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SIOX - Market Data & News

Today, Sio Gene Therapies Inc Incs (NASDAQ: SIOX) stock fell $0.01, accounting for a 0.49% decrease. Sio Gene Therapies opened at $2.03 before trading between $2.05 and $1.99 throughout Wednesdays session. The activity saw Sio Gene Therapiess market cap fall to $140,508,037 on 448,833 shares -below their 30-day average of 466,453.

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Its current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. The company is also expanding the reach of gene therapy to highly prevalent conditions such as Parkinson's disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies.

Visit Sio Gene Therapies Inc's profile for more information.

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Sio Gene Therapies (SIOX) falls 0.49% in Light Trading on August 4 - Equities.com

Recombinant proteins require strict quality control, as quality issues affect the reliability of the intended downstream application. Cell and gene therapy research in particular demands a higher quality of protein that adheres to strict quality control and has comprehensive validation.

In this exclusive SelectScience webinar, Dr. Deborah Moore-Lai, Director of Protein Development at Abcam, will introduce a new range of premium bioactive proteins. During the session, Moore-Lai will be detailing Abcams manufacturing workflow, as well as outlining the companys mission to support scientists in achieving their goals in cell and gene therapy research and other research areas.

Key learning objectives

Who should attend

Scientists interested in learning more about how Abcam can support their research through the development and supply of high-quality, bioactive proteins.

Certificate of attendance

All webinar participants can request a certificate of attendance, including a learning outcomes summary for continuing education purposes.

Speaker name: Dr. Deborah Moore-Lai, Director of Protein Development, Abcam

Professional bio: Deborah Moore-Lai joined Abcam in 2019 to lead the new Proteins Initiative, which included building out a new laboratory space and recruiting a new team of scientists with skills in protein expression, purification, and assay development. Before joining Abcam, Deborah spent 16 years working in industry in both the reagent and therapeutic spaces. For many years, she led the Antibody Production team at Cell Signaling Technology. From there Deborah joined Merck Research Laboratories, where she led the team responsible for antigen and antibody generation within Biologics Discovery.

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Developer deep dive: Designing proteins and reagents for cell and gene therapy research on SelectScience - SelectScience

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Regulatory feedback obtained on OTL-200 (MLD) and OTL-203 (MPS-IH) programs

New HAE collaboration with Pharming Group highlights broad potential for HSC gene therapy

Multiple presentations from neurometabolic programs at MPS Symposia including additional follow-up in MPS-IH

Cash and Investments of Approximately $270M Provide Runway into First Half 2023

BOSTONandLONDON, Aug. 04, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today reported financial results for the quarter ended June 30, 2021, as well as recent business updates and upcoming milestones.

This past quarter Orchard has shown great progress against multiple core strategic objectives across the portfolio, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. Obtaining regulatory clarity from the FDA on our investigational OTL-200 program in early-onset MLD represents a tremendous step toward making a treatment option available for young patients in the U.S. A second neurodegenerative program in MPS-IH is also advancing toward a pivotal trial, incorporating recent feedback from both the U.S. and EU regulatory agencies. In our earlier stage pipeline, were very excited for our new collaboration with Pharming exploring the potential of HSC gene therapy in hereditary angioedema.

Summary of Recent Publication and Business Updates

Data presentations at MPS 2021

Presentations from investigational hematopoietic stem cell (HSC) gene therapy programs in mucopolysaccharidosis type I Hurler syndrome (MPS-IH) and mucopolysaccharidosis type IIIB (MPS-IIIB) were featured at the 16th International Symposium on MPS and Related Diseases on July 23-25, 2021.

Collaboration with Pharming Group for hereditary angioedema (HAE)

On July 1, 2021 Orchard Therapeutics and Pharming Group N.V. announced a strategic collaboration to research, develop, manufacture and commercialize OTL-105, a newly disclosed investigational ex vivo autologous HSC gene therapy for the treatment of HAE. OTL-105 is designed to increase C1 esterase inhibitor (C1-INH) in HAE patient serum to prevent hereditary angioedema attacks. In preclinical studies, to date, OTL-105 demonstrated high levels of SERPING1 gene expression via lentiviral-mediated transduction in multiple cell lines and primary human CD34+ HSCs. A link to the full announcement can be found here.

Clinical and Regulatory Updates

In June 2021, Orchard announced several portfolio updates following recent regulatory interactions for the companys investigational programs in metachromatic leukodystrophy (MLD), MPS-IH and Wiskott-Aldrich syndrome (WAS). A link to the full announcement can be found here.

Research Programs

Orchard plans to announce new preclinical data from research programs in frontotemporal dementia with progranulin mutations (GRN-FTD) and Crohns disease with mutations in the nucleotide-binding oligomerization domain-containing protein 2 (NOD2-CD) in the second half of 2021.

Second Quarter 2021 Financial Results

Research and development expenses were $21.8 million for the second quarter of 2021, compared to $31.6 million in the same period in 2020. The decline is primarily due to non-cash impairment charges of $5.7 million taken in the second quarter of 2020 and other savings associated with our corporate restructuring. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and development milestone payments under the companys agreements with third parties, and personnel costs to support these activities.

Selling, general and administrative expenses were $14.3 million for the second quarter of 2021, compared to $15.7 million in the same period in 2020. The decrease was primarily due to savings associated with personnel and related changes.

Net loss was $36.6 million for the second quarter of 2021, compared to $47.5 million in the same period in 2020. The decline in net loss as compared to the prior year was primarily due to savings realized in our operating expenses as a result of the companys May 2020 updated strategy and corporate restructuring. The company had approximately 124 million ordinary shares outstanding as of June 30, 2021.

Cash, cash equivalents and investments as of June 30, 2021, were $269.3 million compared to $191.9 million as of December 31, 2020 and excludes the $17.5 million in upfront payments from the collaboration with Pharming Group N.V. entered into on July 1, 2021. The increase was primarily driven by net proceeds of $143.6 million from the February 2021 private placement, offset by cash used for operating activities and capital expenditures. The company expects that its cash, cash equivalents and investments as of June 30, 2021 will support its currently anticipated operating expenses and capital expenditure requirements into the first half of 2023. This cash runway excludes the additional $67 million that could become available under the companys credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers granted by the FDA following future U.S. approvals.

About Libmeldy / OTL-200

Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.

Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

About Orchard

Orchard Therapeuticsis a global gene therapy leader dedicated to transforming the lives of people affected by severe diseases through the development of innovative, potentially curative gene therapies. Ourex vivoautologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy inMilan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S.headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us onTwitterandLinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (TwitterandLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, the therapeutic potential of Libmeldy (OTL-200) and Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding its ongoing preclinical and clinical trials, including the timing of enrollment for clinical trials and release of additional preclinical and clinical data, the likelihood that data from clinical trials will be positive and support further clinical development and regulatory approval of Orchard's product candidates, and Orchards financial condition and cash runway into the first half of 2023. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchards ability to commercialize its product candidates, if approved, or Libmeldy, including the risk that Orchard may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedJune 30, 2021, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Condensed Consolidated Statements of Operations Data (In thousands, except share and per share data) (Unaudited)

Condensed Consolidated Balance Sheet Data (in thousands) (Unaudited)

Contacts

Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com

Media Benjamin Navon Director, Corporate Communications +1 857-248-9454 Benjamin.Navon@orchard-tx.com

Link:
Orchard Therapeutics Reports Second Quarter 2021 Financial Results and Highlights Recent Business Updates - StreetInsider.com

July 19, 2021 -- There are lots of unfounded fears about the COVID-19 vaccines floating around, and one of the most pervasive is the idea that these new shots aren't really vaccines, but that they will somehow change your genes or insert themselves into the DNA of your cells.

You may see people posting on social media about the vaccines being a kind of gene therapy, and they're partly right, but in the end this idea often misses some important details about how the vaccines work. They can't change your genes, and they don't stay in your body for more than a few days.

But plenty of people have distorted the way the vaccines work into something that could sound sinister. For example, in January, the Weston A. Price Foundation, a group that discourages vaccination, hosted a podcast where David Martin, PhD, described by FactCheck.org as a "financial analyst and self-help entrepreneur," called the vaccines gene therapy.

"a vaccine is supposed to trigger immunity. It's not supposed to trigger you to make a toxin," Martin said. "It's not a vaccination."

Except that these shots are vaccines, according to the US Food and Drug Administration (FDA), and they don't cause you to make a toxin.

So where did this idea get started?

"Like many rumors, there's sort of an element of truth," says Beth Thielen, MD, PhD, a pediatric infectious disease specialist at the University of Minnesota Medical School.

But the truth is that the vaccines involve sound science that sounds complicated to most people not educated in the field.

The vaccines made by Pfizer and Moderna use tiny oily envelopes called lipid nanoparticles to slip a single strand of genetic material called messenger RNA (mRNA) into our cells.

The Johnson & Johnson vaccine is slightly different. It uses double-stranded DNA inserted into a common, but inert virus called an adenovirus. This DNA also contains the instructions for building the spike protein. Once inside the cell, these instructions are read and translated into mRNA.

These bits of mRNA go into the jellied liquid called cytoplasm that makes up the body of our cells.

"Where they join about 200,000 other pieces of messenger RNA that are also sitting in every cell's cytoplasm, because our cells make proteins and enzymes all the time," says Paul Offit, MD, director of the vaccine education center at Children's Hospital of Philadelphia.

The mRNA chains are basically work orders that spell out the instructions for making the spike proteins that stud the outside of the coronavirus that cases COVID-19. The virus uses its spikes to dock onto our cells and infect them.

It's one of the viruses' most recognizable features. Our cells read this mRNA and use them to assemble the spikes. The spikes migrate to the outside of our cells where they are recognized and remembered by our immune system.

These spikes, by themselves, are not dangerous. They can't make anyone sick. They are essentially mug shots that help the body recognize and fight off the real perpetrator when it comes along.

The mRNA chains from the vaccines only last for a couple of days before they break down and the pieces are swept away by the body's normal waste disposal system.

Messenger RNA is genetic material, so in that sense, the vaccines are genetically based therapy.

But the FDA classifies them as vaccines, not gene therapy.

"I think people hear that and they think 'Oh my God, You're going to alter my DNA," Offit says. "That's not possible."

For the vaccines to alter a person's genes, Offit explains, the mRNA instructions would have to enter the cell's control center, the nucleus. The nucleus is walled off from the rest of the cell by its own membrane. To get past that membrane, the mRNA would have to have an enzyme called a nuclear access signal, Offit says, "which it doesn't have."

Even if it could get into the nucleus, the single strand of mRNA would have to be translated back into a double stranded DNA.

HIV, the virus that causes AIDS, can do this. It uses an enzyme like reverse transcriptase to insert itself into our chromosomes. The mRNA in the vaccines lacks this enzyme, so it can't turn back into DNA.

The DNA adenovirus used in the Johnson & Johnson vaccine does enter the nucleus of our cells, but it never integrates into our chromosomes.

Even after those two steps, there's a third firewall between the vaccines and our genes: Another enzyme, called an integrase, would be needed to stitch the new DNA into the DNA of our cells. That's also not in the vaccines.

"So the chances are zero that that can happen," Offit says.

One way to think about mRNA is to imagine if a friend wanted to make a delicious salad that you have the recipe for, Thielen says.

"You'd go to your cookbook, you'd copy the recipe on a note card and give it to them," she says. They can make the salad, but they don't have the cookbook, the original cookbook. You didn't change the cookbook, you just gave them a Post-it note or something that's temporary and that's meant to be," she says.

It's true that these are some of the first vaccines to work this way, but the technology was years in the making. The science was given a final push by billions in funding that was made available through Operation Warp Speed.

The vaccines have now been given to millions of people. They are some of the most effective in the world at preventing severe outcomes from COVID infections. So far, they are holding up well against all the viral variants.

While very rare side effects have been linked to the vaccines, so far, the FDA has determined that the benefit from taking one far outweighs these rare risks for most people.

"I've been astonished, actually,at how well it seems to be working. And so, I think it is very exciting from a vaccine development standpoint that we have new tools in our armamentarium to make new vaccines," Thielen says.

But there's still more to learn.

"I think we need to do dedicated studies of this platform to really understand how long the protection lasts and how well does it adapt to other vaccine targets like RSV. I think that remains to be seen," Thielen says.

Medscape Medical News

The rest is here:
Chance That COVID-19 Vaccines Are Gene Therapy? 'Zero' - WebMD

Media Advisory

Friday, July 16, 2021

Results provide hope for children with aromatic L-amino acid decarboxylase deficiency.

A new study published in Nature Communications suggests that gene therapy delivered into the brain may be safe and effective in treating aromatic L-amino acid decarboxylase (AADC) deficiency. AADC deficiency is a rare neurological disorder that develops in infancy and leads to near absent levels of certain brain chemicals, serotonin and dopamine, that are critical for movement, behavior, and sleep. Children with the disorder have severe developmental, mood dysfunction including irritability, and motor disabilities including problems with talking and walking as well as sleep disturbances. Worldwide there have been approximately 135 cases of this disease reported.

In the study, led by Krystof Bankiewicz, M.D., Ph.D., professor of neurological surgery at Ohio State College of Medicine in Columbus, and his colleagues, seven children received infusions of the DDC gene that was packaged in an adenovirus for delivery into brain cells. The DDC gene is incorporated into the cells DNA and provides instructions for the cell to make AADC, the enzyme that is necessary to produce serotonin and dopamine. The research team used magnetic resonance imaging to guide the accurate placement of the gene therapy into two specific areas of the midbrain.

Positron emission tomography (PET) scans performed three and 24 months after the surgery revealed that the gene therapy led to the production of dopamine in the deep brain structures involved in motor control. In addition, levels of a dopamine metabolite significantly increased in the spinal fluid.

The therapy resulted in clinical improvement of symptoms. Oculogyric crises, abnormal upward movements of the eyeballs, often with involuntary movements of the head, neck and body, that can last for hours and are a hallmark of the disease, completely went away in 6 of 7 participants. In some of the children, improvement was seen as early as nine days after treatment. One participant continued to experience oculogyric crises, but they were less frequent and severe.

All of the children exhibited improvements in movement and motor function. Following the surgery, parents of a majority of participants reported their children were sleeping better and mood disturbances, including irritability, had improved. Progress was also observed in feeding behavior, the ability to sit independently, and in speaking. Two of the children were able to walk with support within 18 months after receiving the gene therapy.

The gene therapy was well tolerated by all participants and no adverse side effects were reported. At three to four weeks following surgery, all participants exhibited irritability, sleep problems, and involuntary movements, but those effects were temporary. One of the children died unexpectedly seven months after the surgery. The cause of death was unknown but assessed to be due to the underlying primary disease.

Jill Morris, Ph.D., program director, NIHs National Institute of Neurological Disorders and Stroke (NINDS). To arrange an interview, please contact nindspressteam@ninds.nih.gov

Pearson TS et al., Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons, Nature Communications, July 12, 2021. https://doi.org/10.1038/s41467-021-24524-8

This study was supported by NINDS (R01NS094292, NS073514-01).

The NINDS NINDS is the nations leading funder of research on the brain and nervous system.The mission of NINDS is to seek fundamental knowledge about the brain and nervous system and to use that knowledge to reduce the burden of neurological disease.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

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funded study finds gene therapy may restore missing enzyme in rare disease - National Institutes of Health

ByGina Wadas

Viruses are experts at infiltrating the body, as the SARS-CoV-2 virus (and resulting COVID-19 pandemic) have amply demonstrated. But their efficiency in targeting specific and isolated cells also make them useful drug delivery vehicles, known as viral vectors.

Viral vectors are modified viruses that can act as couriers to transport therapeutic "packages" to specific diseased cells. These packages contain instructions with modified or designed DNA or RNA to correct or supplement a faulty or missing gene. For instance, the Johnson & Johnson COVID-19 vaccine uses viral vectors to transport modified genetic material from the SARS-CoV-2 virus to cells, generating an immune response.

Though viral vector-based gene therapies are among the most advanced treatments for many congenital and acquired diseases, producing them is complex and costly.

"One of the major challenges in viral vector gene therapy is how to improve the quality, purity, and cost of the manufactured viral vectors, so that we can use the smallest possible effective dose, reduce immune side effects, and lower the cost of treatments," said Hai-Quan Mao, associate director and core faculty member of the Institute for NanoBioTechnology. He is also a professor in the departments of Materials Science and Engineering and Biomedical Engineering and a core faculty member at the Translational Tissue Engineering Center.

Hai-Quan Mao

Associate director, Institute for NanoBioTechnology

To address this challenge, Mao and his team are teaming up with Nolan Sutherland, senior scientist at bluebird bio, a Cambridge, Massachusetts-based biotechnology company that develops gene therapies. The partnership started about two years ago when Yizong Hu, a biomedical engineering PhD student under the mentorship of Mao, was at an annual meeting for the American Society for Gene and Cell Therapy presenting his research on a new particle assembly technology. Sutherland heard the presentation and approached Hu to discuss the technology and its application to the production of lentiviral vectors, which are made from a family of viruses that infect people by reverse transcription of their RNA into DNA in their host cells' genome.

Sutherland thought that the Mao team's approach might help streamline transfection, a key step in producing viral vectors. During transfection, a polymer solution is combined with a mixture of DNA plasmids to form transfection particles, a cumbersome procedure involving complicated solution blending and strictly timed dosing.

Mao, Hu, and Yining Zhu, also a biomedical engineering PhD student, developed a more effective and shelf-stable formulation of DNA particles in a ready-to-dose form. They also discovered that size-controlled sub-micron particles are most effective in transfecting cells and producing viral vectors. This production method is based on the team's years of experience in controlling transfection vehicle characteristics to enhance performances and stability.

The team members validated their findings with Sutherland at bluebird bio using that company's bioreactor. They compared the new method with the industry standard, and the results showed improved vector production yield, shelf stability, handling stability, and quality control of the transfection process.

"With the drastic increase in demand for lentiviral vector-based cell therapy products ... this new technology will greatly improve the production quality, consistency, and yield of our therapeutic LVVs," Sutherland said.

The team reported its findings in Nano Letters and is scaling up production with an eye to transferring the technology to the marketplace.

"This work represents a great example how we can partner with corporate collaborators to accelerate the translation of discoveries on the bench to the industry. This type of collaboration with industry provides us opportunities to identify the technical gaps in the engineering solutions that we develop, and fine tune them to better address the real-world problems in a more targeted fashion," Mao said.

According to Sutherland, the partnership with Mao and his team has "allowed bluebird to pursue high risk/reward innovation in a space outside of its core expertise. The team has a keen eye for application to industry which has made the partnership incredibly productive."

Team members say that this new particle engineering technology will find a wide range of applications in the manufacture of a variety of viral vectors for gene and cell therapy applications.

Also contributing to the project are Jordan Green, professor in the Department of Biomedical Engineering and associate member at the INBT, and Sashank Reddy, assistant professor of plastic and reconstructive surgery at Johns Hopkins Medicine, medical director at Johns Hopkins Technology Ventures, and affiliate faculty member at the INBT.

Continued here:
Researchers partner with industry to create better gene therapy tools - The Hub at Johns Hopkins

Scientists from Ohio State University have developed a novel method for delivering gene therapy to specific regions of the brain. Now, they have evidence from a small clinical trial in children that the treatment could address a rare, inherited neurodegenerative disease. And they believe their technique could eventually be used to treat more common brain diseases, like Alzheimers and Parkinsons.

The Ohio State team developed the gene therapy to treat aromatic L-amino acid decarboxylase (AADC) deficiency, which hampers the bodys ability to make dopamine and serotonin and results in developmental delays and a range of motor and behavioral symptoms. The gene therapy uses a viral vector to carry DNA-expressing AADC to the brain.

In seven children with AADC deficiency, the gene therapy boosted the metabolism of dopamine, the researchers reported in Nature Communications. Within three months, six of the children were no longer experiencing oculogyric crises (OGC), which are eye spasms that commonly occur in children with the disorder. After a year, six of the participants had normal head control, the researchers said.

During the trial, the Ohio State team delivered the gene therapy directly to the midbrain, monitoring the spread of the DNA in real time using MRI imaging. They reported dramatic improvements in several symptoms beyond OGC, including sleep disturbances and irritable mood, according to the paper. Improvements in motor function took longer to emerge, they wrote, but were markedly better than what would normally be expected to occur in the natural course of AADC deficiency for patients in this age range (49 years) who have severe motor impairment.

RELATED: Taking gene therapy to the masses with innovations in diabetes, Alzheimer's and more

The ability to deliver gene therapy directly to the brainand monitor its effects with advanced imagingcould enhance efforts to treat a range of neurodegenerative disorders, said Krystof Bankiewicz, M.D., Ph.D., neurological surgery professor at the Ohio State College of Medicine, in a statement.

In fact, a handful of companies are working toward that goal. They include Lexeo Therapeutics, which is developing a gene therapy to deliver the APOE2 gene into the central nervous system in the hopes that it will slow the development of Alzheimers in people with two copies of the the high-risk APOE4 variant.

Another company examining brain-delivered gene therapy is VectorY, which recently raised $38 million to advance its work. VectorY is using viruses to carry genes into the brain that can encode therapeutic antibodies. It is using the newly raised funding to complete preclinical work on therapies for amyotrophic lateral sclerosis and Alzheimers.

Based on the success of their early-stage trial in AADC deficiency, the Ohio State researchers are planning clinical trials of their brain-delivered gene therapy in other incurable, debilitating diseases, they said.

The gene therapy approach described here represents many years of careful work to develop and to understand effective ways to deliver gene therapy to the brain, they wrote in the study. This work provides a framework for the treatment of other human CNS genetic diseases, and iterative refinement of the individual components of this approach will facilitate broader application.

More here:
Gene therapy delivered to the brain shows promise in children with rare neurodegenerative disease - FierceBiotech

Kriya Therapeutics cofounder and CEO Shankar Ramaswamy.

Over the past several years, breakthroughs in gene therapy have led to treatments for rare diseases that were deadly just a decade ago. Take Zolgensmain 2019, it was the first gene therapy approved by the FDA to treat spinal muscular atrophy, a rare genetic disease that affects the mobility of infants and children. But gene therapies have historically had two drawbacks: They are only used for rare diseases, and they carry a hefty price tag (treatment with Zolgensma costs $2.1 million).

Kriya Therapeutics is trying to overcome these obstacles by creating gene therapies for the massesand manufacturing them at a lower cost. On Wednesday, the startup announced that it had raised a $100 million Series B funding round to get it closer to this goal. The round was led by investors from Patient Square Capital and also involved investors from QVT, Dexcel Pharma, Foresite Capital, Bluebird Ventures, Transhuman Capital, Narya Capital, Amplo and the Juvenile Diabetes Research Foundation T1D Fund.

We think this is going to be an extraordinarily important therapeutic class that will revolutionize the treatment of many diseases, says Jim Momtazee, managing partner at Patient Square Capital.

The Silicon Valley-based company was founded in late 2019 by three pharmaceutical industry alums, including a former cofounder of Spark Therapeutics and the former president of United Therapeutics Corp. Shankar Ramaswamy, Kriyas CEO, was part of the foundational team at Roivant Sciences. The new round brings the companys total funding to $180 million; the company declined to reveal its valuation.

Kriyas main focus is its uniquely designed Adeno-associated virusesviruses that are harmless when they enter the body, but deliver instructions to cells that then pump out genes that are missing in some people with genetic diseases. Though the company still plans to develop treatments for rare diseases, what sets it apart is its focus on more common genetic diseases, like some forms of diabetes and obesity. So far, gene therapy has been really constrained in many respects from achieving its full potential, Ramaswamy says. We are believers in gene therapy being applied to rare diseases as well as prevalent diseases.

Ramaswamys goal is to build a company that can go from genetic target discovery to manufacturing and then full commercialization of new therapies, unlike a typical biotech startup that might partner with a large pharmaceutical company for the later stages of development (Ramaswamy says the company will be open to partnerships, but can bring a drug to commercialization on its own). Once the company discovers and develops new gene therapies, its 51,000-square-foot manufacturing facility in North Carolina can produce Adeno-associated viruses at scale to deliver the genes to patients in need. Ramaswamy says that capability will bring down the cost, with savings passed along to patients. I think the innovations that were delivering will make gene therapies much more affordable and accessible to patients, he says. We are very committed to not being a burden on the healthcare system.

The companys current pipeline of products are all preclinical, though Ramaswamy says that they plan to submit Investigational New Drug applications to the FDA for several products in late 2022 and early 2023. So far the company is developing gene therapies for type 1 diabetes, solid tumors and two eye conditions: geographic atrophy and uveitis. In the U.S., more than 3 million people combined have at least one of these conditions, meaning Kriya has a huge pool of potential customers. By comparison, there are fewer than 25,000 children and adults with spinal muscular atrophy in the country.

Ramaswamy says that the new capital will go toward continuing the companys explosive growthit now has 80 full-time employeesas well as refining its vector delivery platforms and manufacturing capabilities. In the future, the money will allow the company to continue to develop new gene therapies for diseases both common and rare. Were taking a very new approach, which is to think more broadly, Ramaswamy says.

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This Company Raised $100 Million To Bring Gene Therapy To The Masses - Forbes

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will host a virtual manufacturing day for analysts and investors. The event will be webcast live on Tuesday, July 27, 2021, from 10:00 a.m. to 1:00 p.m. ET.

Topics of discussion will include the companys unique three-pillar approach to the manufacturing process, its manufacturing capabilities, the regulatory environment for gene therapy manufacturing, and the immunology of gene therapy. A question and answer session will follow each formal presentation.

The event will feature presentations from Taysha senior leaders:

Registration for this event is available through LifeSci Events. A live video webcast will be available in the Events & Media section of the Taysha corporate website. An archived version of the event will be available on the website for 60 days.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including our preclinical product candidates, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, the potential market opportunity for these product candidates, our corporate growth plans and our plans to establish a commercial-scale cGMP manufacturing facility to provide preclinical, clinical and commercial supply. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

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Taysha Gene Therapies to Host Manufacturing Day - Business Wire

UDG Healthcare company Ashfield has launched a new network offering an end-to-end approach for the commercialisation of cell and gene therapies.

The network, called EmerGENE, will enable Ashfield to support small and midsize biotech companies with the commercialisation of their discoveries, the agency said in a statement.

EmerGENE was created by a multidisciplinary team and combines experts from Ashfield Health, Ashfield Engage and Ashfield Advisory.

It aims to deliver expert-led guidance and services to biotech companies throughout their clinical to commercial journey.

The network will provide guidance on commercialisation strategic support, early clinical development, distribution and logistics, market access and patient and HCP engagement and support.

At Ashfield, we look to embed ourselves into our customers businesses and use our expertise to create tangible solutions which best meet their needs, said Amar Urhekar, global president at Ashfield Health.

EmerGENE is no different, and with over 1,200 cell and gene therapy clinical trials currently underway globally, and 1,000 different manufacturers exploring cell and gene therapies right now, its clear that there is demand for support in this space, he added.

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Ashfield launches 'end-to-end' cell and gene therapy commercialisation network - PMLiVE