global Gene Therapy Market Report, 2021-2028

Insight by Method

The ex-vivo market segment of the gene delivery technologies market witnessed the largest share in 2020. Its transduction efficiency is the major factor behind the high share achievement, making it an ideal candidate to be used in research settings.

In this market, the in-vivo delivery method market segment is expected to obtain a lucrative growth rate over the estimation period as it features a high preference for highly targeted gene deliveries. Researchers are extending research & development for the market segment. For example, Oregon Health and Science University built the gene-editing tool "Crispr-Cas9" in 2020, which enables genetic code editing for blind people.

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Gene Delivery Technologies Market: Report Scope

Report Attribute

Details

Market Size 2020 Value

USD 2.64 Billion

Market Outlook for 2028

USD 7.86Billion

Expected CAGR Growth

CAGR 150% from 2021 - 2028

Base Year

2020

Forecast Year

2021 - 2028

Top Market Players

Horizon Discovery Group Co., QIAGEN, Oxford Biomedica, SignaGen Laboratories, Hoffmann-La Roche AG, Vectalys, Sirion-Biotech GmbH and Others

Segments Covered

By Mode, By Application, By Method, By Region

Geographies Covered

North America, Europe, Asia Pacific, Latin America and Middle East & Africa

Customization Options

Customized purchase options are available to meet your research needs. Explore customized purchase options

Geographic Overview: Gene Delivery Technology Market

The North American region registered a significant share of the global gene delivery technology market. Various clinical trials are used to access the efficacy of gene therapies to treat hereditary, cancer, genetic mutations, and rare disorders in the U.S. This factor is the key driver of the gene delivery technologies demand growth in North America.

In addition, the availability of better clinical infrastructure also contributes to market growth. Many companies are marketing gene delivery products and accessories, which will boost the gene delivery technologies industry growth prospects. The U.S. has already announced many research projects combined with other leaders under its Horizon 2020 plans. This project will also cover other vector-based gene delivery trials for rare diseases.

Moreover, the Asia Pacific gene delivery technologies industry is anticipated to account for a profitable gene delivery technology market growth rate over the assessment period. The region is well known for the developed pharmaceutical industry even with its large population size, and low labor costs.

Browse the [127+ Pages] Detail Report "Gene Delivery Technologies Market Share, Size, Trends, Industry Analysis Report, By Mode (Biological [Adenovirus, Retrovirus, AAV, Lentivirus, Other Viruses, Non-viral], Chemical, Physical); By Application (Gene Therapy, Cell Therapy, Vaccines, Research); By Method; By Regions; Segment Forecast, 2021 2028" with in-depth TOC: https://www.polarismarketresearch.com/industry-analysis/gene-delivery-technologies-market

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The market is primarily segmented on the basis of mode, application, method, and geographic region.

Gene Delivery Technology Market: By Mode Outlook

Gene Delivery Technology Market: By Application Outlook

Gene Delivery Technology Market: By Method Outlook

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At 15.0% CAGR, Global Gene Delivery Technologies Market Size Will Reach USD 7.86 Billion By 2028: Polaris Market Research - PRNewswire

EXTON, Pa., Jan. 10, 2022 /PRNewswire/ -- Castle Creek Biosciences, Inc., a late-clinical stage cell and gene therapy company focused on developing and preparing to commercialize disease-modifying and potentially curative therapies for rare genetic diseases, today announced it has acquired Novavita Thera, Inc., a preclinical gene therapy company focused on rare liver and metabolic diseases. The acquisition expands Castle Creek's technology platform by adding in vivo capabilities to its existing ex vivo approach, and broadens Castle Creek's development pipeline beyond skin and connective tissue disorders to rare liver diseases.

"This acquisition is a significant inflection point for Castle Creek and positions us to expand our research and development efforts using a versatile, dual technology platform that will accelerate the discovery of disease-modifying and potentially curative therapies for people living with rare diseases," said Matthew Gantz, president and chief executive officer of Castle Creek Biosciences. "The ability to leverage both ex vivo and in vivo based approaches is a distinct advantage that few cell and gene therapy companies can offer. We are now in position to pursue new indications for devastating rare diseases, while also advancing our ongoing pivotal clinical trial in recessive dystrophic epidermolysis bullosa (RDEB)."

With the acquisition of Novavita Thera, formerly aCytotheryx, Inc., company, Castle Creek will initially develop a gene therapy for hereditary tyrosinemia type 1 (HT1),a rare inborn error of metabolism caused by a lack of the enzyme fumarylacetoacetate hydrolase (FAH) which leads to accumulation of tyrosine and its metabolites in the liver. HT1 affects approximately 1:100,000 live births and leads to cirrhosis, liver failure, hepatocellular carcinoma, and is ultimately fatal if untreated. Liver transplantation is currently the only curative treatment available for HT1.

Castle Creek will advance the development of LV-FAH, a potential therapy based on a lentiviral vector containing a functional copy of the human FAH gene that is administered directly to the patient through the portal vein. The therapy is designed to transduce hepatocytes and deliver the FAH enzyme that is deficient in these cells.Castle Creek plans to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for LV-FAH in HT1. Castle Creek also continues to progress several additional candidates targeting other rare liver and metabolic diseases and skin and connective tissue disorders.

In connection with the acquisition, Joseph Lillegard, MD, PhD, has joined Castle Creek as chief scientific officer. Dr. Lillegard is a board-certified pediatric and adult general, thoracic and fetal surgeon at the Children's Hospital of Minnesota, and led the cell and gene therapy research lab at Mayo Clinic that discovered LV-FAH. Robert A. Kaiser, PhD, DABT, has also joined the company as vice president of preclinical development. Dr. Kaiser is a board-certified toxicologist with over a decade of experience designing, conducting, and reporting preclinical and IND-enabling studies. Dr. Lillegard and Dr. Kaiser will be the company leads for Castle Creek's recently announced research collaboration with Mayo Clinic to advance discovery and development of investigational gene therapy candidates for the treatment of osteogenesis imperfecta and classical Ehlers-Danlos syndrome.

"It is an exciting time to join Castle Creek, a company that has already established an impressive research and development program in cell and gene therapies with proven clinical development and in-house manufacturing capabilities," said Dr. Lillegard. "I look forward to collaborating with the company's dedicated team on development of novel gene therapies. We believe our work to evaluate the safety of in vivo lentiviral vector administration in HT1 has the potential to be a precedent setting approach that can be applied to a range of new therapeutic areas for underserved patient populations."

About Castle Creek Biosciences, Inc.

Castle Creek Biosciences, Inc. is a late-clinical stage cell and gene therapy company focused on developing and preparing to commercialize disease-modifying and potentially curative therapies for patients living with rare genetic diseases. Castle Creek's most advanced product candidate, dabocemagene autoficel (FCX-007, D-Fi), an ex vivo, autologous gene therapy, is currently being evaluated in a Phase 3 clinical trial for the localized treatment of chronic wounds due to recessive dystrophic epidermolysis bullosa (RDEB). The company is also evaluating FCX-013, an ex vivo, autologous gene therapy, in a Phase 1/2 clinical trial for the treatment of moderate to severe localized scleroderma.In addition, LV-FAH, an in vivo, investigational gene therapy candidate, is being assessed in preclinical studies for the treatment of hereditary tyrosinemia type 1 (HT1).Castle Creek is pursuing discovery and development of early-stage novel product candidates utilizing its dual platform of ex vivo and in vivo technologies to expand its robust pipeline. The company operates an in-house, commercial-scale manufacturing facility in Exton, Pennsylvania. Castle Creek Biosciences, Inc. is a portfolio company of Paragon Biosciences, LLC. For more information, visit https://castlecreekbio.com/or follow Castle Creek on Twitter @CastleCreekBio.

About Paragon Biosciences, LLC

Paragon is a global life science leader that creates, builds and funds innovative biology-based companies in three key areas: cell and gene therapy, adaptive biology and advanced biotechnology. The company's current portfolio includes Castle Creek Biosciences, CiRC Biosciences, Emalex Biosciences, Evozyne, Harmony Biosciences, Qlarity Imaging, Skyline Biosciences, and a consistent flow of incubating companies created and supported by the Paragon Innovation Capital model. Paragon stands at the intersection of human need, life science, and company creation. For more information, please visit https://paragonbiosci.com/.

Media Contacts

Adam DaleyBerry & Company Public Relations212.253.8881[emailprotected]

Karen CaseyCastle Creek Biosciences302.750.4675[emailprotected]

SOURCE Castle Creek Biosciences, Inc.

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Castle Creek Biosciences Acquires Novavita Thera to Expand Innovative Cell and Gene Therapy Platform - PRNewswire

Risky biotechnology start-ups that soared in recent years can't catch a break in 2022. Beam Therapeutics (NASDAQ:BEAM) recently signed a major research deal with Pfizer (NYSE:PFE) that could be worth up to $1.35 billion, and hardly anyone seemed to notice.

This was clearly great news for Beam Therapeutics, but a stock market scorned for clinical-stage biotech stocks didn't respond the way anyone familiar with the company would have expected. Instead of surging higher in response to the Pfizer deal, Beam Therapeutics stock actually fell nearly 2% on the day of the announcement.

Nearly all biotech stocks are in the doghouse lately and it looks like the market may have missed something here. Let's look closer to see if Beam Therapeutics is a smart buy at the moment.

Image source: Getty Images.

Over the past few years, Pfizer has watched its peers experiment with CRISPR-based gene editing techniques without making any significant investments. Beam Therapeutics' base-editing technology, though, really got the big pharma company's attention.

Instead of removing and replacing entire sections of genetic material like Intellia, andCRISPR Therapeutics (NASDAQ:CRSP), Beam Therapeutics is pioneering a more precise method called base editing. This involves altering just one letter of genetic material at a time, which is a lot more useful than it might seem. Around half of all known genetic variations associated with diseases are caused by single-point mutations.

Pfizer will give Beam Therapeutics a $300 million payment up front to discover new drug candidates aimed at three undisclosed targets that won't compete with Beam's existing programs. Beam's eligible for up to $1.05 billion in milestone payments if all three go on to become a commercial success.

Beam Therapeutics is eligible to receive royalties at an undisclosed percentage of global sales for each future program. Beam Therapeutics even has an option to co-develop and co-commercialize one of the future candidates for a larger cut of sales.

Beam Therapeutics finished September with $934 million in cash after operations burned through $329 million during the first nine months of the year. Pfizer's cash injection should raise the company's cash balance high enough to get through 2023 before it needs to tap investors for more.

There's no telling whether Pfizer will decide to license a candidate from Beam Therapeutics. If Pfizer drags its feet, the gene-editing start-up has some preclinical-stage programs of its own that might have a chance to impress investors before it's time to raise capital again.

The most advanced candidate in Beam's pipeline at the moment, BEAM-101 is an experimental gene therapy for the treatment of sickle cell disease. The company doesn't expect to begin enrolling patients into the first clinical trial with BEAM-101 until the second half of 2022.

With a recent market cap of $4.5 billion, there's already a lot of success for Beam's pipeline priced into the stock. Unfortunately, the road ahead could be a lot more challenging than investors are anticipating. Last year, Vertex Pharmaceuticals (NASDAQ:VRTX) and collaboration partner CRISPR Therapeutics reported highly encouraging results from a clinical trial with CTX001 that started way back in 2018.

CTX001 is an experimental gene therapy for sickle cell disease that's similar to BEAM-101 in that it encourages the production of fetal hemoglobin. If early interim data that Beam Therapeutics posts a couple of years from now doesn't appear competitive with CTX001, the stock could take a tumble. While this is a top gene-editing stock to watch, it's still a little too risky to buy right now.

This article represents the opinion of the writer, who may disagree with the official recommendation position of a Motley Fool premium advisory service. Were motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.

See the rest here:
Is Beam Therapeutics a Good Stock to Buy Now? - The Motley Fool

Amicus Therapeutics is moving on from an early-phase Batten disease program after follow-up data showed the therapy didn't stop the fatal nervous system disease from progressing long-term.

The company disclosed the pipeline trim in a preliminary revenue guidance announcement ahead of its Wednesday presentation at the J.P. Morgan Healthcare Conference.

Amicus was advancing a gene therapy program for CLN6 Batten disease, a type of the rare genetic disorder that causes development regression and typically begins in childhood. Back in 2019, Amicus shared data on AAV-CLN6 that showed the gene therapy stabilized children's motor and language functions.

But the CLN6 program will now be discontinued after the company got a look at long-term extension data from a phase 1/2 trial.

RELATED:Amicus shares early look at Batten disease gene therapy

The company found the disease stabilization that had occurred during the earlier portion of the trial was not sustained at the two-year mark. Amicus plans to review the data with the CLN6 Batten disease community to support continued research efforts to find better treatments and cures which are so desperately and urgently needed, according to the guidance announcement.

During its last earnings report in November 2021, Amicus had said it would be ramping up manufacturing activities and regulatory discussions for the program.

Meanwhile, the company will advance its CLN3 Batten disease program, which is currently in a phase 1/2 trial. A readout from the trial and additional preclinical data are expected in 2022, Amicus said. Once the data are released, the company can begin work on a pivotal trial.

Continued here:
JPM 2022: Amicus axes gene therapy program for type of Batten disease, advances another - FierceBiotech

I said, I am an RNA scientist. I can do anything with RNA, Dr. Karik recalled telling Dr. Weissman. He asked her: Could you make an H.I.V. vaccine?

Oh yeah, oh yeah, I can do it, Dr. Karik said.

Up to that point, commercial vaccines had carried modified viruses or pieces of them into the body to train the immune system to attack invading microbes. An mRNA vaccine would instead carry instructions encoded in mRNA that would allow the bodys cells to pump out their own viral proteins. This approach, Dr. Weissman thought, would better mimic a real infection and prompt a more robust immune response than traditional vaccines did.

It was a fringe idea that few scientists thought would work. A molecule as fragile as mRNA seemed an unlikely vaccine candidate. Grant reviewers were not impressed, either. His lab had to run on seed money that the university gives new faculty members to get started.

By that time, it was easy to synthesize mRNA in the lab to encode any protein. Drs. Weissman and Karik inserted mRNA molecules into human cells growing in petri dishes and, as expected, the mRNA instructed the cells to make specific proteins. But when they injected mRNA into mice, the animals got sick.

Their fur got ruffled, they hunched up, they stopped eating, they stopped running, Dr. Weissman said. Nobody knew why.

For seven years, the pair studied the workings of mRNA. Countless experiments failed. They wandered down one blind alley after another. Their problem was that the immune system sees mRNA as a piece of an invading pathogen and attacks it, making the animals sick while destroying the mRNA.

Eventually, they solved the mystery. The researchers discovered that cells protect their own mRNA with a specific chemical modification. So the scientists tried making the same change to mRNA made in the lab before injecting it into cells. It worked: The mRNA was taken up by cells without provoking an immune response.

More:
How The mRNA Vaccines Were Made: Halting Progress and Happy Accidents - The New York Times

Leptin is a hormone that comes from fat cells. It helps control food intake by sending signals about hunger to the hypothalamus in the brain. This process regulates appetite.

Leptin regulates energy levels by maintaining a balance between hunger and appetite. The hormone triggers the body to respond by eating more when energy levels are low and eating less when energy levels are stable or high.

People who have high levels of body fat have high circulating levels of leptin.

Research shows that having elevated leptin levels can lead to leptin resistance, making weight loss difficult.

This article looks at what the leptin hormone is, what the leptin diet involves, and the advantages and disadvantages of following the leptin diet.

Scientists discovered leptin, a protein that functions as a hormone, in 1994.

Leptin is one of the main hormones responsible for maintaining body weight. Leptin helps people balance how much food they consume by regulating hunger levels. The hormone also controls how much energy a person uses throughout each day.

Leptin comes from fat cells within the body. It enters the blood supply and travels up to the brain. The hormone must cross the blood-brain barrier, a membrane that protects the brain from harmful toxins, to get to the hypothalamus. The hypothalamus is the area in the middle of the brain that controls hormone regulation, among other important functions.

At the hypothalamus, leptin can function by signaling that the body does not need any more food. This response causes the person to feel full. If leptin levels are low, or leptin does not reach the hypothalamus, a person will continue to feel hungry.

Leptin regulates body weight and is an important marker for energy storage. This means if the body has excess energy stored as fat, leptin signals the hypothalamus to reduce appetite and burn excess body fat for fuel. This response helps a person maintain a moderate body weight.

However, when a person has high amounts of body fat, they can develop a resistance to leptin, which leads to abnormally high leptin levels.

Having leptin levels that are too low is less common. Low leptin levels can occur in severe childhood obesity and delayed puberty.

When leptin levels are below average, the brain thinks no body fat is present. This reduced level can cause symptoms of uncontrollable hunger, resulting in excessive food intake. Leptin injections are a way of reducing this problem.

After scientists discovered the hormone in 1994, Byron J. Richards created a diet named after it: the leptin diet.

The goal of the leptin diet is to return leptin levels to normal and create balance within the body. The leptin diet has five main principles:

The leptin diet permits most types of food, but guidelines suggest avoiding chemical additives and processed sugars and sticking to fresh and organic produce.

The leptin diet encourages other lifestyle changes, such as getting plenty of sleep and participating in regular physical activity.

A 2021 study suggests that diets high in fat, carbohydrates, fructose, and sucrose and low in protein are drivers of leptin resistance. The researchers concluded that leptin resistance might be reversible by reducing calories.

However, this research has some limitations, such as small sample sizes, so further evidence is required to verify these claims.

The leptin diet includes limiting snacking and shortening your daily eating window. If a person reduces how much they snack, this could create a calorie deficit necessary for weight loss.

A leptin diet is a sensible approach to weight loss for some people, as the diet promotes eating healthily without harsh restrictions but encourages a routine.

However, at present, no studies are investigating the effects of the leptin diet on weight loss and leptin levels.

It is important to remember that all bodies are different, and a diet that meets the nutritional demands of one person will not always work for someone else.

For example, limiting the number of meals to three per day and cutting out snacking may be effective for a person with a low activity level. However, it is unlikely to meet the energy demands of a person who leads an active lifestyle, exercises intensely, or has a physically demanding job.

Many factors can impact energy needs, including age, pregnancy, breastfeeding, and certain medical conditions.

A person should consider consulting a healthcare professional like a registered dietitian if they are interested in improving health through dietary changes.

Increasing research suggests that obesity causes people to develop leptin resistance.

When someone carries an excessive amount of body fat, they will have too much leptin circulating in the blood. This excess results in that person becoming leptin-resistant. This resistance means their brain stops responding to the leptin signals traveling up to it. It also means their body continues to produce leptin, contributing to elevated leptin and leptin resistance.

Research suggests that weight loss and energy-restricted diets may help reverse leptin resistance.

Leptin is the hormone that controls appetite. Leptin informs the brain when a person has eaten enough, reducing appetite, and produces hunger signals when a person requires energy.

As with any weight loss plan, a person should approach the leptin diet with caution. The diet may be effective for some people, but it may not meet the nutritional demands of every person. Check with a doctor before starting any significant weight loss diet.

Link:
Leptin: Benefits and risks of the leptin diet - Medical News Today

CAR-T cell therapy has been a boon for treating blood cancers. Since the technology was first brought to the clinic, CAR-T has offered patients months or years of life after they had exhausted all other treatment options and would have died within weeks.

Its been incredible, said Marcela Maus, an immunologist and cell therapist at Mass General Cancer Center. Weve seen patients who had multiple lines of therapies and progressed after all of those, [then] get CAR-T and go into long-term remission.

But CAR-T does have hefty limitations, and scientists like Maus are researching ways to overcome some of its major shortcomings. These issues have prevented CAR-T from being used safely and effectively outside of leukemia and myeloma, and even patients who have responded spectacularly to CAR-T usually see their cancers return. The therapies are also still incredibly costly and carry risks, including a reaction known as a cytokine storm that can be life-threatening.

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Potential solutions to these problems are still in the early stages, but scientists are beginning to get a vision of what the future of CAR-T cell therapy might look like. It could involve synthetic biology to engineer a more advanced cell, or engineering other parts of the T cell to make it work better in the challenging environment around a tumor.

The field is growing tremendously, Maus said. Different people are working on different issues then, ideally, the data kind of decides whats going to be the next big thing.

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Heres a look at what experts see as some of the most promising approaches.

Current CAR-T cells use their CAR, or chimeric antigen receptor, to identify and kill cancer cells. These are synthetic proteins that bind to a specific target, like a protein on a cell surface membrane, and then activate the T cell to kill any cell carrying this target.

Armed with a CAR, T cells become pros at killing cancer cells that have their target, but theyll also kill normal cells that happen to carry the protein, too. Once a CAR-T cell is in the body, there isnt much a clinician can do to rein it in if it starts causing a lot of toxicity.

Once we let the CAR out, theyre like teenage kids, Maus said. You can maybe watch, but you cant really control them. So, theres some desire to be able to turn them on or off at will.

So, researchers are also trying to create CAR-T cells that they can manually activate or deactivate. As a group, these are known as controllable CARs, and most work by engineering an additional genetic circuit in the CAR-T cell. In theory, clinicians should be able to activate a switch on the genetic circuit that induces the CAR-T cell to activate their CAR and express it on the T cells surface membrane, thereby activating the receptor. Then, after a while, the T cell will disarm.

The goal is really getting our hands back on the steering wheel for a bit, Maus said.

There are several ways that synthetic biologists are doing this. In one example, researchers engineered a CAR with a protein switch that activates the receptor in the presence of blue light. In another example, researchers added a gene to CAR-T cells that force it to create its CAR and express it on the cell surface, thereby activating it, only in the presence of ultrasound radiation.

That way, it can be focused into a specific location, said Peter Yingxiao Wang, a synthetic biologist at University of California, San Diego, who works on controllable CARs. When the light or ultrasound is on the tumor locally, they can activate the CAR gene to trigger killing. Anywhere else, the CAR T-cells will be benign.

The idea is that the clinician can focus the light or ultrasound onto the tumor to get CAR-T cells to begin killing there. Once that signal is turned off, the CARs should disarm or slowly degrade and deactivate the CAR-T cells killing function. This way, even if the CAR does kill healthy tissue, the damage will theoretically be limited to the area around the tumor.

But this is an infant field right now, Wang added. A lot of these studies are just proof of concepts to show that theyre technically achievable. If you want to move to clinical trials, all of the components must be optimized.

Scientists also must show that theyre truly safe in humans, and that keeping the damage to a smaller surface area will be enough to outweigh the risks in treating tumors located near vital organs like the heart.

Other researchers are working on developing new CARs that can function like a biomolecular computer, able to make simple logical decisions to target cancer cells. Conventional CARs can cause dangerous toxicity because they only use one protein to identify cancer cells, and it may be impossible to discover the perfect target that exists only on cancer cells and not at all on healthy cells.

You can never uniquely define cancer or any other healthy tissue just by one marker, explained Wilson Wong, a synthetic biologist at Boston University. It just doesnt work. Its like trying to find a person and saying, he has black hair. Its like, oh, my God, youll never find him.

But it might be possible to distinguish cancer cells from healthy ones by looking at multiple proteins on a single cell. So, researchers like Wong have begun building more advanced CAR T-cells that use genetic circuits that only activate a CAR under more complex conditions, like the presence of several specific proteins that arent often seen in combination on healthy cells.

In this sense, the CAR is making a logical decision like basic Boolean computing, and synthetic biologists call this technique logic-gating.

Theres a lot of cool genetic circuits you can build, said Yvonne Chen, a synthetic biologist at UCLA. One can think of conditional systems to obliterate cancer cells. One can build OR-gates, AND-gates, and NOT-gates, and layer them on top of one another.

Although, Chen added, a drawback of logic-gating is that by increasing the complexity of the system, you might also be increasing the chance something goes wrong. Its important not to overcomplicate the design. Sophisticated circuits are exciting, but sometimes the solution itself causes problems. For example, for an AND-gate, you also make it easier for the tumor to escape. If the tumor loses either target A or B, it escapes from therapy, she said.

Another issue with conventional CAR-T therapy is that after a while, T cells can simply stop working. Solid tumors, like lung or pancreatic cancer, often have strategies to defend themselves from immune system attacks, including those from CAR-T cells. That makes it harder for CAR-T cells to treat solid tumors and can provide an opening for the tumor to return or progress.

So, researchers like Chen are working on armoring the CAR T-cell against the hostile signals in the microenvironment around a solid tumor. One of these signals is called TGF-beta, a protein which can help shut down T cell activity and help cancer cells avoid death and detection from the immune system. Chen was able to create a CAR cell that is not only resistant to TGF-beta, but can actually subvert the signal and become more deadly when it encounters TGF-beta.

Instead of being dysfunctional, they become activated, Chen said. That actually converts a tumor defense mechanism into a stimulatory signal for our T cells and tells them, youre in an environment where youre likely to encounter a tumor cell. Get ready.

Other scientists are working to keep CAR-T cells which can lose power over time functional for longer. Even with a good antigen, the T cells rapidly lose function, said Shivani Srivastava, an immunologist at the Fred Hutchinson Cancer Research Center who works on this problem. If you trigger a T cell or CAR over and over again, that causes the cell to become exhausted rather than turning into a memory cell or something else.

In one case, Stanford immunologist Crystal Mackall engineered a CAR-T cell that takes breaks before returning to work. She did this by creating a transient CAR that can be turned on or off. It can enhance [the T cells] function and limit how exhausted they are by giving them periodic rest, Srivastava said. Thats a really interesting strategy in principle.

But most of the tactics that scientists have tried so far in the realm of armored CAR-T cells havent worked in the long term, Srivastava said. You need a strategy that can help the CAR T-cells persist long enough to eradicate the cancer and prevent its return, which might be a lifelong project for the immune system.

Well have to find the right combination that will be durable, she said. Often we can find strategies that enhance function for only a short period of time.

Some future approaches might see T cells abandoned altogether. Scientists are slapping synthetic receptors on new or different cell types, such as natural killer cells. One company, called CoImmune, is putting CARs on a synthetic cell called a CIK cell, or cytokine-induced killer cell.

This is a novel cell type. They dont occur in nature, explained Charles Nicolette, the biotechs chief executive.

Theyre made by taking white blood cells and growing them while exposing them to certain immune molecules called cytokines. The advantage of creating new cell types is that biologists can combine certain useful traits from other immune cells, Nicolette said. For example, CIK cells could have the NK cells natural ability to distinguish normal cells from malignant ones and the CAR T-cells enhanced ability to kill.

One day, UCLAs Chen hopes to take this concept even further. To her, the ideal cancer-killing cell would not be derived from anything biological, but a completely artificial cell.

Instead of taking a cell from a patient, but rather build a completely defined, minimal cell that can do what we want and nothing else. It cannot evolve. Cannot mutate. Then, self-destruct when you dont want it there, she said. But, she added, creating synthetic cells like that would be unimaginably challenging, and it might not be possible to create a cell thats both persistent but also unchangeable.

Still, a scientist can dream.

Read more:
STAT's guide to the next generation of CAR-T therapies - STAT

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COVID-19 can affect the global economy in three main ways: by directly affecting production and demand, by creating supply chain and market disruption, and by its financial impact on firms and financial markets. Our analysts monitoring the situation across the globe explains that the market will generate remunerative prospects for producers post COVID-19 crisis. The report aims to provide an additional illustration of the latest scenario, economic slowdown, and COVID-19 impact on the overall industry.

Regional Analysis

All the regional segmentation has been studied based on recent and future trends, and thedomainis forecasted throughout the prediction period. The countries covered in the regional analysis of the Global Cancer Gene Therapy market report are U.S., Canada, and Mexico in North America, Germany, France, U.K., Russia, Italy, Spain, Turkey, Netherlands, Switzerland, Belgium, and Rest of Europe in Europe, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, China, Japan, India, South Korea, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), and Argentina, Brazil, and Rest of South America as part of South America.

Key Benefits of the report:

This study presents the analytical depiction of the global Cancer Gene Therapy industry along with the current trends and future estimations to determine the imminent investment pockets.

The report presents information related to key drivers, restraints, and opportunities along with detailed analysis of the global Cancer Gene Therapy market share.

The current market is quantitatively analyzed from2021to2031to highlight the global Cancer Gene Therapy growth scenario.

Porters five forces analysis illustrates the potency of buyers & suppliers in the market.

The report provides a detailed global Cancer Gene Therapy market analysis based on competitive intensity and how the competition will take shape in the coming years.

Customization Before Buying, Visit @https://www.insightslice.com/request-customization/285

Table of Contents

Chapter 1: Cancer Gene Therapy Market Overview

Chapter 2: Global Economic Impact on Industry

Chapter 3: Global Market Competition by Manufacturers

Chapter 4: Global Production, Revenue (Value) by Region

Chapter 5: Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6: Global Production, Revenue (Value), Price Trend by Type

Chapter 7: Global Market Analysis by Application

Chapter 8: Manufacturing Cost Analysis

Chapter 9: Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10: Marketing Strategy Analysis, Distributors/Traders

Chapter 11: Market Effect Factors Analysis

Chapter 12: Global Cancer Gene Therapy Market Forecast

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About Us:

We are a team of research analysts and management consultants who share a common vision to help individuals and organizations achieve their short-term and long-term strategic goals by expanding high-quality research services. insightSLICE was established to support mature companies, start-ups and non-profit organizations in various industries, including packaging, automotive, healthcare, chemicals and materials, industrial automation, consumer products, electronics and semiconductors, IT and telecommunications, and energy. Our internally experienced team of analysts has extensive experience in the research industry.

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Cancer Gene Therapy Market 2021 Industry Outlook, Comprehensive Insights, Growth and Forecast 2031 | Celgene, SIBIONO, Anchiano Therapeutic, Achieve...

Just like at last year'sJ.P. Morgan Healthcare Conference,M&A is so 2019.

In the third winter of COVID-19, the drug development world hasnt executed on the type of megamergers we saw in 2019, such as Bristol Myers Squibbs $74 billion Celgene deal,which dropped in the lead-up to the healthcare industry's largest gathering.

Of course, we don't know whats happening in private Zoom rooms at the moment, or who managed to make some company-defining contacts at the second virtual edition of the conference.

Galapagos, with $5.6 billion to spend and a thinned late-stage pipeline, made it known Thursday morning that there are, in fact, private Zoom meetings to hash out potential acquisitions. The biopharmas chief business officer wasnt shy to say Galapagos is speaking with a host of companies as the company works on a rebound for 2022.

RELATED:2022 forecast: Biopharma M&A lags in 2021. Will drugmakers still look for bolt-on deals or large transactions?

But what was made public is that biopharmas stuck to licensing and research partnerships this week at JPM, extending a trend that has been going on for months. Backloaded, billion-dollar biobucks pacts are increasingly standard.

This year, I think, is a reflection of last year, and its much more licensing, much more tie-up, said Stuart Henderson, global life sciences industry leader at Accenture, in an interview with Fierce Biotech.

There are a few key factors driving the focus on licensing and collaborations, according to Henderson.

Premiums on acquisitions have increased, thus limiting affordability; venture capital funding has skyrocketed three times in the past five years, reducing the need for pharmas money; biotechs are going all the way to market on their own with tighter labels and more defined populations, shrinking the need for pharmas help in selling drugs; and the economic value of Big Pharma buying a late-stage biotech has declined, he said.

The virtual conference started with multiple licensing and research handshakes revealed Monday morning. Bayer kicked off the week with a $1 billionbiobucks beton a gene therapy deal with Mammoth, using the CRISPR science out of the lab of Jennifer Doudna, Ph.D.

RELATED:JPM22, Day 3: In short order, Perrigo part of a new landscape; Legend, J&J prep for cilta-cel launch

Next up was Novartis,ponying up$163 million to opt in on a COVID-19 antiviral after its collaborator Molecular Partners showed a 78% risk reduction in early-stage patients. Then, Pfizerdoled out$300 million upfront for Beam Therapeutics base editing technique. The COVID-19 vaccine maker madeanother two dealsMonday morning, including a financials-free agreement with Acuitas Therapeutics for its lipid nanoparticle delivery system for use in mRNA vaccines and therapies. Add to that a $100 million biobucks pact with Codex DNA.

This was allbefore 7 a.m. ET on Monday. Pfizers pandemic rival, Moderna, made some waves with its owndevelopment dealat $45 million upfront for Carisma Therapeutics in vivo engineered chimeric antigen receptor monocyte therapeutics in cancer. BioNTech followed with a $750 million milestone paymentspactwith Crescendo Biologics; Bristol Myers Squibb offered awhopping $3 billionin biobucks for cell therapies from Century Therapeutics; and Acadia was stoked tosigna $60 million upfront tie-up with Stoke Therapeutics.

Thats at least nine collaboration and licensing deals in the first day. You get the pattern. It was medtech that brought the M&A: Medtronicoffered$925 million for cardiac mapper Affera, and Exact Sciences willspend $190 millionon testing lab PreventionGenetics.

Henderson said some of the biotechs attracting licensing interest are those with platform technologies and digital biology companies that are attempting to cut the billion-dollar costs of drug discovery and development.

RELATED:JPM22, Day 4: Vertex aims to be cystic fibrosis leader into the 2030s; Gritstone hopes for new cancer biomarker; Galapagos' rebound year

The licensing announcements have slowed since those first days. But could that meanarms will be linked in coming weeks, as final negotiations are hashed out and due diligence is conducted? We hope so.

We know pharma is looking. Plenty of companiesPfizer, Moderna, BioNTech, Novartis and many morehave cash burning a hole in their pockets and pipelines to fill.

Read more:
JPM 2022: Licensing and research tie-ups trump M&A at conference in 'reflection of last year' - FierceBiotech

The evolution of advanced therapy medicinal products, or ATMPs, is bringing real world results, write James Fry, partner, and Isabel Teare, senior legal adviser from leading law firm Mills & Reeve.

The number of cell and gene therapy products now on the market around the world is near the 100 mark, according to recent research by McKinsey & Company, with many more in development.

These ground-breaking therapeutics can have a profound impact on previously intractable conditions. Products like Novartiss gene therapy Zolgensma, for example, are costly but have the potential to act as a single-dose cure for young patients with spinal muscular atrophy.

London-based Orchard Therapeutics has had some important wins with its ex vivo autologous gene therapy approach, as it seeks to address multiple therapeutic areas with profound unmet need.

The UKs powerful research base and established life sciences sector means that it is well placed to be part of this story. The Cell and Gene Therapy Catapult reports that, in 2020, the UK was host to around 12 per cent of ongoing ATMP global clinical trials some 154 separate studies. A striking statistic.

As more products are rolled out to patients, the journey for others both in overcoming technical obstacles and gaining public acceptance improves. But in the global race to bring these sophisticated products through from concept to clinic, some specific challenges stand in the way.

One of these is the need for a different style of regulatory engagement. The traditional methods used by regulators in dealing with small molecule pharmaceuticals show major limitations when applied to ATMPs.

Regulators have many years of in-depth experience with small molecule medicines. This can mean that regulatory guidance focuses on this section of the market and tends to be prescriptive in nature.

In contrast, regulation of large molecule, cell and gene therapies should, say those close to the industry, be based more on a philosophy rather than prescriptive rules, and should allow a greater degree of flexibility.

To remain at the forefront of this exciting sector, rapid technological progress needs to be matched by a surefooted regulatory response. With many ATMP projects created in the hands of smaller spin-outs or growing mid-sized companies there is no spare cash or capacity for mistakes.

Developers and regulators need to work together to make sure that safe and effective therapies can reach the patients that so badly need them.

A collaborative approach

Developers of innovative therapies can gain real benefits from early and regular engagement with regulatory agencies. The aim here is to smooth the product development path and, at the same time, avoid wasted effort and expense.

With development costs high, collaboration is a must to drive down complexity and uncertainty. The pressures of COVID-19 pandemic may have helped in strengthening the collaborative approach between regulators and developers, and many hope to see these changes embedded in day-to-day practice.

Think holistically

Regulatory strategy needs to form part of the overall development picture. For example, the shelf-life of ATMPs, and also their starting materials, can be very short.

Building an understanding of the practical issues into the supply chain is essential. Issues around cross-border transfers with possible delays for customs checks can seriously undermine the business model for this generation of products.

Likewise, point of care manufacturing may be a necessary element of treatment something regulators may allow for if they appreciate its importance.

New ways to shape clinical trials

Designing clinical trials of ATMPs to make sure that they are not excessively cumbersome or open-ended requires early planning and engagement.

Regulators are working on tools that can help innovators in this area, such as novel trial structures like basket or umbrella trials.

These can be deployed to evaluate multiple hypotheses with the overall goal of improving the efficiency of trial evaluation.

As clinical development progresses it may become necessary to adapt the trial, and regulators need to recognise this. Long term follow-up may also be necessary.

Regulators learning from each other

With advanced therapies setting new challenges, developers can take heart from seeing regulators learning from each other and sharing lessons internationally.

Many products in this area address rare diseases, so a coordinated approach makes sense in serving a group of patients spread around the world.

Likewise, regulators can benefit from looking to novel examples elsewhere.The Japanese authorities offer a special conditional approval that looks at safety and predicted efficacy, before allowing products a provisional authorisation for the market. This enables treatment availability to patients for a number of years during which evidence to establish efficacy is gathered.

An unexpected benefit

The COVID-19 pandemic, while stretching healthcare provision to its utmost, has had some positive impacts. Widespread recognition of the importance of life science innovation is one.

But accelerating new approaches and ways of working with and between regulators is another less obvious benefit. We have looked at how this can strengthen and support cell and gene therapy development and are optimistic that these changes are here to stay.

And the UK governments recent Life Sciences Vision shows a real commitment to pushing through in many of these areas.

View post:
Cell and gene therapies: How to encourage and promote the move to market - Business Weekly

Researchers atUniversity Health Network (UHN) and the University of Toronto have received $24 million to advancetechnology to repair and rebuild organs outside the bodyfor patients in need.

The project, led byShaf Keshavjee, is one of only seven across Canada selected to receive funding in the Government of CanadaNew Frontiers in Research Fund(NFRF) Transformation competition, following an international consultation.

"The Ex Vivo Lung Perfusion (EVLP) system we developed here in Toronto has revolutionized lung transplantation in the past decade. Now, it's been translated around the world to increase lung transplant access and it's being extended to other organs," says Keshavjee, a professor and vice-chair for innovation in thedepartment of surgeryin U of Ts Temerty Faculty of Medicinewho is surgeon-in-chief at UHN and a senior scientist atToronto General Hospital Research Institute.

"With this transformative grant, we now have the opportunity to take ex vivo technology to the next level, where we can repair and rebuild organs for transplant."

Atul Humar, director of the AjmeraTransplant Centre(photo byTim Fraser)

Over 4,500 people in Canada are currently waiting for an organ transplant, and more than 270 die each year as the need for transplant greatly exceeds availability.

Ex vivo perfusion systems use specialized machines to maintain, evaluate and treat organs before transplant. They have a huge impact on increasing the number of organs that can be considered for transplant.

TheToronto Lung Transplant Program,led by Keshavjee, has used this technology to double the number of lung transplants performed and lives saved at UHN.

"The New Frontiers grant will allow us to advance applications for lungs and further develop ex vivo systems for other organs, such as liver, kidney, heart and pancreas," says Atul Humar, a co-principal investigator on the project, professor in thedepartment of medicineat U of T and director of theAjmera Transplant Centre at UHN.

Brad Wouters, UHN's executive vice president, science and research, notes that this major grant will enable multidisciplinary teams to develop new, cutting-edge approaches to extend the time that donated organs can be used, and also enable treatment and repair of unsuitable organs to allow treatment of more patients.

It will also help the teams refine and improve equitable organ allocation guidelines for all patients, he adds.

The advancements that this team has made and their continued success is made possible by support from provincial and federal governments, industry partners, external charitable agencies, generous philanthropy from the UHN Foundation and our incredible patient partners, says Wouters, who is also a professor in thedepartment of radiation oncologyat U of T. This award recognizes the tireless efforts of the team, and this support, which have been key to achieving global impact.

The New Frontiers Research Fund was designed to support large-scale, Canadian-led interdisciplinary research projects with the potential to realize real and lasting change.

The fund falls under the strategic direction of theCanada Research Coordinating Committeeand is administered by the Tri-Agency Institutional Programs Secretariat on behalf of Canada's three research granting agencies: theSocial Sciences and Humanities Research Council, theCanadian Institutes of Health Researchand theNatural Sciences and Engineering Research Council.

Over the course of this project, the team of over 20 researchers at U of T, UHN, national and international partner sites will develop sophisticated ex vivo platforms to:

Longer ex vivo preservation prior to transplant will enable many world-first therapeutic applications that will, ultimately, create more organs for clinical transplant.

One example is to use gene therapy to make an organ more like the recipient's cells and help to address the current hurdle of organ rejection by the immune system. Researchers at UHN are also working on changing an organ's blood type so the sickest people can get access to the next available organ, instead of waiting for one that exactly matches their blood a delay that currently can take several months before a match is found.

Another transformative goal is to use medicines and light therapies in the ex vivo circuit to eliminate viral or bacterial infections that previously prevented an organ to be considered for transplant.

"This grant gives us a unique opportunity to extend personalized medicine to every organ group," saysMarcelo Cypel, a professor in the department of surgery at U of T and surgical director of the Ajmera Transplant Centre, who is also a co-principal investigator on the project.

"Not only will it enable longer preservation, this research will let us treat and improve organs. It has the potential to change the paradigm in the field of transplantation."

The change will include several advanced applications, such as the engineering of new organs using stem cells with the goal to make organs available for all in need. Significant progress has already been made in generating new kidneys, lungs and tracheae (windpipe), and their applications will be tested further during the six-year project term.

With the involvement of a multidisciplinary team housed in a world-class centre at UHN, the project will bring personalized medicine to transplant, and go beyond solid organs.

Siba Haykal, plastic and reconstructive surgeon and project co-principal investigator, will lead research involving vascularized composite allotransplantation the transplant of limbs, face, trachea and composite tissues, such as skin and muscles.

"These are very delicate tissues that can't survive outside the body for very long and are very susceptible to rejection," she explains, adding that the current treatment involves high doses of life-long anti-rejection medication for transplant recipients.

Haykal and the team want to develop a system to preserve limbs and tissues out of the body without blood flow for longer periods. This will enable the application of new cell therapies to adapt these tissues to the recipient prior to surgery.

"Whether they have been disfigured by burns or from trauma or cancer, if they've had an amputation and need prosthetic limbs or if they require a new airway, transplantation provides hope for these patients who currently don't have many options," says Haykal, who is an assistant professor in the department of surgery at U of T.

"If we can use techniques that reduce the amount of anti-rejection medication and maybe one day get to a stage where they don't need it anymore, that would have a huge impact on the patient's quality of life."

Humar adds, "I have seen so many people who have literally been at death's door and have been completely turned around by transplant and live a full and healthy life. If we can offer that to more patients, then for me that would be an incredible achievement.

"This funding will also help us disseminate our knowledge, and facilitate other hospitals across Canada and around the world build upon what we're doing at UHN."

This story wasoriginally postedon the University Health Network website.

Continue reading here:
UHN and U of T receive $24-million federal grant for transplant research - News@UofT

DUBLIN--(BUSINESS WIRE)--The "Global Cell and Gene Therapy Manufacturing Services Market 2021-2027" report has been added to ResearchAndMarkets.com's offering.

The global cell and gene therapy manufacturing services market is projected to grow at a potential CAGR during the forecast period (2021-2027).

The key aspect that drives the growth of the market includes the rising incidence of cancer coupled with the increasing R&D by pharmaceutical companies and partnerships & agreements between pharmaceutical companies and contract development and manufacturing organizations (CDMOs). Benefits of partnering with a cell or gene therapy CDMOs include scalability, speed to market, access to technical expertise without overhead costs, and cost-efficiency.

Covid-19 has affected economies and industries in several countries due to lockdown, travel bans, and business shutdowns. The global healthcare industry is one of the major industries facing serious disruptions such as breaks in supply chains and disruptions in manufacturing due to lockdown and office shutdowns. However, an increased number of investigational studies based on cell therapies for the treatment of COVID-19 patients have led to a positive impact on the market.

The global cell and gene therapy manufacturing services market is segmented based on therapy, scale, indication, and end-user. Based on therapy, the market is segmented into cell and gene therapy. Among, therapy, the cell therapy segment is expected to grow at a decent rate during the forecast period.

The attributable factor for the growth of the segment is increasing awareness about cell therapy coupled with the development of genomics methods for cell analysis. Based on the scale, the market is segmented into pre-commercial/ R&D scale manufacturing, commercial-scale manufacturing. Among scale, pre-commercial/ R&D scale manufacturing holds a lucrative share in the market during the forecast period owing to the expansion of cell and gene pipelines across the globe.

North America held a major market position in the global cell and gene therapy manufacturing services market in 2020. The presence of key companies involved in the development of cell and gene therapy and growing investments in the field are some of the key factors driving the growth of the regional market. Europe is the second-largest market in 2020 owing to the presence of a strong workforce coupled with strong facilities.

Further, Thermo Fisher Inc., Merck KGaA, and F. Hoffmann-La Roche Ltd., among others are some of the prominent players operating in the market.

Strategic initiatives may support an increase in the market share of the players during the forecast period. The manufacturers are extensively investing in new technologies. Moreover, new launches & developments, partnerships and collaborations, and mergers and acquisitions are some of the growth strategies adopted by the players to sustain in the highly competitive market.

Key Topics Covered:

1. Report Summary

1.1. Research Methods and Tools

1.2. Market Breakdown

2. Market Overview and Insights

2.1. Scope of the Report

2.2. Analyst Insight & Current Market Trends

2.3. Porter's Analysis

3. Competitive Landscape

3.1. Key Company Analysis

3.1.1. Thermo Fisher Scientific Inc.

3.1.1.1. Overview

3.1.1.2. Financial Analysis

3.1.1.3. SWOT Analysis

3.1.2. Merck KGaA

3.1.3. Lonza Group Ltd.

3.2. Key Strategy Analysis

3.3. Impact of COVID-19 on Key Players

4. Market Determinants

4.1. Motivators

4.2. Restraints

4.3. Opportunities

5. Market Segmentation

5.1. Global Cell and Gene Therapy Manufacturing Services Market by Therapy

5.1.1. Cell Therapy

5.1.2. Gene Therapy

5.2. Global Cell and Gene Therapy Manufacturing Services Market by Scale

5.2.1. Pre-commercial/ R&D Scale Manufacturing

5.2.2. Commercial Scale Manufacturing

5.3. Global Cell and Gene Therapy Manufacturing Services Market by Indication

5.3.1. Cancer

5.3.2. CVD

5.3.3. Orthopedic

5.3.4. Infectious Diseases

5.3.5. Others

5.4. Global Cell and Gene Therapy Manufacturing Services Market by End User

5.4.1. Pharmaceutical and Biotechnology Companies

5.4.2. Academic and Research Institutes

6. Regional Analysis

6.1. North America

6.1.1. US

6.1.2. Canada

6.2. Europe

6.2.1. UK

6.2.2. Germany

6.2.3. Italy

6.2.4. Spain

6.2.5. France

6.2.6. Rest of Europe

6.3. Asia-Pacific

6.3.1. China

6.3.2. India

6.3.3. Japan

6.3.4. South Korea

6.3.5. Rest of Asia-Pacific

6.4. Rest of the World

7. Company Profiles

7.1. bluebird bio, Inc.

7.2. Boehringer Ingelheim International GmbH

7.3. Catalent, Inc.

7.4. Cell and Gene Therapy Catapult

7.5. Innovative Cellular Therapeutics Co., Ltd.

7.6. Charles River Laboratories International, Inc.

7.7. F. Hoffmann-La Roche Ltd.

7.8. FUJIFILM Holdings Corp.

7.9. Miltenyi Biotec B.V. & Co. KG

7.10. Nikon CeLL innovation Co., Ltd.

7.11. Novartis AG

7.12. Oxford Biomedica plc

7.13. Samsung Biologics

7.14. Takara Bio Inc.

7.15. WuXi AppTec Co., Ltd.

For more information about this report visit https://www.researchandmarkets.com/r/66e8ge.

Read more here:
Global Cell and Gene Therapy Manufacturing Services Market Research Report 2021-2027 Featuring Prominent Players - Thermo Fisher, Merck KGaA, and F....

JUPITER, Fla. A fun-loving baby with plenty of energy. That's how Linda Trantham describes her daughter Shandra.

"She loved to go to the beach even when she was a toddler and jump in the sea and go to Dubois Park," Linda said.

But one day that changed.

"I first noticed when we went to England on vacation and there was an indoor pool with a wave machine. And the next wave coming along and she like had a funny walk," she added.

A doctor diagnosed Shandra with Friedreich's Ataxia, known as FA, when she was 12 years old. It's an inherited rare disease that affects the nerves and muscles.

WPTV

Shandra herself noticed the change.

"I really couldn't walk in a straight line anymore and I had scoliosis, a sideways curvature of the spine. I had chest pain with exercise," Shandra explained. " So it's estimated that there are about 15-thousand people with it in the entire world. It's like four to five thousand in the U.S."

Shandra graduated from Jupiter High School and went on to the University of Florida. She's now 24 years old and in the Ph.D. program. She's on a path to help people dealing with neurological diseases.

WPTV

She said she's doing gene therapy research in a lab at UF.

"Working on gene therapy in general, kind of contributes to the project for FA, so it's really nice being in the center of all that and getting to see firsthand all of the things that are happening in the development," she said. "My hope is that science will come far enough along that we can just treat people on an individual basis, it would be really cool.

"I'm amazed and in some ways not surprised she's so inquisitive and into science," said Linda.

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Jupiter High School graduate helping in gene therapy research at UF - WPTV.com

The This research service discusses the cell and gene therapy (CGT) market and highlights some key roadblocks in viral vector manufacturing. While many CGT candidates exist in the pipeline, there is a huge capacity deficit that the industry is collaboratively trying to address.

New York, Dec. 21, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Technology Developments in Viral Vector Manufacturing for Cell and Gene Therapies" - https://www.reportlinker.com/p06192548/?utm_source=GNW

Scalability, costs, reproducibility, and overall process efficiency are some of the main pain points at each step of the viral vector manufacturing process.Many industry stakeholders are capitalizing on innovative, sustainable business models and capacity expansion investments to address shortage issues.

Biotechnology companies, such as Merck, Novartis, and Pfizer, and key contract development and manufacturing organizations, such as Thermo Fisher Scientific, Catalent, and FUJIFILM Diosynth Technologies, are investing in new capacities, expanding capacities, and developing innovative technologies to stay ahead in the CGT market. The research covers emerging technologies and trends, challenges, and opportunities across the manufacturing workflow, from upstream (viral vector production) to downstream (viral vector purification). Key developments in upstream processes for viral vector production include advanced transfection agents, novel plasmids, suspension-adapted cell culture, and stable producer cell lines. The research also discusses the general industry shift toward adopting automation, digitization, and advanced analytical processes, including on-line and in-line analytics and robust real-time analytics, to highlight the importance of analytical tools throughout the value chain. Smart technologies, such as automation and digital tools, and the adoption of artificial intelligence and big data support progress in process control and optimization while improving overall efficiencies and safety. The CGT industry works through orchestrated collaborations to develop reference standards and build process analytical technologies (PAT) to optimize manufacturing further. The research presents a birds eye view of key stakeholders and their innovative platforms and a snapshot of the collaborative ecosystem to understand the CGT industrys dynamic and fast-paced nature.Read the full report: https://www.reportlinker.com/p06192548/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Technology Developments in Viral Vector Manufacturing for Cell and Gene Therapies - Yahoo Finance

RESEARCH TRIANGLE PARK The nearly 30 million Americans who suffer from rare diseases have received some good news.

The National Institutes of Health, the U.S. Food and Drug Administration, and a cadre of pharmaceutical companies and non-profit organizations have teamed up to speed the development of new gene therapy treatments. Its good news for North Carolina as well, which is home to close to 50 gene therapy and rare disease-focused businesses that provide jobs for several thousand Tar Heel residents.

Whats called theBespoke Gene Therapy Consortium or BGTC was launched a little less than two months ago. Its part of the NIHs Accelerating Medicines Partnership (AMP), a public/private collaboration to speed drug development across different diseases. Ten global pharmaceutical companies and half as many non-profit patient organizations, as well as 11 NIH institutes, centers and initiatives have signed on.

The project is managed by the Foundation for the National Institutes of Health, whose mission is to promote biomedical discoveries that improve peoples lives.

Currently there are about 7,000 rare diseases in this country, 5,000 of which are due to genetic factors, according to BGTC. A single damaged gene causes nearly 80% of rare genetic illnesses, leaving millions of patients in the U.S. to suffer without much hope of improvement. Currently, only two of these diseases have FDA-approved gene therapy treatments.

BGTC said most rare inherited diseases stem from a specific gene mutation that is already known, which makes gene therapy a promising solution.

A customized or bespoke therapy could correct or replace defective genes with functional ones, according to NIH Director Francis S. Collins, M.D., Ph.D. There are now significant opportunities to improve the complex development process for gene therapies that would accelerate scientific progress and, most importantly, provide benefit to patients by increasing the number of effective gene therapies, he said.

But development is costly, complex and time consuming. And rare disorders by definition affect only a small number of patients. So most pharma companies arent willing to invest years of research and millions of dollars to bring a single-disease gene therapy to market, said Joni L. Rutter, Ph.D., acting director of NIHs National Center for Advancing Translational Sciences.

BGTC hopes to change that paradigm. The consortium wants to start with a common gene delivery vector known as the adeno-associated virus (AAV). Its considered one of the most effective gene delivery platforms for many human diseases.

The partnership said it will support a series of research projects and clinical trials to create new tools for AAV clinical development and regulatory evaluation. The BGTC aims to make it easier, faster and less expensive to pursue bespoke therapies in order to incentivize more companies to invest in this space and bring treatments to patients, Rutter pointed out.

The goal, over time, is to find ways to cut up-front gene therapy development costs, standardize the technology, and make it available for a broader range of diseases. The BCTC program will create a universal set of analytical tests to speed up gene and vector manufacturing. And the consortium will look for ways to streamline the regulatory framework.

By leveraging on experience with a platform technology and by standardizing processes, gene therapy product development can be accelerated to allow more timely access to promising new therapies for patients who need them the most, said Peter Marks, M.D., PhD., director of the FDAs Center for Biologics Evaluation and Research.

The consortium members will contribute about $76 million over the next five years to back its projects. BGTC said it will fund research for between four and six clinical trials each focused on a different rare, single-gene disease for which no gene therapies or commercial programs currently exist. Three disease areas are targeted: Lupus, Alzheimers and Type 2 diabetes.

People with terrible genetic disorders are in dire need of solutions, Collins said. The BGTC promises to transform the field of gene therapy so we can treat, or even cure rare diseases for which no current therapy exists.

North Carolina, with its substantial biotechnology and academic resources, has become a prominent incubator for leading-edge biotechnology startups. Gene therapy is a fast-developing area within our states biotech infrastructure, said Sara Imhof, Ph.D., senior director of precision health for the North Carolina Biotechnology Center. Our related ecosystem is well positioned to contribute to and benefit from the Bespoke Gene Therapy Consortium. Its an exciting time, both for the patients who desperately need the benefits gene therapy can provide and for our innovators and industry leaders who are dedicated to this important area of science.

Original post:
Rare disease consortium is good news for NC gene therapy companies - WRAL TechWire

Wilmot Cancer Institute researchers are a step closer to understanding the complex gene interactions that cause a cell to become malignant. In a new Cell Reports study published today, the group used network modeling to hone in on a set of such interactions that are critical to malignancy, and likely to be fertile ground for broad cancer therapies.

Discrete genetic mutations that can be targeted by drugs have only been identified for a small fraction of cancer types. But those mutations rely on a downstream network of non-mutated genes in order to cause cancer. Those downstream genes and their intricate interactions may be common across many cancers and could offer a giant leap forward in cancer therapy.

One of the lead authors of the study, Hartmut Hucky Land, Ph.D., who is the deputy director of the Wilmot Cancer Institute and the Robert and Dorothy Markin Professor of Biomedical Genetics at the University of Rochester Medical Center and has worked to identify common core features of cancers for over 10 years. His goal is to find cancers shared vulnerabilities and exploit them.

Targeting non-mutated proteins that are essential to making cells cancerous is a broader approach that could be used in multiple cancers, said Land, but its hard to find these non-mutated, essential genes.

That is why Land turned to Matthew McCall, Ph.D., MHS, a Wilmot Cancer Institute investigator who is an associate professor of Biostatistics and Computational Biology at URMC, for collaboration. McCall, who is the other lead author of the study, developed a new network modeling method, called TopNet, that the group paired with genetic experiments in cells and mice to pinpoint functionally relevant gene networks.

Lands group previously identified a very diverse set of non-mutated genes that are crucial to cancer. In this study, the group wanted to see how those genes interact starting with a subset of 20 genes. Increasing or decreasing the expression of one gene in cultured cells would have numerous effects on the expression levels of the other genes in the set.

There were so many interactions, you could waste a lot of time, energy and money testing interactions that might not be useful, McCall said. To hone in on the interactions that are more likely to be useful, we used network modeling, and compared our model networks back to the lab findings, McCall said.For context, the number of possible gene network models considered by TopNet was many times greater than the estimated number of atoms in the universe. After weeding out models that didnt closely fit the observed data and further focusing in on gene interactions that appeared in at least 80 percent of the models, the team was left with a manageable set of 24 high-confidence gene interactions. Subsequent experiments demonstrated that these interactions often play an important role in malignancy.

Dr. McCalls elegant and mind-boggling methodology is essentially helping us disentangle a hairball of genetic networks, said Land. These networks are usually very messy and its nearly impossible to extract useful information from them. But Dr. McCall has found a way to cut through this Gordian knot.

The group has already tested a sampling of the genetic interactions revealed by TopNet, and confirmed via experiments in cells and mice that the interactions are functionally linked. Next, the group intends to test the limits of TopNet, with the intent to use this method to find potential cancer therapies that are broadly effective.

This work was completed as part of a $6.3M National Cancer Institute Outstanding Investigator Award granted to Land in 2015 and a K99/R00 grant from the National Human Genome Research Institute to McCall. Helene McMurray, Ph.D., assistant professor of Biomedical Genetics and Pathology and Laboratory Medicine at URMC was the first author of the study.

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Honing in on Shared Network of Cancer Genes - URMC

DUBLIN, Dec. 21, 2021 /PRNewswire/ -- The "Global Advanced Therapy Medicinal Products CDMO Market Size, Share & Trends Analysis Report by Product (Gene Therapy, Cell Therapy, Tissue Engineered), Phase, Indication, Region, and Segment Forecasts, 2021-2028" report has been added to ResearchAndMarkets.com's offering.

The global advanced therapy medicinal products CDMO market size is expected to reach USD 12.9 billion by 2028, according to the report. It is expected to expand at a CAGR of 12.0% from 2021 to 2028.

The advanced therapy medicinal products are a group of biological products for human use that involve gene therapy products, cell therapy products, and tissue-engineered products. The growth of the market is credited to the increasing clinical trials of ATMP and the rising awareness and belief among researchers regarding the benefits of advanced therapy. The COVID-19 pandemic has significantly disrupted the cell and gene therapy industry due to the complexity in the manufacturing process.

The COVID-19 pandemic has adversely affected the overall medical industry, but the pandemic boosted the operations and development of advanced therapy due to the high requirement of the products such as mesenchymal stromal cells (MSCs) for the treatment of the virus. The regulations put forward by the FDA and government authorities have created a safe environment for the healthcare workers and allowed emergency approval for the supply of essential raw materials and faster development of the vaccines and other therapy products.

Technological advancement has been a major part of tissue engineering in the last few years. This method helps to replace or restore the injured tissues and organ function. Similarly, gene and cell therapy is attracting many patients for the treatment of rare diseases, the cases of which are augmenting globally.

Advanced Therapy Medicinal Product CDMO Market Report Highlights

Key Topics Covered:

Chapter 1 Methodology and Scope

Chapter 2 Executive Summary

Chapter 3 Advanced Therapy Medicinal Products CDMO Market: Variables, Trends, & Scope3.1 Market Segmentation and Scope3.2 Market Dynamics3.2.1 Market Driver Analysis3.2.1.1 Rising number of clinical trials for ATMP3.2.1.2 Increasing outsourcing activities3.2.1.3 Growing awareness of the treatment3.2.2 Market Restraint Analysis3.2.2.1 Stringent regulatory approvals3.2.2.2 High cost of outsourcing3.3 Penetration & Growth Prospect Mapping3.4 Advanced Therapy Medicinal Products CDMO: Market Analysis Tools3.4.1 Industry Analysis - Porter's3.4.1.1 Porter's Five Forces Analysis3.4.2 PESTEL Analysis

Chapter 4 Advanced Therapy Medicinal Products CDMO Market: Product Estimates4.1 Market Share Analysis, 2020 & 20284.2 Gene Therapy4.2.1 Gene therapy market, 2016 - 2028 (USD Billion)4.3 Cell Therapy4.3.1 Cell therapy market, 2016 - 2028 (USD Billion)4.4 Tissue Engineered4.4.1 Tissue engineered market, 2016 - 2028 (USD Billion)4.5 Others4.5.1 Market, 2016 - 2028 (USD Billion)

Chapter 5 Advanced Therapy Medicinal Products CDMO Market: Phase Estimates5.1 Market Share Analysis, 2020 & 20285.2 Phase I5.2.1 Phase I market, 2016 - 2028 (USD Billion)5.3 Phase II5.3.1 Phase II market, 2016 - 2028 (USD Billion)5.4 Phase III5.4.1 Phase III market, 2016 - 2028 (USD Billion)5.5 Phase IV5.5.1 Phase IV market, 2016 - 2028 (USD Billion)

Chapter 6 Advanced Therapy Medicinal Products CDMO Market: Indication Estimates6.1 Market Share Analysis, 2020 & 20286.2 Oncology6.2.1 Oncology market, 2016 - 2028 (USD Billion)6.3 Cardiology6.3.1 Cardiology market, 2016 - 2028 (USD Billion)6.4 Central Nervous System6.4.1 Central nervous system market, 2016 - 2028 (USD Billion)6.5 Musculoskeletal6.5.1 Musculoskeletal market, 2016 - 2028 (USD Billion)6.6 Infectious Disease6.6.1 Infectious disease market, 2016 - 2028 (USD Billion)6.7 Dermatology6.7.1 Dermatology market, 2016 - 2028 (USD Billion)6.8 Endocrine, Metabolic, Genetic6.8.1 Endocrine, metabolic, genetic market, 2016 - 2028 (USD Billion)6.9 Immunology & inflammation6.9.1 Immunology & inflammation market, 2016 - 2028 (USD Billion)6.10 Ophthalmology6.10.1 Ophthalmology market, 2016 - 2028 (USD Billion)6.11 Haematology6.11.1 Haematology market, 2016 - 2028 (USD Billion)6.12 Gastroenterology6.12.1 Gastroenterology market, 2016 - 2028 (USD Billion)6.13 Others6.13.1 Others market, 2016 - 2028 (USD Billion)

Chapter 7 Advanced Therapy Medicinal Products CDMO Market: Regional Analysis

Chapter 8 Company Profiles8.1 Strategic Framework8.2 Company Profiles8.2.1 Celonic8.2.1.1 Company Overview8.2.1.2 Financial performance8.2.1.3 Product Benchmarking8.2.1.5 Strategic Initiatives8.2.2 Bio Elpida8.2.2.1 Company Overview8.2.2.2 Financial performance8.2.2.3 Product Benchmarking8.2.2.6 Strategic Initiatives8.2.3 CGT Catapult8.2.3.1 Company Overview8.2.3.2 Financial performance8.2.3.3 Product Benchmarking8.2.3.6 Strategic Initiatives8.2.4 Rentschler Biopharma SE8.2.4.1 Company Overview8.2.4.2 Financial performance8.2.4.3 Product Benchmarking8.2.4.6 Strategic Initiatives8.2.5 AGC Biologics8.2.5.1 Company Overview8.2.5.2 Financial performance8.2.5.3 Product Benchmarking8.2.5.6 Strategic Initiatives8.2.6 Catalent8.2.6.1 Company Overview8.2.6.2 Financial performance8.2.6.3 Product Benchmarking8.2.6.6 Strategic Initiatives8.2.7 Lonza8.2.7.1 Company Overview8.2.7.2 Financial Performance8.2.7.3 Product Benchmarking8.2.7.5 Strategic Initiatives8.2.8 WuXi Advanced Therapies8.2.8.1 Company Overview8.2.8.2 Financial performance8.2.8.3 Product Benchmarking8.2.8.5 Strategic Initiatives8.2.9 BlueReg8.2.9.1 Company Overview8.2.9.2 Financial performance8.2.9.3 Product Benchmarking8.2.9.6 Strategic Initiatives8.2.10 Minaris Regenerative Medicine8.2.10.1 Company Overview8.2.10.2 Financial performance8.2.10.3 Product Benchmarking8.2.10.5 Strategic Initiatives8.2.11 Patheon8.2.11.1 Company Overview8.2.11.2 Financial performance8.2.11.3 Product Benchmarking8.2.11.5 Strategic Initiatives

For more information about this report visit https://www.researchandmarkets.com/r/rjc62f

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

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Outlook on the Advanced Therapy Medicinal Products CDMO Global Market to 2028 - Rising Number of Clinical Trials for ATMP is Driving Growth -...

SAN RAFAEL, Calif. and SHANGHAI, Dec. 16, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) and Skyline Therapeutics (formerly Geneception), a gene and cell therapy company focused on developing novel treatments for unmet medical needs, today announced a multi-year global strategic collaboration for the discovery, development and commercialization of Adeno-Associated Virus (AAV) gene therapies to treat genetic cardiovascular diseases.

The partnership will leverage Skyline Therapeutics' integrated AAV gene therapy platform based on its proprietary vector engineering and design technology and manufacturing capability to develop innovative gene therapies with a focus on genetic dilated cardiomyopathies (DCM), a group of progressively advancing, devastating diseases with no targeted treatment options.

Under the agreement, BioMarin and Skyline Therapeutics will collaborate on discovery and research through to an Investigational New Drug Application (IND). BioMarin brings experience in gene therapy development, cardiovascular biology and insights into genetic basis of diseases, and Skyline contributes its expertise in developing gene therapy products including vector engineering and design technology and manufacturing capabilities to this collaboration. Each company will advance the programs through clinical development in their pre-defined territories.

In support of its R&D efforts for the collaborative projects, Skyline Therapeutics will receive an undisclosed payment associated with signing, comprising an upfront payment and an equity investment from BioMarin, and is eligible to receive pre-specified payments for R&D, regulatory and commercial milestones.

BioMarin will have the rights to commercialize therapeutic products resulting from the collaboration in its territories, including the United States, Europe, and Latin America, and Skyline Therapeutics will be responsible for commercialization in the Asia-Pacific region. In addition, Skyline Therapeutics will be eligible to receive royalty payments on future sales from BioMarin in its territories.

"We are thrilled to announce what we anticipate will be a fruitful collaboration at the interface between Skyline's innovative approach to AAV vector engineering and design and our team's proven expertise in creating and developing gene therapies," said Kevin Eggan, Group Vice President, Head of Research and Early Development, from BioMarin.

"We are excited to partner with Skyline Therapeutics to tackle these genetic forms of dilated cardiomyopathy. This collaboration strengthens our leadership in cardiac gene therapy and extends our R&D collaboration to Asia, where a large number of patients suffer from these devastating diseases," said Brinda Balakrishnan, Group Vice President, Corporate and Business Development at BioMarin. "We look forward to fostering this collaboration and bringing transformative medicines to patients worldwide."

"Dilated cardiomyopathy is a serious cardiac disorder in which structural or functional abnormalities of the heart muscle can lead to complications such as arrhythmia and heart failure, resulting in substantial morbidity and mortality. Mutations in many genes are associated with the development of DCM, among other etiologies for the disease," said Jay Hou, Chief Scientific Officer at Skyline Therapeutics. "Together with BioMarin's team we have identified a number of critical genes associated with DCM. We are delighted to work closely with BioMarin and apply our AAV vector technology to interrogate these new targets and develop novel treatments for DCM patients."

"The collaboration with BioMarin leverages both companies' capabilities in the development of gene therapies. With the BioMarin team, we share the goal of working in concert to develop therapies for genetic cardiovascular disease that address high unmet medical needs," said Amber Cai, CEO of Skyline Therapeutics. "Together, we will utilize gene therapy to tackle cardiovascular diseases with a disease modifying trailblazing approach that could change the treatment paradigm in these conditions."

About Dilated Cardiomyopathy (DCM)

DCM is a common cause of heart failure and end-stage DCM, which often leads to heart transplantation. Despite improvements in pharmacotherapy and care, the five-year survival rate of DCM is only about 50%. Hundreds of thousands of patients suffer from the genetic forms of DCM in U.S., EU, China, and Japan. More than 50 genes associated with DCM have been identified, accounting for 40-50% of familial DCM cases. Many of these genes encode proteins with important known functions in cardiomyocytes related to cytoskeletal, sarcomere and nuclear envelope biology. Our aim is to correct the pathways altered by these genetic contributors to DCM through AAV based gene therapy, in each case addressing the root cause of the disease.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare genetic diseases. The company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit http://www.biomarin.com.Information on such website is not incorporated by reference into this press release.

About Skyline Therapeutics

Skyline Therapeutics is a fully integrated gene and cell therapy company dedicated to the discovery, development and delivery of innovative therapies. Established in 2019, Skyline Therapeutics has built a proprietary AAV-based gene therapy platform that integrates novel capsid engineering and vector design, analytical and process development, and state-of-the-art GMP manufacturing capabilities that support large scale clinical-grade vector production. The Skyline team of world-class experts and leaders in science, technology and business brings industry-leading know-how and is advancing a pipeline of diversified programs that address multiple diseases including ocular, neurological, metabolic and blood disorders. Skyline Therapeutics is also broadening its therapeutic expertise to cover more disease areas with high unmet need such as cardiovascular disorders through strategic partnerships. Headquartered in China, Skyline Therapeutics currently has research, development and manufacturing capabilities in Shanghai and Hangzhou. http://www.skytx.com

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: expectations related to themulti-year global strategic collaboration with Skyline for the discovery, development and commercialization of AAV gene therapies for dilated cardiomyopathy and pre-specified payments to Skyline for R&D, regulatory and commercial milestones, and the rights to commercialize therapeutic products resulting from the collaboration in its territories, including the United States, Europe, and Latin America. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. Additional important factors to be considered in connection with forward-looking statements are detailed from time to time under the caption "Risk Factors" and elsewhere in BioMarin's Securities and Exchange Commission (SEC) filings, including BioMarin's Quarterly Report on Form 10-Q for the quarter ended September 30, 2021, and future filings and reports by BioMarin. BioMarin undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

References

Contacts:

Investors

Media

Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 455-7451

Felisa Feng

Skyline Therapeutics

[emailprotected]

SOURCE BioMarin Pharmaceutical Inc.

http://www.biomarin.com

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BioMarin and Skyline Therapeutics Announce Strategic Collaboration to Develop Novel Gene Therapies for Cardiovascular Diseases - PRNewswire

Paris (AFP) Camille Abbey was two months away from giving birth to twins when she sensed something wasn't right.

"I felt strange all week. I had swelled enormously. I had a lot of trouble moving," said the 33-year-old French journalist.

Abbey's midwife found her blood pressure spiking and sent her to hospital where doctors confirmed she had preeclampsia, a potentially life-threatening complication.

The risks of preeclampsia have been known about for centuries, but there is still no cure or prevention and a lack of awareness remains a problem.

"Although one in every 12 pregnancies are affected by preeclampsia, many pregnant women have never heard of the disease," Patricia Maguire, director of the Institute for Discovery at University College Dublin, told AFP.

Doctors made a breakthrough 10 years ago that allowed them to develop the first diagnostic test, and Maguire is working on a second one, with trials under way in Ireland.

Early diagnosis is lifesaving because the condition sets in without symptoms.

Once they appear, the only way to stop complications like liver and kidney failure, or death, is to deliver the baby -- even if it is premature.

A 2021 study showed that hypertensive disorders of pregnancy have increased in the last nearly 30 years, though better screening and population growth may account for the rise.

The study in the BMC Pregnancy and Childbirth journal also said that most deaths occur in low-income settings where doctors say the toll is likely higher than reported.

An estimated 76,000 women and 500,000 babies die every year due to hypertensive pregnancy conditions like preeclampsia and eclampsia, a rare complication, according to a 2015 study in the Journal of Family Medicine and Primary Care.

Sarah Kilpatrick, an obstetrician-gynecologist specialising in preeclampsia at Cedars-Sinai hospital in Los Angeles, says educating women is crucial.

"The hard part for women is many times they feel fine," Kilpatrick told AFP, "so you can't even believe you have something like preeclampsia."

- Like 'lightning' -

Emmanuelle Honore survived eclampsia -- named after the Greek word for lightning -- which can develop if preeclampsia goes undetected and lead to potentially fatal seizures.

Her decision to walk herself to hospital after monitoring her own blood pressure saved her life.

"They didn't wait for the blood test results to come back to decide to do an emergency cesarean," said the Paris-based archaeologist, 37.

Immediately after giving birth, she began to convulse.

"It was a question of minutes. The baby and I were incredibly lucky," she told AFP. "During the seizure I felt myself leaving my own body."

Today, her son is healthy and turns three in February.

But the eclampsia has left Honore psychologically scarred and unable to give birth again.

"For me, there is a before and an after -- it's two different lives."

The world's first diagnostic test, developed by kidney specialists Ananth Karumanchi and Ravi Thadhani by linking the levels of two proteins to the disorder's onset, has been available in Europe for a decade.

It is in clinical trials in the United States, where preeclampsia disproportionately affects Black women due to a higher prevalence of risk factors like diabetes, obesity and stress, the American Heart Association says.

"(The trials) include the diversity of women in the United States and by the end of this year we will be able to determine whether the diagnostics have just as much benefit and specificity as in Europe," Thadhani told AFP.

The test developed by Maguire not only detects preeclampsia with a blood sample, it uses artificial intelligence to provide a timeframe for safe delivery, allowing as much time as possible for the foetus to develop.

An article published in May said the test had the potential to be streamlined so that kits could be sent to hospitals with easy-to-collect samples analysed using online information.

"The big dream is to reach every person who needs this test across the world," Maguire said.

Even after labour, preeclampsia sufferers are not necessarily out of danger.

Kilpatrick said about one in 10 women whose preeclampsia was their first time with high blood pressure need to stay on anti-hypertensive medication after giving birth.

When her twins were born in October 2020, Abbey thought her ordeal was over but her blood pressure failed to go back to normal.

She was later diagnosed with the dangerous preeclampsia-related condition HELLP (Hemolysis, Elevated Liver enzymes and Low Platelets) syndrome, which affects some 15 percent of women with severe preeclampsia.

While her premature babies received state-of-the-art care, Abbey said she stayed in a post-op recovery room for days where doctors could do little more than monitor her vital signs and try to control her blood pressure.

Vassilis Tsatsaris, an obstetrician-gynecologist at Paris' Port Royal hospital, said research on finding treatments for preeclampsia and other complications has been impeded by the risks for the unborn baby.

"We live in an era where therapeutic innovations for cancer, vaccines or gene therapy are advancing very, very quickly," he told AFP.

"Unfortunately when it comes to pregnancy, things move much more slowly."

2021 AFP

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Preeclampsia: an ongoing battle to save lives - FRANCE 24