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Regenxbio (RGNX) is a pioneer in gene therapies with a wide set of licensing agreements and an internal pipeline. The company focuses on adeno-associated virus (AAV) gene therapies for gene replacement and antibody delivery pursuing markets in retinal, neurodegenerative, and liver diseases. With a market cap of ~$1.3B (enterprise value of ~$1B), ~$400M of cash on the balance sheet, and ~$35M in revenue, Regenxbio is well-positioned to complete its milestones around manufacturing and clinical development into 2020.

The core investment thesis for Regenxbio is described below.

Validated technology platform to develop successful AAV gene therapies:

Strong financial position:

Undervalued internal assets:

From its 52-week high, Regenxbio's stock is down over 40%. The stock reached a low point from COVID-19 development. This is likely due to a lack of near term catalysts for the stock. With additional data for their lead asset in wet AMD coming in the first half of 2020, the initiation of phase II trials for the asset, and the sales ramp up for Zolgensma, the stock has a few potential catalysts coming up. As a result, there is an attractive entry point for investors to become an owner in Regenxbio.

Figure 1: RGNX daily chart (Source: Capital IQ)

The opportunity is that Regenexbio's licensing agreement with AveXis (NVS) on a medicine called Zolgensma to cure spinal muscular atrophy can potentially alone earn Regenxbio $3B-$4B in revenue. Zolgensma is an AAV gene therapy that delivers a transgene of SMN1 to cure the disease. About 20K people in the US have the disorder. With the medicine being priced at a little over $2M per patient, the market potential is well over $40B.

In 2014, Regenxbio licensed their AAV technology to AveXis to cure spinal muscular atrophy. The deal included various milestone payments to Regenxbio and importantly a mid-single to low double-digit royalties on net sales.

This one deal alone beyond the 20 similar deals Regenxbio has and its internal pipeline makes the company an attractive business. A simple DCF analysis with various assumptions, with the most important being including a capital expenditure of $400M for the clinical work with their flagship internal product, supports that Regenxbio is undervalued:

Figure 2: DCF model for RGNX (Source: Internal)

All the modeling done doesn't really help anyone figure out why this opportunity exists? Why is Regenxbio undervalued? Doubts around the Zolgensma scale up? Worries that capital from licensing deals will be wasted on an internal drug pipeline?

It's unprecedented that a drug company's platform is worth a lot more than the internal pipeline of drugs. From Regenxbio's latest corporate presentation, the company's main internal program, RGX-314, is focused on wet AMD:

Figure 3: Overview of Regenxbio's main asset (Source: Corporate Presentation)

RGX-314 is an AAV therapy for wet AMD. The slide describes the problem (leaky blood vessels in the eye) and how large it is (~2M patients) along with the vector (AAV8) and delivery cargo (anti-VEGF Fab). However, the company doesn't mention Eylea or other wet AMD medicines that are already approved. Regenxbio alludes to issues around delivery of drugs like Eylea (REGN), but doesn't go too deep on this slide or the presentation in general about how competitive their RGX-314 program will really be amongst clinicians.

An important point for any gene therapy is delivery whether it's a transgene or a CRISPR protein. A major reason why Regenxbio focused on wet AMD and ophthalmology in general is that delivering something to the eye is a lot easier than delivering something to the brain. RGX-314 is undergoing a phase I/II trial focused on establishing safety; the pivotal trial will come later. For the phase I portion, the company met their primary endpoint and showed safety so far. They have also shown how increasing doses of their gene therapy reduces the number of injections. This is going to be an important experiment and data set to argue for clinicians to switch over from something like Eylea.

The real value in Regenxbio is in its AveXis deal and the various licensing partnerships:

Figure 4: Regenxbio licensing partnerships (Source: Corporate Presentation)

Figure 5: Regenxbio licensing partnerships (Source: Corporate Presentation)

Figure 6: Regenxbio licensing partnerships (Source: Corporate Presentation)

This business model is enabled by Regenxbio's core technology focused on AAV7-10 and natural or close-to-natural variants:

Figure 7: Overview of Regenxbio's platform (Source: Corporate Presentation)

Over the next two years, the key milestones are:

The business can continue to strike up more licensing deals and expand current ones. The margin of safety here is that Regenxbio is undervalued just for its deal with AveXis and its various licensing deals that provide periodic payments based on progress and potentially more royalties if the drugs are approved and commercialized.

This seems to be a case where the market is focusing on the company's internal pipeline. Regenxbio's headline drug is interesting but unlikely to be competitive. Whereas, the business has a wonderful platform and licensing business that is being ignored. Simple valuations show these cash flows are not being fully appreciated. As a result, Regenxbio is going to grow revenue without any additional work and still has the potential to strike again through the 20 or so deals it has.

Figure 8: Key upcoming milestones for Regenxbio (Source: Corporate Presentation)

Regenxbio's lead candidate is focused on wet age related macular degeneration (AMD). The disease is a severe form of macular degeneration, a condition in which layers of macula get progressively thinner. The wet form is caused by abnormal blood vessels grown under the macula and retina where leaky blood vessels cause problems with vision, ultimately leading to blindness. The current standard-of-care is an anti-vascular endothelial growth factor (anti-VEGF) therapy. Patients require monthly injection of anti-VEGF to stop the growth of leaky blood vessels. Wet AMD is not a genetic disease, but it is a large and established market for Regenxbio to capture.

To frame the market opportunity of wet AMD, a few facts are helpful:

196 million people worldwide & 288 million by 2040 have AMD

10% have wet AMD, but is the leading cause of blindness

175,000 new patients annually in US

Growing number of patients due to aging population

Current treatments are regular injections of anti-VEGF

Figure 9: Overview of wet AMD (Source: JMS)

For wet AMD, competition comes from Genentech, Regeneron (REGN), and Adverum (ADVM). Genentech sells Lucentis at a price of $1850 per dose. Regeneron sells Eylea at $1150 per dose. Genentech's Avastin is also used off lab and is becoming more popular due to its cheap price of $60 per dose; the medicine is currently used for metastatic colorectal cancer. For gene therapies in wet AMD, the sole competitor is Adverum Biotechnologies. With 20M people with wet AMD, the total market opportunity for these medicines are in the billions of dollars.

Where Genentech's and Regeneron's medicines require multiple doses over the lifetime of a patient, a gene therapy has the potential to be curative and remove the multiple dosing requirement. For wet AMD, over 50% recurrence rate in the first year after treatment has stopped, and over 25% recurrence rate in the second year after treatment has stopped For drugs like Eylea and Lucentis, monthly intravitreal injection creates large burden for patients and create difficulty in dosing for clinicians. These problems allow Regenxbio to potentially capture the market with a gene therapy:

Figure 10: Wet AMD market (Source: Reportlinker)

Regenxbio's lead asset, RGX-314 is pursuing wet AMD. So far the company has shown:

Dose dependent protein expression levels and drug efficacy

Sustained protein expression for over 1.5 years

Long term efficacy demonstrated for Cohort 3 for rescue-free patients

No serious adverse events (SAE), but mild adverse events (AE) such as inflammation

Significant improvement in visual acuity for rescue free patients

Figure 11: Trial design of Regenxbio's lead asset (Source: Corporate Presentation)

RGNX

Cohort 1

Cohort 2

Cohort 3

Cohort 4

Cohort 5

Dose

3 x 109 gc/eye

1 x 1010 gc/eye

6 x 1010vg/eye

1.6 x 1011 gc/eye

2.5 x 1011 gc/eye

Rescue Injection Free

Not Available

Mean 4.7 rescue inj.

Not Available

Mean 3.8 rescue inj.

3 / 6 Patients

Mean 1.3 rescue inj.

5 / 12 Patients

Mean 2.2 rescue inj.

9 / 12 Patients

Mean 0.8 rescue inj.

Duration

52 Weeks

52 Weeks

78 Weeks

52 weeks (2H 2020)

52 weeks (2H 2020)

Best Corrected Visual Acuity (BCVA)

In ETDRS letters

Mean: -2.0

Range: -8/+10

Mean: +7

Range: -4/+15

Mean: +8

Range: 0/+21

Mean: +2

Mean: +4

Central Subfield Thickness (m)

Mean: -14

Range -81/+92

Mean: +26

Range -7/+62

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Regenxbio Is A Leader In Gene Therapies - A Case Where The Platform Is Worth More Than The Pipeline - Seeking Alpha

The UK company MicrofluidX has closed a 1.6M (1.4 M) seed funding round to develop a microfluidic platform that could produce cell and gene therapies more cheaply than conventional cell cultures.

The funding round was led by UKI2S, a national seed investment fund targeting early-stage companies, as well as Longwall Ventures and Cambridge Angels.

MicrofluidX will use the funding to establish a prototype of its technology. With this prototype, the company then aims to compare the performance of its microfluidics approach to current cell culture techniques used to produce gene and cell therapies.

In the dynamic cell therapy space, one of the major bottlenecks facing the field is the manufacture and scaling process, as manual cell culturing techniques are often required. Applying microfluidic technology to the manufacturing process may be an answer to this issue.

Although it is less developed at the manufacturing level, microfluidics technology has long been part of research in cell biology. It has several advantages over conventional cell cultures. For example, it allows cell cultures to be controlled more precisely on chips, increases automation, and can reduce the consumption of expensive ingredients in the cell culture process by a factor of twenty.

According to MicrofluidX, its platform could scale up microfluidics far beyond just biology research. The aim is to run dozens of cell cultures in parallel, with the capacity to produce cells more cheaply and with a higher yield than with current manufacturing techniques.

The result is that we can leverage all the inherent advantages of microfluidic cell culture at a scale never seen before, MicrofluidXs founder and CEO, Antoine Espinet, told me. This leads to much lower bioprocessing costs, better control over the final product, and faster translation from research to commercialization.

In particular, the company is investigating its technologys capacity to produce immune T-cells a common type of cell used in immunotherapies such as CAR T-cell therapies and other cell types.

Whilst the regulatory agencies are now warming up to cell and gene therapies, there are still growing pains, especially around manufacturing, Pablo Lubroth, an investor with UKI2S, told me.

It is essential to not only support companies that produce the therapies themselves, but also companies that are developing enabling technologies to ensure these therapies can be effectively commercialized and therefore have a tangible benefit to the patient.

As well as manufacturing, microfluidics is gaining traction in diagnostics and screening. For example, another UK startup, Lightcast Discovery, was founded last year to screen cells using microfluidics and beams of light. Additionally, the Belgian nanofluidics company miDiagnostics last month raised 14M to commercialize its silicon chip diagnostics in collaboration with Johns Hopkins University in the US.

Image from Shutterstock

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UK Startup to Manufacture Cell and Gene Therapies with... - Labiotech.eu

Sangamo Therapeutics(SGMO) announced the official closing of the global deal withBiogen(BIIB) for gene regulation therapies in neurology, explains biotechnology sector expertJohn McCamant, editor ofThe Medical Technology Stock Letter.

More from John McCamant: Ziopharm: Catalysts in T-Cell Therapy

With roughly $650 million in cash post-deal, SGMO is trading just above its cash positive despite having the broadest pipeline in the gene therapy/editing/regulation field.

Of all things in the press release, management mentioned the cash would be used to progress the pipeline, plus the potential filing of a BLA for SB525 for hemophilia A.

Impressive Terms For Early Stage

As a reminder, Biogen paid Sangamo $350 million upfront, including a $125 million license fee and an equity investment in Sangamo stock of $225 million (@$9.21 per share).

Sangamo is also eligible to receive up to $2.37 billion in potential milestones, including $925 million on pre-approval milestones and $1.335 billion on first product sales (that does not include the royalties that are also part of the deal).

In the blockbuster markets being developed such as Alzheimers and Parkinsons diseases, SGMO will earn high-single-to-low double digit royalties on potential sales. This is a pre-IND stage deal remarkable financial terms for this early stage of development.

Hem A Update On Schedule

Pfizer(PFE) cancelled its R&D Day that was set for March due to the virus, but the Company will hold a quarterly earnings call on April 28. There is some chance that Pfizer updates investors with some of the highlights it was preparing for the R&D Day, including the SB-525 program with Sangamo.

Either way, SGMO will still be updating investors when the two longest treated patients in the ALTA study hit their 12-18 month follow-up for the duration of Factor VIII production, sometime this summer.

See also: Activision Blizzard: "Stay-at-Home" Entertainment

The 12-18 month durability data is key for SGMO given that BMRNs first 12-18 month durability data was the turning point for Wall Streets belief in their gene therapy. Sangamo is a buy under $20 with a target price of $30.

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Sangamo- Early-Stage Progress in Gene Therapy - Yahoo Finance

-New, 100 million facility will more than double existing production capacity

-Supports large-scale commercial manufacturing of viral and gene therapy products

DARMSTADT, Germany, April 21, 2020 /PRNewswire/ -- Merck, a leading science and technology company, today announced a second Carlsbad, California, U.S.A.-based facility for its BioReliance viral and gene therapy service offering. The new, 100 millioncommercial facility is expected to open next year.

Building on Merck's success in helping customers commercialize their gene therapies made possible by viral vectors, the company's expansion will help innovators produce at a scale that ensures these therapies reach more patients in need

"Viral vector manufacturing has transitioned from a niche industry to the cornerstone of the future of biopharmaceuticals," said Udit Batra, member of the Merck Executive Board and CEO, Life Science. "Few companies have the scale and quality systems in place for manufacturing commercial viral vector products. Building on our success in helping customers commercialize their gene therapies made possible by viral vectors, our expansion will help innovators produce at a scale that ensures these therapies reach more patients in need."

Merck's Life Science business sector facility in Carlsbad manufactures gene therapies for its customers globally. Gene therapy involves the delivery of a genetic payload into patient cells to produce a therapeutic effect such as correction of a mutated gene or retargeting of an immune cell to fight cancer. Diseases such as hemophiliaand cancer are being investigated using this technique where a single dose may cure the disease. Viral vectors are often called the most complex therapeutic manufactured today.The gene therapy market, which accounted for $1 billion in 2018, is expected to reach $10 billion by 2026, according to a recent Biotech Forecasts global market analysis and industry forecast.

Merck's new, 140,000-square-foot manufacturing facility will support viral and gene therapy production at the 1000-liter scale using its Mobius single-use equipment. The site is part of the Life Science business' expanding product and service offering to the viral and gene therapy marketplace. Merck has close to three decades of experience in cell and gene therapy, and its Carlsbad, California, U.S.A site has been involved in the gene therapy area since 1997, near the time that clinical trials for gene therapy began. In the interim, the company manufactured viral vectors for two cell and gene therapy products.

This expansion underscores Merck's continued investment in viral and gene therapies from clinical to commercial scale and marks the second major investment at its Carlsbad facility in recent years. In 2016, the investments resulted in nearly doubling its former production capacity. The upgraded facility grew from 44,000 square feet to 65,000 square feet. Today, the Carlsbad site is home to 16 modular viral bulk manufacturing cleanroom suites with single-use equipment and two fill/finish suites for gene therapy, viral vaccine and immunotherapy products. With the expansion, the company will add 11 suites, bringing the total to 27, used in various steps of manufacturing.

In addition to contract development and manufacturing services for viral vectors, Merck also provides seamless manufacturing and testing services at its pharma and biopharma testing sites globally.

Merck recognizes that cell and gene therapy has resulted in major advancements in medicine. The company supports these therapies under consideration of ethical and legal standards; it has established an independent, external Bioethics Advisory Panel. This panel provides guidance on various topics, including gene editing and stem cells usage, in which its businesses are involved. The company has also defined a clear operational position taking into account scientific and societal issues.

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All Merck news releases are distributed by e-mail at the same time they become available on the Merck Website. Please go to http://www.merckgroup.com/subscribe to register online, change your selection or discontinue this service.

About MerckMerck, a leading science and technology company, operates across healthcare, life science and performance materials. Around 57,000 employees work to make a positive difference to millions of people's lives every day by creating more joyful and sustainable ways to live. From advancing gene editing technologies and discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices the company is everywhere. In 2019, Merck generated sales of 16.2 billion in 66 countries.

Scientific exploration and responsible entrepreneurship have been key to Merck's technological and scientific advances. This is how Merck has thrived since its founding in 1668. The founding family remains the majority owner of the publicly listed company. Merck holds the global rights to the Merck name and brand. The only exceptions are the United States and Canada, where the business sectors of Merck operate as EMD Serono in healthcare, MilliporeSigma in life science and EMD Performance Materials.

Photo - https://mma.prnewswire.com/media/1156781/Merck_Gene_Therapy.jpg

SOURCE Merck

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Merck Boosts Commercial Viral Vector and Gene Therapy Manufacturing Capacity - Yahoo Finance

Add a new, 100 million California facility to the growing gene therapy infrastructure.

Merck KGaA announced they are opening a second gene therapy and viral vector factory in Carlsbad, California. The new center will be just one point in the global supply chain Big Pharma is rapidly erecting to keep manufacturing capacity for the new technology at pace with clinical development. Over the past year, Novartis, PTC Therapeutics, Pfizer and Vertex each announced or opened new facilities in Switzerland, North Carolina and New Jersey that will help build gene therapy.

In November,Reutersreported that 11 drugmakers had set aside $2 billion for the manufacturing effort. They were led by Novartis, the giant behind the second FDA-approved gene therapy in Zolgensma which planned to spend $500 million, and Pfizer, which has yet to get a gene therapy approved but will spend $600 million. Catalent and Thermo Fisher, meanwhile, each spent over a billion dollars acquiring companies involved in gene therapy or viral vector manufacturing.

Merck KGaA last shored up its gene therapy manufacturing in 2016 the year before the first gene therapy was approved in the US when they expanded their original Carlsbad facility from 44,000 to 65,000 feet. At 140,000 feet, the new facility will be more than double the size of its older neighbor. Gene therapies and the viral vectors used to deliver them will be cooked up in 1,000 liter bioreactors metal vats roughly the size of 260 gallons of milk. Merck has not said when it is scheduled for completion.

Although Merck KGaA lists no gene therapies in its most recent pipeline, the German drugmaker uses its facilities to help biotechs focused on the emerging modality to manufacture their products. The list of approved gene therapies can still be counted on one hand, but hundreds are now in clinical trials.

Manufacturing emerged as a pressing bottleneck almost as soon as Spark Therapeutics got Luxturna, a treatment for a form of inherited blindness, approved as the US first gene therapy. In an op-ed in STAT last year, Sparks head of technical operations, Diane Blumenthal, described the process of building in Philadelphia one of the first in-house gene therapy manufacturing facilities. With regulators increasingly open to approving the treatments quickly, she encouraged other companies to invest in manufacturing in advance of even knowing if the therapy works, particularly because gene therapy requires customization in ways few other modalities do.

And she warned the problem will only get more pressing.

There isnt a gene therapy manufacturing playbook yet to guide the development of gene therapies, Blumenthal wrote. Manufacturing a gene therapy is only half the battle. The other half is making enough of it, doing that as efficiently as possible, and getting it to the patients who need it. These challenges become even more urgent to tackle as the industry shifts to the next chapter in gene therapy development, from treatments made in small batches for small patient populations to bigger volumes for larger rare-disease populations and commercial scale.

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Merck KGaA adds to gene therapy manufacturing boom with 100M facility in California - Endpoints News

Every week there are numerous scientific studies published. Heres a look at some of the more interesting ones.

New Protein IDed that May Cause Alzheimers

Scientists at the University of Tokyo tested 19,151 individual genes looking for their effect on amyloid beta levels. Amyloid beta is one of the proteins that accumulates in the brains of Alzheimers patients and is generally viewed as one of the primary drivers of the disease. They identified a new protein using CRISPR/Cas9 gene editing, called calcium and integrin-binding protein 1 (CIB1). They found that cells without functional CIB1 genes generate abnormally high levels of amyloid beta protein. The research was published in FASEB Journal.

We believe this is the first time anyone has used this CRISPR/Cas9 genetic screening technique to look for changes in amyloid beta production, said Yukiko Hori, co-first author and lecturer at the University of Tokyo.

In normal, healthy cells, CIB1 is not directly involved with processing amyloid beta, but it stays attached to another protein, gamma secretase inside cells and at the cell membrane. In cells that dont have CIB1, gamma secretase stays inside the cell longer and doesnt leave the membrane. Amyloid beta undergoes multiple steps before reaching its final form. Normally, gamma secretase processes amyloid beta precursors to help produce the final amyloid beta protein. This happens inside the cell, then gamma secretase moves to the cells outer surface membrane.

Patients diagnosed with early-stage Alzheimers disease have lower levels of CIB1 in their brains, while people with late-stage Alzheimers have higher-than-healthy levels of CIB1.

We cannot say for certain why CIB1 is increased in late-stage Alzheimers disease, said Taisuke Tomita, who runs the research lab where the study was conducted. What is important is that in both the early and late stages of Alzheimers disease, something is abnormal about the regulation of CIB1.

Possible Approach to Improving Gene Therapy

Investigators at the University of Groningen have developed a technique that may improve gene therapies. They use DNA/lipid complexes (lipoplexes). Because the viruses used traditionally in gene therapies can cause an immune response and the cells endosomes tend to degrade DNA or other particles, the lipoplex provides protection. They can fuse with the endosome membrane, which prevents degradation.

Gene Promoters that Can Be Used to Treat Neurological Diseases

Researchers at Princeton Neuroscience Institute have developed new gene promoters that act like switches to turn on gene expression. They can be used in gene therapy, with a particular interest in neurological diseases such as Parkinsons and Alzheimers. Viruses are used to carry genes into cells during gene therapy, typically adeno-associated viruses. The Princeton team used promoters found in herpes viruses, which take up less space than existing promoters and allow the transport of larger genes or multiple genes. They are also long-lasting.

Biosensor to Detect SARS-CoV-2 in the Air

Researchers at Switzerland-based Empa, ETH Zurich and Zurich University Hospital have developed a sensor that has the potential to identify SARS-CoV-2, the novel coronavirus that causes COVID-19, in the air. The work is led by Jing Wang at Empa, who usually works on measuring and analyzing airborne pollutants. The sensor has reliably shown it can identify the first SARS-CoV virus that was responsible for the SARS pandemic in 2003. It has numerous similarities to SARS-CoV-2. Tests showed that the sensor can clearly distinguish between the very similar RNA sequences of the two viruses, Jing Wang said. And the results appear in minutes.

Possible Gene Therapy for Glaucoma

Glaucoma is a common condition of the eye involved fluid buildup in the front part of the eye. It affects more than 64 million people globally and is the leading cause of irreversible blindness. Current treatments include eye drops, laser or surgery. Researchers at the University of Bristol demonstrated that a single injection of a gene therapy using CRISPR and a gene called Aquaporin 1 targeting the ciliary body, where fluid is produced within the eye, led to reduced eye pressure.

More Evidence Parkinsons is an Autoimmune Disease

A study co-led by investigators at the La Jolla Institute for Allergy and Immunology (LJI) adds to the theory that Parkinsons disease is at least partly an autoimmune disease. The research was published in Nature Communications. Science has known for some time that the clumps of a damaged protein known as alpha-synuclein build up in the dopamine-producing brain cells of Parkinsons disease patients. The clumps lead to death of the cells and cause motor symptoms and cognitive decline.

Once these cells are gone, theyre gone, said Cecilia Lindestam Arlehamn, first author of the study and LJI research assistant professor. So if you are able to diagnose the disease as early as possible, it could make a huge difference.

A 2017 study showed that alpha-synuclein attracted certain type of T-cells, causing them to mistakenly attack brain cells, which potentially contributed to the progression of Parkinsons. The new findings found that the T-cells that react to alpha-synuclein are the most abundant when patients are first diagnosed with the disease. They tend to disappear later in the disease and by 10 years after diagnosis, few patients still have them.

This tells us that detection of T-cell responses could help in the diagnosis of people at risk or in early stages of disease development, when many of the symptoms have not been detected yet, said LJI professor Alessandro Sette, who co-led the research with David Sulzer of the Columbia University Medical Center. Importantly, we could dream of a scenario where early interference with T-cell responses could prevent the disease from manifesting itself or progressing.

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Research Roundup: New Protein Linked to Alzheimer's Identified and More - BioSpace

Asklepios BioPharmaceutical, Inc. (AskBio), a prominent U.S.-based clinical-stage AAV gene therapy firm, has recently announced that it has acquired BrainVectis, a Paris-based gene therapy firm renowned for its work in neurodegenerative disorders, especially on therapies that regenerate brain cholesterol metabolism for patients suffering from Huntington's disease along with other disorders.

For the record, BrainVectis was initiated by Nathalie Cartier-Lacave, M.D., and has support and funding from Sorbonne University, the French Alternative Energies, and INSERM. Currently, Dr. Cartier-Lacave of Atomic Energy Commission (CEA) and her team are concentrating on developing gene therapies that increase the CYP46A1 enzymes expression in the brain.

Evidently, this enzyme is associated with numerous disorders where brain cholesterol metabolism dysfunction is concerned in the neurodegenerations pathology.

Sources cite that AskBio would leverage BrainVectis synthetic and capsid promotor design platforms and its capacity to advance the development of this platform and manufacturing solutions along with other BrainVectis projects.

Speaking on the move, Sheila Mikhail, CEO and Co-Founder, AskBio, said that the companys acquisition of BrainVectis provides it with a remarkable opportunity to work jointly while transforming patients lives.

Huntington's disease is one of the diseases that is irreversible and devastating and presently has no effective treatments. However, BrainVectis in-depth world-leading knowledge of neurodegenerative diseases including Huntingtons disease could allow AskBio to expand its portfolio for these diseases as well.

Prior to this news, AskBio had made headlines when it revealed that it has appointed Don Haut, Ph.D., as its new Chief Business Officer to handle business development execution and strategy.

Reportedly, Dr. Haut has over two decades of healthcare experience and was previously serving to Histogenics Inc. and Sherlock Biosciences as their Chief Business Officer.

Commenting on which, Mikhail said that Don would be a key addition to the company as he holds a wealth of business and strategy development experience within the medical technology and biotechnology fields. His expertise would help the company to effectively advance its existing platforms.

Source Credit: https://www.contractpharma.com/contents/view_breaking-news/2020-04-22/askbio-acquires-brainvectis/

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AskBio acquires BrainVectis to expand its gene therapy portfolio - News.MarketSizeForecasters.com

RANCHO CORDOVA, Calif., April 24, 2020 /PRNewswire/ --ThermoGenesis Holdings, Inc.Holdings, Inc. (Nasdaq: THMO), a market leader in automated cell processing tools and services in the cell and gene therapy field, announced today that it was featured on multiple new outlets, including a FOX40 news segment titled, "Rancho Cordova Company Says It's Working on Potential Treatment for Coronavirus," regarding the ThermoGenesis and ImmuneCyte joint venture and its research collaborator's efforts toward developing a systemic antibody approaches against COVID-19, ranging from convalescent plasma therapy and polyclonal antibody, to high-affinity monoclonal antibody drug candidates.

Using the rapid IgM/IgG antibody detection technology currently marketed by the Company, along with its own proprietary multi-component automated cell separating system, ThermoGenesis is working towards developing strategies to identify and isolate polyclonal antibodies from the blood of individuals who have recovered from the disease using convalescent whole blood or plasma, which could then be used as a potential therapeutic treatment for patients with of COVID-19.

As previously announced, the Company's joint venture partner, ImmuneCyte, has acquired from its collaborators worldwide intellectual property of four high-affinity monoclonal antibody drug candidates against COVID-19virus. These high-affinity neutralizing monoclonal antibodies were also identified from convalescent whole blood from individuals who have recently recovered from COVID-19.

The FOX40 News is accessible via the link below:

The KCRA3 News is accessible via the link below:

The in-depth local Sacramento Bee news article is accessible via the link below:

Commenting on the recent new coverage Chris Xu, PhD, an immunologist and Chief Executive Officer of ThermoGenesis, said, "We are taking a systematic approach to address several critical unmet needs in the fight against COVID-19, namely rapid tests and effective therapies. By working closely with our global collaborators, we had a head start on this critical research." Dr. Xu also noted that the U.S. Food and Drug Administration is fast tracking therapeutic products related to SARS-CoV-2, which the Company hopes may ultimately include ThermoGenesis' various antibody therapeutic approaches.

About ThermoGenesis Holdings, Inc.

ThermoGenesis Holdings, Inc. develops, commercializes and markets a range of automated technologies for CAR-T and other cell-based therapies. The Company currently markets a full suite of solutions for automated clinical biobanking, point-of-care applications, and automation for immuno-oncology, including its semi-automated, functionally-closed CAR-TXpressplatform, which streamlines the manufacturing process for the emerging CAR-T immunotherapy market. For more information about ThermoGenesis, please visit:www.thermogenesis.com.

About ImmuneCyte Life Sciences, Inc.

ImmuneCyte, a joint venture which is 19% owned by ThermoGenesis, develops and commercializes diagnostic and therapeutic technologies for COVID-19. The Company also provides clients the opportunity to bank their own immune cells when the cells are "healthy and unaffected" as a future resource for cellular immunotherapies, such as CAR-T. ImmuneCyte utilizes a proprietary CAR-TXpress platform, a GMP compliant close-system capable of automated separating and cryopreserving different components from blood, simultaneously. For more information about ImmuneCyte Life Sciences Inc., please visit: http://www.immunecyte.com.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements are not statements of historical facts, but rather reflect the current expectations of ThermoGenesis concerning future events and results. Forward-looking statements based on ThermoGenesis' current assumptions, expectations and beliefs are generally identifiable by use of words "may," "might," "will," "should," "expects," "plans," "anticipates," "believes," "estimates," "predicts," "potential" or "continue," or similar expressions and involve significant risks and uncertainties that could cause actual results, developments and business decisions to differ materially from those contemplated by these statements. Such forward-looking statements, including statements regarding the intended use of net proceeds from the registered direct offering as well as those factors concerning our expectations, involve risks, uncertainties and other factors, some of which are beyond our control, which may cause our actual results, performance or achievements, or industry results, to be materially different from any future results, performance, or achievements expressed or implied by such forward-looking statements. These risks, uncertainties and factors include, but are not limited to, market and other conditions, as well as those risk factors discussed in Item 1A of our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission ("SEC") as well as other documents that may be filed by ThermoGenesis from time to time with the SEC, which are available atwww.sec.gov. We undertake no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. You are cautioned not to unduly rely on such forward-looking statements when evaluating the information presented in this press release.

Company Contact:Wendy Samford916-858-5191ir@thermogenesis.com

Investor Contact:Paula Schwartz,Rx Communications917-322-2216pschwartz@rxir.com

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Ever since their son Michael was diagnosed with a rare genetic disorder, Terry and Georgia Pirovolakis have been racing to find a cure. They were getting close, and then everything came grinding to a halt.

"We had a whole bunch of researchers around the world doing different things. All of our research has basically stopped," says Terry Pirovolakis.

Michael is two-and-a-half years old and has spastic paraplegia type 50 (SPG-50), a rare neurodegenerative disease caused by a missing gene that is progressively robbing him of his ability to walk.

Over the past year, his parents have raised more than $1 million to help finance a clinical trial for custom gene therapy that promises to halt the disease. But the COVID-19 pandemic has paralyzed research around the world, and their hope along with it.

"It's hard to accept the fact that we were making so many gains," says Georgia Pirovolakis. "You know, we were hoping he would be walking by September."

Michael is just one of the many casualties of the global health crisis. From new gene therapies to help children like Michael, to the latest in cancer treatments, thousands of clinical trials deemed non-essential are on hold.

Brad Wouters is executive vice-president of Science and Research at Toronto's University Health Network, Canada's largest academic hospital. He says an example of the impact is a patient currently being treated for pancreatic cancer.

"He was part of a medical research study here that looked at the underlying genetics of that tumour, and that information revealed a potential new therapy that could be very effective for that patient in immunotherapy. But we only have access to that drug through a clinical trial. And so he won't get it."

Wouters says the only new research happening at the moment is around COVID-19. About 200 of UHN's researchers have pivoted to that, but thousands more risk losing their jobs, Wouters says, because in the past month alone UHN has lost $6 million in industry funding. It's why UHN is asking the federal government for help.

"We've been doing everything we can to try and support these jobs and support this research sector," says Wouters. "But it's over a month now, and we're really at a tipping point where we're going to see significant job losses if something can't be done."

Canada's $3-billion medical research industry relies on that brain trust, much of it made up of international students, says Martha Crago, vice-principal of Research and Innovation at McGill University.

"We wouldn't want to lose this wonderful set of brains coming into our country and helping bring solutions to the public domain," Crago says. "We need to do what we can to keep them."

The pandemic will be over at some point, Crago says, and those researchers will be needed to help regain lost scientific momentum.

Meanwhile, the Pirovolakis family fear they're running out of time.

All the therapies Michael has been doing to maintain the movement he has now, like physical and occupational therapy, have been cancelled. They're trying to improvise at home to keep him moving, and to keep their hope alive too.

The stalled research efforts means a cure for Michael may be delayed for up to a year. That could mean the difference between him ever walking or not.

"It means he's going to degrade," says Terry Pirovolakis. "The progression of the disease is going to kick in, he'll slowly become more and more paralyzed."

It's why the couple are trying to keep research into Michael's gene therapy going.

They're considering using money the family has raised so far to finance early toxicology tests on a potential treatment researchers in the U.S have already developed. They are also lobbying Canada's National Research Council to help develop a treatment or help with funding.

Georgia Pirovolakis says there are so many unknowns, and she can't bear to dwell on them.

"I just look at one day at a time. I'm not thinking about, you know, tomorrow. I'm not thinking about one month from now. I'm not thinking about the research stopping, him potentially not getting cured."

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'We're really at a tipping point': COVID-19 brings research into other medical conditions to a grinding halt - CBC.ca

Global Gene Therapy for Inherited Genetic Disorders Market: Overview

Rapid advances in mammalian DNA sequencing technologies over the past several years have enabled the identification of the aberrant genes responsible for a vast spectrum of genetic disorders. Gene therapy as a novel approach inarguably holds profound potential in finding universal therapeutic alternatives to treating inherited genetic disorders. Gene therapy for inherited genetic disorders entails introducing a functional copy of the defective gene to make up for the missing function, and can be accomplished using in vivo or ex vivo gene transfer.

Gene therapy for inherited genetic disorders has generated groundswell of interest in the research fraternity in finding cure for or in treatment of Mendelian genetic error causing rare diseases. Particularly, gene therapy in recent years has held promising potential in the treatment of a range of recessive gene disorders most notably sickle cell anemia, hemophilia, muscular dystrophy, cystic fibrosis, and other monogenic disorders. The axes of developments in the gene therapy for inherited genetic disorders market have been in the U.S., Europe, China, and Australia.

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Global Gene Therapy for Inherited Genetic Disorders Market: Notable Developments

Growing body of clinical studies done on mice models have unrivalled troves of preclinical data, which bodes well for the effectiveness of gene therapy for inherited genetic disorders. New approaches in the gene therapy for inherited genetic disorders market are being adopted to bring progress in this direction. In this regard, Salmeterol, a medicine approved for asthma, has shone a new light. The vasodilator to be used along with gene therapy has shown potential in increasing the effectiveness of the therapy for Glycogen storage disease type II (Pompe disease).

A team of investigator led by the researcher at Duke University Medical School discussed the preclinical data recently at 2019 annual meeting of the American Society of Gene & Cell Therapy. The preclinical data showed that the Asthma medicine reduces the accumulation of toxic glycogen accumulated in lysosome. The researchers concluded that it holds potential as an adjunctive therapy, and building on that may pave way for novel approaches on gene therapy for inherited genetic disorders.

Efforts to translate the findings of clinical research on gene therapy for inherited disorders to make the therapy a part of standard treatment has caught momentum in recent times. In this regard, vectors containing non-viral vectors have attracted the attention of scientists. A team of researchers at Fred Hutchinson Cancer Research Center in 2019 found that gold nanoparticles enable them to deliver gene-editing tools to blood stem cells in lab models. This might, they opined, pave way for more practicaland accessiblegene therapies for inherited disorders, notably for treating life-threatening blood disorders. Gene therapies were mediated by CRISPR. In the coming years they hope to collaborate with companies with commercial interest to develop the therapy for patient populations.

Some of the bigplayerseyeing promising stakes in the gene therapy for inherited genetic disorders market areSpark Therapeutics Inc., Orchard Therapeutics, Novartis AG, bluebird bio Inc., and BioMarin Pharmaceutical.

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Global Gene Therapy for Inherited Genetic Disorders Market: Key Drivers

Since 2000, scores of clinical trials involving patients with inherited genetic disorders have raised hopes of the medical fraternity of the potential of gene therapies. Thus far, more than 5000 clinical trials on gene therapy have been conducted, especially for hard-to-treat diseases. Diseases such as inherited blindness and leukemia have seen the efficacy and safety of gene therapies. Advances in bioengineering are expected to invigorate pre-clinical pipelines. In the not-so-distant future, success of more protocols will catalyze the prospects of the gene therapy for inherited genetic disorders market.

Further, advances have been made in viral and non-viral vectors with the purpose of making gene transfer more efficient, thereby boosting the gene therapy for inherited genetic disorders market. Particularly, new approaches emerged with the aim of making vectors more powerful.

Global Gene Therapy for Inherited Genetic Disorders Market: Regional Assessment

On the regional front, Asia Pacific bears considerable potential in the gene therapy for inherited disorders market. Of note, numerous strategic alliances have shifted their focus on the region, particularly China. The North America market has also been rising at a promising pace, driven by several gene-therapy tools and related drugs in the final stages of clinical trials. Favorable reimbursement models has also encouraged research into the gene therapy for inherited disorders.

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Gene Therapy for Inherited Genetic Disorders Market Key Companies and Analysis Top Trends by 2028 - Cole of Duty