StemCell Therapy

Data on all eight patients demonstrate sustained engraftment and supranormal IDUA enzyme expression

Translation of metabolic correction to clinical outcomes in first two patients continues to support potential of hematopoietic stem cell gene therapy in a second neurometabolic disorder

Data support planned initiation of registrational trial in 2021

BOSTON and LONDON, Sept. 01, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics(Nasdaq: ORTX), a global gene therapy leader, today announced additional interim data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203, an investigationalex vivoautologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at theSan Raffaele Telethon Institute for Gene Therapy(SR-Tiget) inMilan, Italy. The readout from the primary endpoint at one year of follow-up is expected in 2021. Today's results are being shared virtually in an invited oral presentation at the 46th Annual Meeting of the European Society for Blood and Bone Marrow Transplantation (EBMT).

We continue to see encouraging data from the ongoing clinical trial in MPS-I, including promising preliminary clinical effects on motor development, acquisition of cognitive skilIs and growth in the first two patients that were treated now 1.5 and 2 years ago, respectively. Additionally, new preliminary analyses of radiological outcome measures suggest that treatment with OTL-203 leads to stabilization or improvement in disease-related neurological abnormalities, as measured by brain and spine MRI, which we look to confirm with longer follow-up, saidMaria Ester Bernardo, M.D., Ph.D., principal investigator at SR-Tiget. "These data, taken together with those from clinical studies of HSC gene therapy for other metabolic disorders and leukodystrophies, support the potential for this therapeutic approach to correct a wide spectrum of multisystemic manifestations of the disease, bringing clinically meaningful benefits for patients.

Interim Study Results

Eight patients with the severe Hurler subtype of MPS-I had been treated with OTL-203 in the ongoing proof-of-concept study, which completed enrollment in December 2019. As of July 2020, all patients had been followed for a minimum of six months, with the longest follow-up extending out to 24 months. Treatment with OTL-203 was generally well-tolerated with a safety profile consistent with the selected conditioning regimen. Consistent with previous analyses, treatment across all eight patients continued to demonstrate:

We continue to see positive trends in all biomarker and clinical measures as we follow patients in the OTL-203 proof of concept study for longer periods of time, saidBobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With a growing amount of data to support advancing this program, we have recently convened a panel of disease experts to develop a design for a registrational trial that we intend to take to the regulators in advance of initiating the study in 2021 and ultimately progressing towards commercialization.

About OTL-203 and MPS-I

Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (also known as GAGs). The accumulation of GAGs across multiple organ systems results in symptoms including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births. There are three subtypes of MPS-I; approximately 60 percent of children born with MPS-I have the most severe subtype, called Hurler syndrome, and rarely live past the age of 10 when untreated.

Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an investigationalex vivoautologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by theSan Raffaele Telethon Institute for Gene TherapyinMilan, Italy.

About Orchard

Orchard Therapeuticsis a global gene therapy leader dedicated to transforming the lives of people affected by rare 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. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, the therapeutic potential of Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding the timing of clinical trials for its product candidates, including the product candidates referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates. 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 severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs; the risk that Orchard will not realize the anticipated benefits of its new strategic plan or the expected cash savings associated with such plan; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be successfully developed, approved 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 or that long-term adverse safety findings may be discovered; 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; and the risk of delays in Orchards ability to commercialize its product candidates, if approved. 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, 2020, 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.

Contacts

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

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

See the rest here:
Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I - GlobeNewswire

The company is to invest 3.4m (around US$4.5m) alongside the grant.

The funds will support research into the manufacturing challenges associated with scaling gene therapies for widespread patient access, to further develop technologies to improve the safety and efficacy of current therapies, and to enable the treatment of genetic diseases with more complicated disease pathways the industry is not yet able to address.

Along with the creation of 11 new jobs in Edinburgh, the developer said it will further enhance its Pro10 platform, an AAV manufacturing process that can be scaled and applied throughout the group.

The grant will also advance development of the tool kit of inducible, repressible, tunable and responsive expression cassettes to be adopted in the current clinical pipeline and new disease targets.

Gene therapy has the potential to treat a wide range of diseases including certain forms of muscular dystrophy, congestive heart failure and some diseases of the central nervous system but, at present, only two market-approved therapies are available.

David Venables, president, AskBio Europe, commented: The grant awarded by Scottish Enterprise supports AskBio in working towards developing even safer and more effective gene therapies through improved development and manufacturing techniques. Science and innovation keep progressing, and that makes this an exciting time to develop this type of therapeutic agent.

AskBios technology is inside both currently approved AAV gene therapies, which include Luxturna, developed by Spark Therapeutics, for the treatment of patients with inherited retinal disease, and Zolgensma, developed by AveXis, for the treatment of patients with spinal muscular atrophy (SMA).

AveXis licenses AskBios self-complementary DNA technology for Zolgensma.

While the promise of such therapies is being shown, significant barriers remain before gene therapies can become more broadly impactful, according to AskBio.

With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, AskBio has generated hundreds of proprietary third generation AAV capsids and promoters, several of which have entered clinical testing.

BioPharma-Reporter (BPR) spoke to Ken Macnamara, (KM), PhD, chief operating officer, AskBio Europe,to get the AAV developers take on the factors preventing gene therapy going mainstream.

BPR: What criteria did AskBio have to fulfill to be awarded this grant?

KM: The research must be highly novel with significant risk from which a successful outcome will accelerate business growth within Scotland and globally.

BPR: What are the current manufacturing challenges associated with scaling gene therapies for widespread patient access?

KM: As we see growing evidence that gene therapy is a viable, transformational medicine, along with an acceleration in the number of AAV therapeutics moving towards regulatory approval, the ability to manufacture these therapies for diseases with large patient populations does not exist today and costs are extremely high.

Many companies can manufacture small batches of therapeutics for clinical applications, but as they approach commercialization, the challenges of production costs and timelines remain an issue. We recognized this more than a decade ago and focused on creating robust, scalable manufacturing capabilities.

Today, the challenges for manufacturing gene therapy are being met by simply adding large amounts of capacity, which is not the long-term answer. There is a significant amount of innovation taking place that will no doubt shape the future of manufacturing AAV gene therapeutics. This work continues today in our Edinburgh and US facilities to further improve the technology.

BPR: What are some of the typical safety and efficacy issues linked to current therapies?

KM: Currently approved gene therapies have provided effective therapy by targeting tissues in the body with an administered gene that produces a new, effective protein. This new gene replaces the defective or missing gene causing the patients underlying disease.

Because the techniques are relatively new, some of the risks may be unpredictable; however, medical researchers, institutions, and regulatory agencies are working to ensure that gene therapy research is as safe as possible.

AAV is not known to cause human disease, and it cannot make more of itself without outside help, so it will not replicate in the body like normal viruses do. AAV is engineered to carry therapeutic genes by removing some of its genetic cargo and replacing it with human gene sequences. This results in an AAV vector, a therapeutic genetic medicine.

Risks associated with AAV gene therapy vector administration include unwanted immune system reactions. The body's immune system may see the newly introduced AAV vectors as intruders and attack them, which may cause inflammation and, in severe cases could be local and mild or throughout a greater area of the body and be more serious. AAV vectors can also target tissues other than the intended tissue. Thus, it's possible that AAV vectors may affect additional cells, not just the targeted cells containing mutated genes. These are called off-target effects. If this happens, healthy cells may be damaged.

BPR: Can you indicate the other significant barriers that remain before gene therapies can become more broadly impactful?

KM: Therapies need to express the gene in the right tissue, at the right level, for the right amount of time. There is a great deal of research happening throughout the gene therapy field to identify the best means of delivering and controlling activation of the genetic material. Furthermore, the response of the patients immune system also needs to be considered based on the therapy. Additional funding, like that from Scottish Enterprise, can help speed up the development process of promising therapies.

BPR: How does AskBio envisage exploring the treatment of genetic diseases with more complicated disease pathways that the industry is not yet able to address?

KM: One of the most exciting advances in modern medicine has been the discovery of how AAV vectors can be used as an effective delivery system for therapeutic genetic material into living tissue. AAV gene therapy has broad therapeutic implications for a vast array of diseases.

Some genetic diseases are caused by mutations in a single gene, while others are a result of mutations in multiple genes, for example, cancer. Additionally, environmental factors, such as smoking and diet, can play a role in diseases. The complexity of these disease characteristics creates variables in developing and testing potential treatments. Currently the gene and cell therapy options that exist today are limited to treating diseases caused by a single gene mutation.

AskBios Edinburgh team leads the gene therapy field in the design and development of synthetic gene expression cassettes. The technology is essential for controlling the expression of AAV therapeutics, thereby improving their safety and efficacy. This R&D project will enable AAV therapeutics to be turned on and off and dialed up or down depending on the amount of drug needed at any given time. This technology provides a desired safety switch and level of variable dosing that previously did not exist. Before this breakthrough, AAV therapeutics could only express at one constant level and could not be turned off, which limited the type of therapeutics for which AAV could be used and may hold the key to treating pathway diseases where multiple genes are affected.

BPR: On the job creation front, is the talent already hired or are you starting a recruitment drive?

KM: The grant allows us to make some positions permanent and bring in new talent.

Ken Macnamara joined AskBio in 2019 with a wealth of R&D, business operations, financial planning, intellectual property and quality/compliance experience gained from start-up to multinational firms. He most recently was COO at Synpromics.

Dr Macnamara began his career at the University of Edinburgh, where he earned a PhD in chemistry before helping to start Lab901 (Scottish SME). There, he was a product development manager responsible for developing the TapeStation and ScreenTape technologies from concept to market success. Lab901 was acquired by Agilent Technologies in 2011. Dr Macnamara then served as R&D director for the Microfluidics business at Agilent.

Read more:
Reducing barriers to mainstream gene therapy - BioPharma-Reporter.com

Paula Cannon. (USC Photo/Richard Carrasco)

An HIV research program led by scientists at USC and the Fred Hutchinson Cancer Research Center in Seattle has received a five-year, $14.6million grant from the National Institutes of Health. The team is advancing a gene therapy approach to control the virus without the need for daily medicines.

The programs co-directors are Paula Cannon, PhD, Distinguished Professor of Molecular Microbiology and Immunology at the Keck School of Medicine of USC, and Hans-Peter Kiem, MD, PhD, the Stephanus Family Endowed Chair for Cell and Gene Therapy at Fred Hutch. Other key partners are David Scadden, MD, a professor at Harvard University, and the biotechnology company Magenta Therapeutics.

The NIH award will support preclinical studies that combine gene editing against HIV with technologies for safer and more effective hematopoietic stem cell transplants. Such transplants, also known as bone marrow transplants, are currently used for severe blood cancers. They renew a patients immune system, which can be damaged by cancer therapies, by infusing healthy donor blood stem cells that can grow into any type of blood or immune cell.

The researchers goal is to build a therapy that prepares patients for a stem cell transplantation using their own cells with little to no toxicity, engineers their own stem cells to fight HIV and stimulates those cells to quickly produce new and engineered immune cells once theyre reintroduced into the patient.

This grant funds a team with an overarching goal of developing what our perfect HIV gene therapy would look like, Cannon said. All of these pieces could happen separately, but the fact that the NIH has funded us as a team means that the sum will be so much bigger than the parts.

Halting HIV without daily drugs

About 38million people worldwide are living with HIV, the virus that causes AIDS. HIV is manageable with daily antiretroviral drugs, but the research team seeks a more durable solution.

Their strategy is inspired by the three cases where patients seem to have been cured of HIV. All had aggressive leukemia and received blood stem cell transplants from donors who also carried a mutation that confers immunity to HIV. The mutation was in the CCR5 gene, which encodes a receptor that HIV uses to infect immune cells and is present in about 1 percent of the population.

Timothy Ray Brown, famously nicknamed the Berlin patient, received such a transplant in 2007; he has been off antiretroviral drugs since then, and the virus remains undetectable in his system. In recent years, patients in London and Dusseldorf have shown similar results.

I think of the Berlin patient as proof of principle that replacing the immune system with one thats HIV-resistant by removing CCR5 is a possible way to treat somebody, Cannon said.

However, the rigors of the blood stem cell transplant process, combined with the difficulty in finding tissue-matched CCR5-negative donors, make it highly unlikely that this will provide more than a very rare treatment.

Three for one gene therapy

The research team will tackle these two major problems. First, to get around the lack of CCR5-negative donors, Cannon has already helped pioneer the use of gene editing to remove CCR5 from a patients own stem cells. This is now an investigational treatment for HIV in a clinical trial at City of Hope in Duarte, California.

She will now combine CCR5 disruption with additional genetic changes, so that the progeny of engineered stem cells will release antibodies and antibody-like molecules that block HIV.

Our engineered cells will be good neighbors, Cannon said. They secrete these protective molecules so that other cells, even if they arent engineered to be CCR5-negative, have some chance of being protected.

Meanwhile, Kiems group is providing a third approach by adapting an emerging cancer treatment called CAR T cell therapy. This re-engineers T cells of the immune system with chimeric antigen receptors (CARs), which are customized to recognize cancer cells.

In this project, Kiem and colleagues will create stem cells whose T cell descendants can instead hunt down HIV-infected cells.

A gentler blood stem cell transplant

The grant also supports two other components that relate to the blood stem cell transplant.

Magenta Therapeutics is developing less-toxic protocols for conditioningpreparing a patients bone marrow to receive a transplant. Traditionally, mild chemotherapy or radiotherapy is needed to make room for newly infused stem cells and to help them re-engraft.

The company is instead using antibody-drug conjugates to deliver this conditioning much more narrowly and to reduce the side effects that occur with systemic chemo or radiation.

Meanwhile, Scadden and his team are addressing another drawback of stem cell transplants and conditioning, the delay before infused stem cells generate new immune cells in sufficient numbers. In cancer patients, this delay leaves them highly susceptible to infection.

Scadden is approaching this using an injectable gel that biochemically resembles the bone marrow environment, to quickly repopulate the immune system with HIV-fighting cells.

With success, the teams research may free HIV patients from the need for daily medication and the expense and potential side effects that come with it. Their work may also improve other therapies based on blood stem cells, for conditions such as cancer, sickle cell disease and autoimmune disorders.

A home run would be that we completely cure people of HIV, Cannon said. What Id be fine with is the idea that somebody no longer needs to take anti-HIV drugs every day because their immune system is keeping the virus under control, so that it no longer causes health problems and, importantly, they cant transmit it to anybody else.

By Wayne Lewis

The rest is here:
Gene therapy research for HIV awarded $14.6 million NIH grant - USC News

Could gene therapy provide a solution to HIV? A new research project aims to find out.

The National Institutes of Health(NIH) has backed researchers at the University of Southern California and the Fred Hutchison Cancer Center with a five-year, $14.6 million grant to develop a gene therapy that could potentially control HIV without the need for daily medications. Most HIV patients take a well-regimented cocktail of medications each day to control the virus. This therapy could change that. According to an announcement from the Keck School of Medicine at USC, the goal will be to develop a therapy that prepares patients for a stem cell transplantation using their own cells with little to no toxicity, engineers their own stem cells to fight HIV and stimulates those cells to quickly produce new and engineered immune cells once they're reintroduced into the patient. The hematopoietic stem cell transplants, also known as bone marrow transplants, have been used to treat some blood cancers. The idea is to infuse an HIV patient withhealthy donor blood stem cells that can grow into any type of blood or immune cell.

The gene therapy strategy has been inspired by three cases where leukemia patients who also had HIV received blood stem cell transplants from donors who also carried a mutation that confers immunity to HIV. The mutation was in the CCR5 gene, which encodes a receptor that HIV uses to infect immune cells and is present in about 1 percent of the population, USC said.

The program will engineer blood cells to remove CCR5 from a patient's own stem cells.That will be combined with other genetic changes so that the progeny of engineered stem cells will release antibodies and antibody-like molecules that block HIV.

In addition to the potential gene therapy treatment, researchers are also assessing whether or not CAR-T treatments will benefit HIV patients. Researchers from Harvard University developed a Dual CAR T-cell immunotherapy that can potentially help fight HIV infection. First reported by Drug Target Review, the HIV-specific CAR-T cell is being developed to not only target and eliminated HIV-infected cells, but also reproduce in vivo to enable the patients to fight off the infection. HIVs primary target it T cells, which are part of the bodys natural immune response.

Todd Allen, a professor of Medicine at Harvard Medical School, said the Dual CAR-T cell immunotherapy has so far provided a strong, long-lasting response against HIV-infection while being resistant to the virus itself.

According to the report, theDual CAR T cell was developed through the engineering of two CARs into a single T cell. Each of the CARs contained a CD4 protein that allowed it to target HIV-infected cells and a costimulatory domain, which signaled the CAR T cell to increase its immune functions. As DTR reported, the first CAR contained the 4-1BB co-stimulatory domain, which stimulates cell proliferation and persistence, while the second has the CD28 co-stimulatory domain, which increases its ability to kill infected cells.

To protect the CAR-T cells from HIV, the team added the protein C34-CXCR4, which prevents HIV from attaching to and infecting cells. When that was added, the researchers found in animal models that the treatment was long-lived, replicated in response to HIV infection, killed infected cells effectively and was partially resistant to HIV infection.

Still, other researchers are looking to those rare individuals who are infected with HIV but somehow on their own are able to suppress the virus without the need for any treatment. Researchers have sought to replicate what this small percentage of patients can naturally do in other patients who require those daily regimens of medications. Through the sequencing of the genetic material of those rare individuals, researchers made an interesting discovery.

The team discovered large numbers of intact viral sequences in the elite controllers chromosomes. But in this group, the genetic material was restricted to inactive regions, where DNA is not transcribed into RNA to make proteins, MedNewsToday reported.

Now the race is on to determine how this can be replicated and used to treat the nearly 38 million people across the globe who have been diagnosed with HIV.

The rest is here:
New HIV Gene Therapy, CAR-T Treatments Could be on the Horizon for Patients - BioSpace

As many as 40,000 people in Germany suffer from retinitis pigmentosa. This hereditary disorder is characterized by loss of photoreceptors in the retina, and can be caused by mutations in many different genes. Depending on the nature of the underlying genetic defect, the severity of the condition can vary between night blindness and progressive visual field loss that can ultimately result in total blindness. The first gene therapies for the disease have recently been approved. However, these approaches have certain disadvantages, which limit their range of application.

A research team led by PD Dr.Elvir Becirovicat the Department of Pharmacology of Natural Sciences (Head: Prof. Dr.Martin Biel) has developed a new strategy in collaboration with Prof. Dr.Stylianos Michalakisof the Opthalmology Clinic in the LMU Medical Center. This approach is designed to compensate for the causative hereditary defect by activating genes with similar functions that are normally repressed in the affected tissues, and utilizes a variant of the CRISPR/Cas9 technology that was first described in 2015. In the online journalScience Advances, the team describes the first successful application of this method in the context of gene therapy.

Currently, two strategies are being used in the development of gene therapies: In the context of gene supplementation, an attempt is made to replace the defective gene with an intact version. However, this is currently only possible for relatively small genes. The second strategy aims to correct disease-causing mutations, but this usually has to be tailored to each individual mutation. In view of the high effort and the associated development costs, a broad application of this strategy is therefore not possible. "To overcome these limitations, we have developed a new strategy," says Becirovic.

Many genes in the human genome fall into families, whose members fulfill similar functions in different cell types, or are activated at different stages during the differentiation of a particular cell type. Our idea was to compensate for the mutant genes loss of function by specifically activating genes that have a similar function but are normally not expressed in retinal cells, says Becirovic. To do so, we delivered a system called Cas9-VPR into the affected retinal cells. The Cas9-VPR system is a derivative of the CRISPR/Cas9 technology that is widely used for the targeted modification of genes. Akin to the classical CRISPR/Cas9 system, Cas9-VPR utilizes the same targeting principle to guide an activating protein to the particular gene of interest.

Becirovic and colleagues made use of a mouse model for retinitis pigmentosa to test the activation approach. These mice lack the light-sensitive rhodopsin protein that is normally expressed exclusively in the rod cells of the retina, which are required for dim light and night vision. The researchers delivered the Cas9-VPR system into the rod cells with the aid of a harmless virus. By introducing Cas9-VPR into the rods of the mice, the scientists switched on genes closely related to the rhodopsin gene, which are normally active in the cones responsible for color and daylight vision. In this way, we were able to compensate for the lack of rhodopsin function in the rod cells, to attenuate the rate of retinal degeneration and improve retinal function without detectable side-effects, says Becirovic.

The authors believe that a similar strategy can be applied to a wide range of genes and genetic diseases, and offers a number of significant advantages over existing strategies. Given the growing importance of gene therapy and its potential benefits for patients, we are convinced that our approach could soon be used in initial clinical feasibility studies, says Becirovic.

Reference: Bhm S, Splith V, Riedmayr LM, et al. A gene therapy for inherited blindness using dCas9-VPRmediated transcriptional activation. Sci Adv. 2020;6(34):eaba5614. doi:10.1126/sciadv.aba5614.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

See more here:
A New Target for Congenital Blindness Gene Therapy Is in Sight - Technology Networks

For the first time ever, a team of neurologists and neurosurgeons atThe Ohio State University Wexner Medical CenterandThe Ohio State College of Medicinehas performed a novel gene therapy brain infusion to treat a patient with Parkinsons disease.

This multicenter, Phase 1bclinical safety studyis sponsored byBrain Neurotherapy Bio, Inc.and funded by theCalifornia Institute for Regenerative Medicineto test GDNF gene therapy in patients with early to moderate stages of Parkinsons disease. The one-time treatment involves infusion of a gene therapy solution into deep structures of the brain that are affected by the disease.

Parkinsons disease is a neurodegenerative movement disorder that affects one million people in the United States. Degeneration of neural pathways deep in the brain causes symptoms such as tremor, slow movement and behavioral abnormalities, said Ohio State neurosurgeon Dr. James Brad Elder who performed the gene therapy surgery on Aug. 25.

The overall goal of this gene therapy treatment strategy is to slow the neurologic deterioration associated with Parkinsons disease by enhancing levels of a naturally occurring growth factor called GDNF. Targeting gene therapy delivery to the putamen, a deep brain structure affected by Parkinsons disease, will hopefully improve overall quality of life, Elder said.

The patient, a 55-year-old Ohio man first diagnosed with Parkinsons disease in 2008, takes medicine to help control his progressively worsening disease. He said the gene therapy surgery gives him hope that his disease wont get any worse, and that he may even feel better without medications. But he added that it could take up to six months before he notices any improvements.

There has long been evidence in animal and cell culture models of Parkinson's disease suggesting that glia cell derived neurotrophic factor (GDNF) has promise as a therapy for the disease, said Dr. Sandra Kostyk, director of theMovement Disorders Divisionat Ohio State Wexner Medical Center. Patients with Parkinsons disease and related disorders are diagnosed and treated in the Movement Disorders clinics and neurosurgery programs at Ohio State.

One of the biggest clinical hurdles has been getting the molecule to the regions in the brain that would benefit these patients the most. GDNF is a relatively large molecule that cant be administered as a pill, nor intravenously, since it cant penetrate the blood brain barrier.

This new targeted gene delivery approach overcomes many of the obstacles that have slowed GDNF clinical trial research and is expected to facilitate the production of a continuous supply of GDNF to a critical region of the brain affected by Parkinsons disease.

This is a onetime treatment strategy that could have ongoing lifelong benefits. Though its hoped that this treatment will slow disease progression, we dont expect this strategy to completely stop or cure all aspects of the disease.Were cautiously optimistic as this research effort moves forward, Kostyk said.

Brain Neurotherapy Bio is a biotechnology startup company founded in 2018 byDr. Krystof Bankiewiczto develop gene therapies for neurological disorders. Bankiewicz is also a member of Ohio State Wexner Medical CentersNeurological Institute.

Ive been investigating therapeutic gene therapy approaches for Parkinsons disease for nearly 30 years, and this marks a significant milestone that may lead to major therapeutic opportunities for those suffering with this devastating condition, said Bankiewicz, who is CEO and chairman of the board of Brain Neurotherapy Bio.

Additional sites for this clinical trial include the University of California San Francisco and the University of California Irvine medical centers. For more information, emailOSUgenetherapyresearch@osumc.edu.

Read the original post:
Wexner Medical Center performs gene therapy brain infusion for Parkinson's disease - The Highland County Press

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, a patient-centric gene therapy company with a mission to eradicate monogenic CNS disease, today announced the appointment of its executive leadership team. This group has significant experience in gene therapy drug development and commercialization, and will enable Taysha to build the corporate culture and infrastructure necessary to advance its extensive pipeline of 18 gene therapy programs, with exclusive options to acquire four additional programs from UT Southwestern Gene Therapy Program. In addition, Sukumar Nagendran, M.D., former Chief Medical Officer of AveXis, and Phillip Donenberg, former Chief Financial Officer of AveXis, have joined the companys Board of Directors. Mr. Donenberg will also serve as the companys Audit Committee Chairman.

Joining the Taysha Board is a unique opportunity to contribute to scientific advancements in CNS gene therapy, said Sukumar Nagendran, M.D., Taysha Board of Directors. In partnership with UT Southwestern, Taysha has built an extensive pipeline of gene therapy candidates for life-threatening CNS diseases with significant unmet medical need.

It is a distinct pleasure to be reunited with many of my former AveXis colleagues that enabled the development and successful commercialization of Zolgensma, said Phillip Donenberg, Taysha Board of Directors. I am excited to contribute to Tayshas efforts to deliver therapies with the potential to improve the lives of patients with devastating CNS disease.

Each member of the Taysha leadership team has significant gene therapy expertise, with an unrelenting, patient-first focus guiding their individual areas of focus. Joining RA Session II, Founder, President and CEO of Taysha, on the management team are the following individuals:

From day one, we set out to build a team that has the passion, experience and talent to achieve our mission of eradicating monogenic CNS disease. Today, we are proud to announce a highly experienced team of CNS gene therapy experts, said Mr. Session. We are also pleased Dr. Nagendran and Mr. Donenberg have joined our Board and will contribute their several years of gene therapy expertise. Their experience in building AveXis will be invaluable as we continue to grow and advance several programs into the clinic.

About Taysha Gene Therapies

Taysha Gene Therapies is a patient-centric gene therapy company with a mission to eradicate monogenic CNS disease. We are focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the CNS in both rare and large patient populations. We were founded in partnership with The University of Texas Southwestern Medical Center, or UT Southwestern, to develop and commercialize transformative gene therapy treatments. Together with UT Southwestern, we are advancing a deep and sustainable product portfolio of 18 gene therapy product candidates, with exclusive options to acquire four additional development programs. By combining our management teams proven experience in gene therapy drug development and commercialization with UT Southwesterns world-class gene therapy research capabilities, we believe we have created a powerful engine to develop transformative therapies to dramatically improve patients lives. More information is available at http://www.tayshagtx.com.

See more here:
Taysha Gene Therapies Builds Experienced Executive Leadership Team to Advance Pipeline of Gene Therapies for Monogenic CNS Disease in Both Rare and...

Sept. 2, 2020 12:00 UTC

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, today announced the appointment of two new executives, Lisa M. Olson, Ph.D., as Chief Scientific Officer and Kevin B. Johnson, Ph.D., as Senior Vice President, Head of Regulatory and Quality. The Company also announced that Jason Ryan will transition from Chief Operating and Financial Officer to a consulting role for personal reasons while a search for his replacement is conducted.

With the additions of Lisa and Kevin to our team, Magenta continues to deepen our technical expertise, bolstering our strong discovery, research, development and regulatory leadership to further our goal of delivering curative immune reset to patients in need, said Jason Gardner, D.Phil., Chief Executive Officer and President, Magenta Therapeutics. We are delighted to welcome Lisa and Kevin on board and look forward to their many contributions to the Magenta mission.

As Chief Scientific Officer, Dr. Olson will provide strategic direction, oversight and execution for Magentas research and discovery efforts. This entails driving research strategy as Magenta continues to optimize its preclinical and clinical pipeline. She will join the executive team and will be a key member of the R&D leadership team.

Dr. Olson is an experienced senior-level pharmaceutical executive, with more than 20 years of experience in research and drug discovery. She comes to Magenta following 15 years in leadership positions at the AbbVie Bioresearch Center, most recently as Vice President, Immunology Discovery and Site Head, where she was responsible for all immunology discovery scientific and portfolio decisions, including new target approval, project advancement and licensing opportunities. Under her leadership, 15 molecules advanced into clinical development, including Upadacitinib that launched last year as Rinvoq. Prior to AbbVie, she served as a Research Fellow and Group Leader in Inflammation & Immunology at Pfizer, Inc. She began her career as an Assistant Professor at Washington University School of Medicine, following a post-doctoral cardiovascular fellowship at the University of Chicago.

Dr. Olson holds a Ph.D. from the University of Illinois at Urbana-Champaign and a Bachelor of Science from Iowa State University.

As Senior Vice President, Head of Regulatory and Quality, Dr. Johnson will lead Magentas global regulatory strategy for the Companys programs across multiple therapeutic areas. He will also be responsible for the oversight and accountability for all quality activities to enable Good Practice (GxP) functions across the portfolio. In this role, Dr. Johnson will provide strategic guidance and leadership to members of the R&D leadership team and the regulatory and quality teams for Magentas portfolio for all phases of product lifecycle.

Dr. Johnson bring years of regulatory, quality assurance and development leadership, coming to Magenta from Imara, Inc., where he served as Senior Vice President, Regulatory Affairs, Quality and Pharmacovigilance, leading successful requests for several regulatory designations with the U.S. Food and Drug Administration (FDA). Prior to his time at Imara, Dr. Johnson led global regulatory strategy and implementation for breakthrough therapy-designated rare disease development programs at Vtesse, later acquired by Sucampo. He also served as Director, Global Regulatory Affairs for Rare Diseases and Gene Therapies at GlaxoSmithKline, where he was part of on the international regulatory team for the European approval of the gene therapy Strimvelis for ADA-SCID, and subsequently secured Regenerative Medicine Advanced Therapy (RMAT) designation for a retinal gene therapy product.

Dr. Johnson holds a Ph.D. in Neurobiology from the University of North Carolina (UNC) School of Medicine; a Master of Business Administration from the Kenan-Flagler School of Business, UNC; and a Bachelor of Science in Chemistry from the University of South Florida.

Along with these leadership team additions, Magenta also announced today that Jason Ryan, Chief Operating and Financial Officer, will step down from that role at the end of September. He will continue to contribute to Magenta in a consulting capacity, and the Company has commenced a search for a replacement.

Jason has been a dynamic and reliable leader at Magenta since he joined us in 2019, leading finance and operations, contributing to our strategic planning efforts, and spearheading two financings during a period of significant growth, said Gardner. We are truly grateful for his contributions to the patients we seek to serve, our employees and business partners.

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with autoimmune diseases, genetic diseases and blood cancers. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking Statement

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption Risk Factors in Magentas Annual Report on Form 10-K filed on March 3, 2020, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200902005236/en/

See the original post here:
Magenta Therapeutics Appoints Lisa M. Olson as Chief Scientific Officer and Kevin B. Johnson as Senior Vice President, Head of Regulatory and Quality;...

REDWOOD CITY, Calif. and RESEARCH TRIANGLE PARK, N.C., Sept. 3, 2020 /PRNewswire/ --Kriya Therapeutics, a next generation gene therapy company focused on developing transformative treatments for highly prevalent diseases,announced today that its CEO, Shankar Ramaswamy, M.D., will present at multiple upcoming healthcare conferences in September and October. These include the following:

Citi's 15th Annual BioPharma Virtual ConferenceDate: Tuesday, September 8thTime: 3:30 PM ET / 12:30 PM PT

H.C. Wainwright & Co. 22nd Annual Global Investment ConferenceDate: Wednesday, September 16thTime: 9:30 AM ET / 6:30 AM PT

Cantor Fitzgerald Virtual Global Healthcare ConferenceDate: Thursday, September 17thTime: 3:20 PM ET / 12:20 PM PT

Chardan 4th Annual Genetic Medicines ConferenceDate: Tuesday, October 6thTime: 9:00 AM ET / 6:00 AM PT

About Kriya Therapeutics

Kriya Therapeutics is a next-generation gene therapy company focused on developing transformative treatments for highly prevalent serious diseases. With core operations in California and North Carolina, Kriya's technology-enabled platform is directed to the rational design and clinical translation of gene therapies for large patient populations. For more information, please visit http://www.kriyatx.com.

Cautionary Note on Forward-Looking Statements

This press release includes forward-looking statements pertaining to our development programs and our proprietary platform. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. The forward-looking statements contained in this press release reflect Kriya's current views with respect to future events, and Kriya does not undertake and specifically disclaims any obligation to update any forward-looking statements.

ContactDan ChenChief Financial Officer[emailprotected]

SOURCE Kriya Therapeutics

https://www.kriyatx.com/

See the rest here:
Kriya Therapeutics To Present At Upcoming Healthcare Conferences - PRNewswire

New Phase 1b Data of Investigational Gene Therapy Compound, VY-AADC (NBIb-1817), Evaluating Three-Year Safety and Clinical Outcomes in Patients with Advanced Parkinsons Disease

Voyager to Participate in Upcoming September Investor Conferences

CAMBRIDGE, Mass., Sept. 03, 2020 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ: VYGR), a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases, today announced data presentations at the International Parkinson and Movement Disorder Virtual Congress 2020 taking place on September 12-16, 2020. The presentations include new two- and three-year data related to its VY-AADC gene therapy treatment for Parkinsons disease being developed in collaboration with Neurocrine Biosciences:

Additionally, the company plans to participate in the following virtual investor conferences in September:

The webcast sessions may be accessed from the Investors & Media section of Voyagers website at http://www.voyagertherapeutics.com. Replays of the webcasts will be archived on the Company's website for at least 30 days.

About Voyager Therapeutics

Voyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. Voyagers wholly owned and partnered pipeline focuses on severe neurological diseases for which effective new therapies are needed, including Parkinsons disease, Huntingtons disease, Friedreichs ataxia, and other severe neurological diseases. For more information, please visit http://www.voyagertherapeutics.com or follow @VoyagerTx on Twitter and LinkedIn.

Investor Relations: Paul CoxVP, Investor Relations857-201-3463pcox@vygr.com

Media: Sheryl Seapy W2Opure949-903-4750sseapy@purecommunications.com

Read more here:
Voyager Therapeutics Announces Upcoming Presentations at the International Parkinson and Movement Disorder Society Virtual Congress 2020 -...

The NIH is pitching $14.6 million into a three for one HIV research program led by USC and the Fred Hutchinson Cancer Research Center that aims to strike the need for daily medication or even achieve a home run cure.

The five-year grant will back preclinical studies that combine gene editing with technology to improve bone marrow transplants. The potential therapy would engineer a patients own stem cells to fight HIV, and stimulate them to produce new immune cells once reintroduced to the patient.

A home run would be that we completely cure people of HIV, Paula Cannon, a USC professor of molecular microbiology and immunology and co-director of the program, said in a statement. What Id be fine with is the idea that somebody no longer needs to take anti-HIV drugs every day because their immune system is keeping the virus under control, so that it no longer causes health problems and, importantly, they cant transmit it to anybody else.

Hans-Peter Kiem, the Stephanus Family Endowed Chair for Cell and Gene Therapy at Fred Hutch, is the co-director. Harvard University professor David Scadden and Magenta Therapeutics are also collaborating on the project.

The approach was inspired by three patients who appear to have been cured of the virus all of whom received blood stem cell transplants from donors who carried a mutation in the CCR5 gene. One of them, dubbed the Berlin patient, has been off antiretroviral drugs since 2007.

I think of the Berlin patient as proof of principle that replacing the immune system with one thats HIV-resistant by removing CCR5 is a possible way to treat somebody, Cannon said.

The program will study the use of gene editing to remove CCR5 from patients stem cells a process which is already in clinical trial for HIV treatment at City of Hope National Medical Center in Duarte, CA. The stem cells will also be engineered to release antibodies and antibody-like molecules that block HIV.

In addition, the grant will fund a Fred Hutch teams endeavor to adapt CAR-T cell therapy to create stem cells whose progeny target HIV-infected cells.

As for preparing a patient for the transplant,Magenta is working on antibody-drug conjugates to replace mild chemotherapy or radiotherapy typically given before the procedure. And Scadden is researching an injectable gel that could help immune cells repopulate more quickly, avoiding a delay.

HIV infection, which currently affects about 1.2 million Americans, has proved to be exceedingly difficult to cure. In July, Merck and Dewpoint inked a deal that allows the pharma to use the Boston-based biotechs biomolecular condensate technology to develop treatments, and potentially a cure, for the HIV virus. The NIH-funded group is hoping to at least control the virus enough to eliminate the need for daily meds. But at best, theyre also eyeing a long sought-after cure.

This grant funds a team with an overarching goal of developing what our perfect HIV gene therapy would look like, Cannon said. All of these pieces could happen separately, but the fact that the NIH has funded us as a team means that the sum will be so much bigger than the parts.

Original post:
Researchers teamed up to develop a 'three in one' HIV treatment and the NIH is throwing in $14.6M - Endpoints News

Fouad Namouni, a storied research exec who went from project leader on Opdivo and Yervoy to the top of the oncology research group at Bristol Myers Squibb, is joining the migration to biotech, picking up a new hat as president of R&D at Blueprint Medicines.

Once again, hes headed into a toe-to-toe showdown with a rival pharma organization.

Namouni will likely be coming on board just one step ahead of an approval for pralsetinib, Blueprints RET rival to Eli Lillys Retevmo, which got out in front with a May approval. Ironically, Lillys deal to buy into RET with its acquisition of Loxo also brought Josh Bilenker and his crew to the pharma giant, marking a rare career trajectory from a biotech into pharma, which has been bleeding talent for years now.

Unlock this story instantly and join 89,400+ biopharma pros reading Endpoints daily and it's free.

SUBSCRIBE SIGN IN

Continued here:
Game change: A frontrunner in the cell therapy 2.0 field offers a first look at their lead therapy. And it's a doozy - Endpoints News

CAMBRIDGE, Mass., Sept. 3, 2020 /PRNewswire/ -- Obsidian Therapeutics, Inc., a biotechnology company pioneering controllable cell and gene therapies, today announced the appointment of Robert Ross, M.D., to its Board of Directors. Dr. Ross currently serves as the Chief Medical Officer of Surface Oncology.

"Rob brings strong clinical development experience, specifically in oncology, which will be invaluable as we advance towards the clinic," said Paul K. Wotton, Ph.D., Obsidian's Chief Executive Officer. "Rob has successfully advanced multiple programs from IND to pivotal trials, as well as led collaborations with industry and academic partners. His experience in progressing novel and innovative therapies, including cell and gene therapies, from the bench to the bedside will expedite the development of the first cytoDRiVE-based programs into human clinical trials to our ultimate goal of treating cancer patients with controllable living medicines."

Dr. Ross added, "Obsidian addresses a key unmet need in cell and gene therapy through the ability to regulate the biological activity of engineered cells, allowing the creation of highly effective, titratable and targeted immune-oncology therapies. I look forward to contributing to Obsidian's growth and clinical progress."

Dr. Ross serves as the Chief Medical Officer of Surface Oncology and oversees all clinical and regulatory operations and development efforts. He is responsible for advancing Surface Oncology's programs into the clinic. Rob has extensive clinical development experience, most recently at bluebird bio where he led the clinical development of genetically modified cellular therapies in betathalassemia and sickle cell disease. Rob was also the head of oncology at bluebird bio, building a multifaceted oncology program, led by an anti-BCMA chimeric antigen T cell therapy in collaboration with Celgene. Previously, he worked at Genentech and Infinity Pharmaceuticals on both small molecule and antibody programs from Phase I through pivotal trials, and was a faculty member at the Dana Farber Cancer Center, treating patients with genitourinary malignancies. Rob earned his bachelor's degree from Stanford University, his master's degree from Harvard Medical School and his medical degree from Columbia University Vagelos College of Physicians and Surgeons. Rob did his residency in internal medicine at the University of California, San Francisco and his fellowship in hematology/oncology at the combined program at the Dana Farber/Massachusetts General Hospital.

About Obsidian Therapeutics Obsidian Therapeutics, Inc. is a biotechnology company pioneering controllable cell and gene therapies to deliver transformative outcomes for patients with intractable diseases. Obsidian's proprietary cytoDRiVE platform provides, for the first time, a technology to develop a new generation of cell and gene therapies in which the level and timing of protein activity are fully controlled in a dose-dependent platform comprises a therapeutic protein of interest fused to a drug-responsive domain (DRD). In the absence of the small molecule drug, the DRD-tagged protein is degraded before it becomes active. In contrast, when the small molecule drug is present, the DRD-tagged protein is stabilized and active, permitting precise control of the timing and level of protein expression. The platform can be applied to design controllable intracellular, membrane and secreted proteins for cell and gene therapies as well as other applications. The Company is headquartered in Cambridge, Mass. For more information, please visit http://www.obsidiantx.com.

Media Contact: Maggie Beller Russo Partners, LLC [emailprotected]646-942-5631

SOURCE Obsidian Therapeutics

Home

Read the rest here:
Obsidian Therapeutics Appoints Rob Ross, MD, to Board of Directors - PRNewswire

LEIDEN, the Netherlands, September 03, 2020 / B3C newswire / -- Amarna Therapeutics, a privately held biotechnology company developing the next-generation SV40-based gene delivery vector platform named SVec that promises to transform gene-replacement and immunotherapy across many disease areas, today announced the appointment of Steen Klysner, Ph.D. as its new Chief Executive Officer (CEO) as per September 1. Founder and CEO Ben van Leent will become a member of Amarnas Supervisory Board.

I am very pleased to welcome Steen Klysner as our new CEO. Steen brings an extensive background as a biotech CEO to Swedish ExpreS2ion Biotech Holding AB & the Danish ExpreS2ion Biotechnologies ApS, preceded by an impressive track record in execution and value creation within the biotech industry. His leadership experience makes Steen an ideal candidate to lead Amarna into its next stage of growth and development. said Thomas Eldered, Chairman of Amarnas Supervisory Board. We are extremely grateful for Ben van Leents leadership and contributions to Amarna as both founder and CEO, and we are excited about the opportunity to focus his outstanding expertise as member of our Board.

I am incredibly honored to have been given the opportunity to lead Amarna, said Dr. Klysner. SVec has the potential to enable major medical breakthroughs, so that patients can be actually cured of life-threatening diseases for which, to date, effective treatment have not become available. Together with Amarnas highly qualified and experienced team, Im fully committed to advance the companys groundbreaking technology into the next important clinical development stages.

Steen Klysner comes to Amarna with over 30 years of experience in the life sciences industry. Prior to joining Amarna, Dr. Klysner served as CEO of the Swedish ExpreS2ion Biotech Holding AB in parallel with the Danish ExpreS2ion Biotechnologies ApS. Earlier, he was Senior Vice President (SVP) of preclinical R&D and SVP of Quality of Allergopharma, the Allergy Business Unit of Merck KGaA. He also served as CEO of Nordic Vaccine in Copenhagen, focusing on the development of non-invasive vaccination based on an integrated nanoparticle adjuvant and delivery platform. Prior to that he has also held positions at Pharmexa, Novo Nordisk and ALK.Dr. Klysner holds a Ph.D. from Technical University of Denmark combined with an Industrial Scientist Research Degree from the Danish Academy of Technical Sciences, a M.Sc. degree in Biochemistry from the University of Copenhagen and a B.Sc. in sports from the University of Copenhagen.Finally, Dr. Klysner is author/co-author of numerous patents and scientific publications in (inter)-national peer-reviewed medical journals.

I am deeply grateful to have had the opportunity to build Amarna to where it is today. In my new role as member of the Supervisory Board, my efforts will be towards helping raise the awareness of Amarna and its SVec gene delivery vector platform and using it to help build a robust pipeline, said Ben van Leent. I have full confidence that Steen, a very passionate and talented leader, will provide the leadership and expertise needed to guide Amarna through the next phases of growth. I look forward to working with Steen to drive forward Amarnas product candidates.

Caption: Steen KlysnerFor high resolution please click the image.

About SV40 vectors: A key to the success of gene therapyToday gene therapy enables the development of a next wave of treatments, with potential to not only treat but also to cure a number of major diseases. Key to the success of gene therapy is the efficient delivery of therapeutic genes into target cells, which is an ability that naturally evolved in viruses, rendering them ideally suited for gene delivery.The Simian virus 40 (SV40) strictly replicates in its natural host, macaque monkeys. The virus cannot replicate in humans and doesnt elicit an immune response, which makes it ideal for developing effective gene therapies. However, the clinical use of SV40 vectors has been hampered by production and safety issues. Amarna has solved this, by developing a novel proprietary SuperVero production cell line and the SVec gene delivery platform.Importantly, SVec can be used to efficiently induce immune tolerance to self-antigens driving degenerative, inflammatory and autoimmune human diseases. Amarna aims to develop SVec-based reverse vaccines for major indications such as neurodegenerative and psychiatric diseases (NDPs), atherosclerotic cardiovascular disease (ACD), obesity, diabetes mellitus (DM), arthritis and chronic obstructive pulmonary disease (COPD).

About Amarna TherapeuticsAmarna Therapeutics is a privately held Biotech company founded in 2008. Its head office is located in Leiden (The Netherlands), and its research facility in Seville (Spain). The company has developed a proprietary SuperVero cell line and SVec gene delivery platform for the development of safe and efficient immunotherapies for major indications within the degenerative, inflammatory and autoimmune disease areas.In October 2019, Amarna secured 10 million in new equity to progress its SVec platform towards clinical studies. The financing round was led by Flerie Invest AB, a Swedish investment company, together with existing shareholders and an innovation credit from the Netherlands Enterprise Agency (RVO.nl).

Contacts

Amarna TherapeuticsSteen Klysner, CEOThis email address is being protected from spambots. You need JavaScript enabled to view it.

LifeSpring Life Sciences Communication, AmsterdamLon Melens+31 6 538 16 427This email address is being protected from spambots. You need JavaScript enabled to view it.

Keywords: Humans; Simian virus 40; Netherlands; Genetic Therapy; Immunotherapy; Gene Transfer Techniques; Immune Tolerance

Published by B3C newswire

Visit link:
Dutch Amarna Therapeutics Announces the Appointment of Steen Klysner as Chief Executive Officer - b3c newswire

PHILADELPHIA, PA, Sept. 03, 2020 (GLOBE NEWSWIRE) -- Spirovant Sciences, a gene therapy company developing treatments and cures for genetic lung diseases including cystic fibrosis, todayannounced that its CEO, Joan Lau, PhD, has been named finalist for the Ernst & Young LLP (EY US) Entrepreneur of the Year 2020 Award in the Greater Philadelphia area. The award honors entrepreneurial business leaders whose ambitions deliver innovation, growth and prosperity as they build and sustain successful businesses that transform our world. Award winners will be announced through a special virtual event in early October.

I am sincerely honored to be named a finalist for the EY Entrepreneur of the Year and to represent our terrific company, our talented team, our inspiring patients, and all the innovative entrepreneurs and scientists in this great region, said Lau. The successes and accomplishments of Spirovant, including being acquired twice in 2019, have resulted from the talent, steadfastness and dedication of our rapidly growing team. These truly exceptional individuals power our mission to deliver innovative gene therapy treatments to patients who have no other options. I thank EY for this honor and its support of entrepreneurialism in Greater Philadelphia and throughout the world.

About EY Entrepreneur of the Year

Entrepreneur of The Year is one of the preeminent competitive award programs for entrepreneurs and leaders of high-growth companies. The nominees are evaluated based on six criteria: overcoming adversity; financial performance; societal impact and commitment to building a values-based company; innovation; and talent management. Since its launch, the program has expanded to recognize business leaders in more than 145 cities in over 60 countries around the world.

Founded and produced by Ernst & Young LLP, the Entrepreneur of The Year Awards are nationally sponsored by SAP America and the Kauffman Foundation. In Greater Philadelphia, sponsors also include PNC Bank, DFIN, SolomonEdwards Group, Ballard Spahr LLP, Morgan, Lewis & Bockius LLP, Murray Devine & Company and Pepper Troutman LLP.

About Spirovant Sciences, Inc.Spirovant is a gene therapy company focused on changing the course of cystic fibrosis and other genetic lung diseases. The company's current investigational gene therapy technologies are designed to overcome the historical barriers that have prevented effective genetic treatments for cystic fibrosis. Spirovant is advancing programs for cystic fibrosis with both AAV and lentivirus vectors. Spirovant is a wholly owned subsidiary of Sumitovant Biopharma Ltd., which is itself a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. Spirovant is located inPhiladelphia, PA.More information is available athttps://www.spirovant.com/.

About Sumitovant BiopharmaLtd.Sumitovant is a global biopharmaceutical company with offices inNew York CityandLondon. Sumitovant is a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. Sumitovant is the majority shareholder of Myovant and Urovant, and wholly owns Enzyvant, Spirovant andAltavant. Sumitovant'spipeline is comprised of early- through late-stage investigational medicines across a range of disease areas targeting high unmet need. For further information about Sumitovant please visithttps://www.sumitovant.com/.

About Sumitomo Dainippon Pharma Co., Ltd.Sumitomo Dainippon Pharma is among the top-ten listed pharmaceutical companies inJapan, operating globally in major pharmaceutical markets, includingJapan, the U.S.,Chinaand the European Union. Sumitomo Dainippon Pharma is based on the merger in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 6,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website athttps://www.ds-pharma.com/.

Media ContactJennifer Guinan

Sage Strategic Marketing

610.410.8111

Jennifer@sagestrat.com

Read the original:
Spirovant CEO Joan Lau Named Finalist for EY Entrepreneur of The Year in Greater Philadelphia - GlobeNewswire

Breakthrough in delivery for cell and gene therapy products has led to a wave of M&A activity as big pharma aims not to miss out on the future of medicine

AIM healthcare index at the centre of innovation, has risen 6% YTD compared with the AIM all share, which has declined 7%

finnLife 50 index has also risen 6% in 2020 led by gains in Synairgen (+2,930%), Avacta (+654%), Omega Diagnostics (+322%) and Tiziana Life Sciences (+283%)

London 25 August 2020 Healthcare companies employing and developing cell and gene therapy (C>) are driving the next wave of innovation in the pharmaceutical industry, leading to increased M&A activity as big pharma aims not to miss out on the future of medicine. The AIM healthcare index has been at the centre of this innovation, rising 6% YTD compared with the AIM all share, which has declined 7%.

These are the findings of finnCaps new quarterly Life Sciences sector report, Rude Health.

Rather than just treating a disease and its symptoms, C> can target the underlying cause of a disease, with long-term benefits and curative potential. C> is now being realised on an applicable level, with many products already approved and the FDA expects to approve 10-20 products a year by 2025.

The financials of the sector are reflective of this rapid progress. In 2018, the market value of C> was $536 million - $1.07 billion; but by 2026 it is set to have a valuation of up to$35.4 billion. Given the high proportion of start-ups in the sector, M&A activity is on the rise, as evidenced by the $3 billion Astellas spent in January 2020 to acquire Audentes Therapeutics, specialists in genetics medicines.

In 2014/2015, M&A activity in the sector was $5 billion; by 2018/2019 it had surged 880% to $49 billion. Much of this is driven by big pharma firms not wanting to fall behind their smaller, more versatile competition, as they did with monoclonal antibody technology. Consequently, they have engaged with M&A to speed up and enhance their own R&D efforts.

The report notes that innovators in C> will be well placed to take part in the land grab that will follow as a result of continued advancements in the sector, and highlights now as a good time for investors and pharmaceutical companies to become involved as the sector is rapidly maturing past its high potential research and development stage with an established pipeline of therapies already being developed.

Some of the key reasons why the report considers the C> sector to be an attractive one for investment are:

Pharmas next wave of innovation. C>s can be potentially curative treatment options as they usually target the underlying cause of disease. In the long term, these therapies could become the backbone of treatment regimens, and solutions to various unmet needs.

Deals. Big Pharma had to play catch-up with monoclonal antibody technology and seems determined not to make the same mistake with C>, as reflected in the high deal activity and high deal values seen within this space.

Sector maturation. Advances in the sector mean that the C> sector is beginning to mature beyond the R&D stage and into commercialisation, with some products already approved, and with a very large future pipeline of therapies.

Revenue.Therapies in this space can command high prices, allowing for high revenue generation, even from rare diseases and limited patient populations.

Despite its vital role in the future of medicine, C> also comes with challenges. The report highlights that the manufacture of C>s is difficult given they are, by definition, personalised for the patient. This means they cannot be batch produced for distribution to multiple patients as more traditional medicines can. For example, Zolgensma, which treats those with motor neurone disease, is priced at $2.1 (1.6) million per therapy, making it the most expensive drug treatment ever.

The report also notes how the payment process for C> requires a reworking of reimbursement systems not used to outlaying so much money up front for a treatment with long-term benefits/curative potential versus continuous, and lower payments for ongoing medicine treatment.

The technologies the report shines a spotlight include CAR-T therapy, stem cell therapy, CRISPR, RNA therapies, among various others.

Arshad Ahad, Research Analyst, Life Sciences, at finnCap, commented:Few technologies in the life sciences sector hold as much promise as Cell and Gene Therapy, with its ability to provide long-term benefits and curative potential. These technologies have been seen as the future of medicine for many years, and now we are closer than ever to that future becoming a reality. If Cell and Gene therapy does become the backbone of treatment regimes in the future, similar to the rise of monoclonal antibodies, then the companies involved are developing expertise in a critical part of the life sciences industry, which should confer a significant competitive advantage as the sector matures further. Now is therefore a good time to invest in the future.

See more here:
FINNCAP'S LIFE SCIENCES REPORT INDICATES CELL AND GENE THERAPY SECTOR IS DRIVING THE NEXT WAVE OF INNOVATION IN PHARMA - PharmiWeb.com

The broker said the market value of the sector is expected to increase to up to US$35.4bn by 2026 from its 2018 value of between US$536mln-US$1.07bn

FinnCap Group PLC () has said healthcare companies employing and developing cell and gene therapy (C>) are driving the next wave of innovation in the pharmaceutical industry, which in turn is causing an uptick in mergers & acquisitions as pharma giants aim to be at the forefront of the next development in medicine.

In a report released on Tuesday, the brokerage firm highlighted that the trend was personified in the AIM healthcare index, which has risen 6% in the year to date as opposed to a 7% decline for the AIM All-Share index.

Small caps involved in the sector that have seen rapid share price rises this year such as (), () and (), have also led gains in finnCaps own finnLife 50 index.

The group also reported that the market value of the C> sector is expected to increase to up to US$35.4bn by 2026 from its 2018 value of between US$536mln-US$1.07bn.

M&A is also on the rise given the high number of startups in the sector, finnCap said, with acquisition activity valued at US$49bn in 2018/19 compared to US$5bn in 2014/15.

Much of this is driven by big pharma firms not wanting to fall behind their smaller, more versatile competition, as they did with monoclonal antibody technology. Consequently, they have engaged with M&A to speed up and enhance their own [research & development] efforts, the broker said.

Rather than just treating a disease and its symptoms, C> can target the underlying cause of a disease, with long-term benefits and curative potential. C> is now being realised on an applicable level, with many products already approved and the [US Food & Drug Administration] expects to approve 10-20 products a year by 2025, they added.

As a result, the report said that now was the time for investors to get involved as the C> sector is rapidly maturing past its high potential research and development stage with an established pipeline of therapies already being developed.

However, finnCap cautioned that there were challenges facing the sector including difficulty in manufacturing the therapies due to their personalised nature as well as a potential reworking of reimbursement systems that are not used to outlaying so much money up front for a treatment with long-term benefits/curative potential versus continuous, and lower payments for ongoing medicine treatment.

Few technologies in the life sciences sector hold as much promise as Cell and Gene Therapy, with its ability to provide long-term benefits and curative potential, said finnCap life sciences analyst Arshad Ahad.

These technologies have been seen as the future of medicine for many years, and now we are closer than ever to that future becoming a reality. If Cell and Gene therapy does become the backbone of treatment regimes in the future, similar to the rise of monoclonal antibodies, then the companies involved are developing expertise in a critical part of the life sciences industry, which should confer a significant competitive advantage as the sector matures further. Now is therefore a good time to invest in the future, the research analyst added.

FinnCap shares were steady at 23p in late-morning trading on Wednesday.

See the rest here:
FinnCap touts cell and gene therapy as next wave of innovation in pharma industry - Proactive Investors UK

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apic Bio Inc., a gene therapy company developing novel treatment options for patients with rare genetic diseases, today announced the appointment of Jorge A. Quiroz, MD, MBA, as Executive Vice President, Chief Medical Officer. Dr. Quiroz brings over 20 years of scientific, clinical, and regulatory experience helping to develop and evolve gene therapies and small molecule drug programs from preclinical to regulatory filing. Most recently, Dr. Quiroz served as Chief Medical Officer of Solid Biosciences where he was responsible for leading the clinical advancement of its gene therapy program.

We are excited to welcome Jorge to Apic during this critical period for the company and our pipeline. His extensive expertise in the clinical and regulatory development of gene therapies for rare diseases will support the advancement of both our SOD1 amyotrophic lateral sclerosis (ALS) and alpha-1 antitrypsin deficiency (Alpha-1) programs, said John Reilly, MS, MBA, Co-founder and Chief Executive Officer of Apic. As CMO, Jorge will also lead the development and build out of our early-stage gene therapy programs derived from our THRIVE platform. We look forward to working with Jorge during this next chapter for Apic as we rapidly advance our therapies into the clinic on behalf of patients and families in need.

I am delighted to join Apic during an exciting period of expansion as we prepare to submit an Investigational New Drug (IND) application for APB-102 for the treatment of patients with SOD1 ALS this year and enter the IND-enabling stage for APB-101 for the treatment of patients with alpha-1 antitrypsin deficiency, said Dr. Jorge Quiroz, EVP and Chief Medical Officer of Apic. The Companys mission, deep scientific foundation, clinical approach, and manufacturing expertise puts us in an excellent position to bring new gene therapy treatments to patients living with rare and monogenic disorders.

Dr. Quiroz previously served as the Head of Neurodevelopment & Psychiatry, Translational Medicine Neurosciences at F. Hoffmann-La Roche AG. He has also served as a Director at Johnson & Johnson Pharmaceutical Research & Development, LLC. He received an MD from the Pontifical Catholic University of Chile and completed his medical training as a Research Fellow at the Laboratory of Molecular Pathophysiology, at the National Institute of Mental Health. Dr. Quiroz is board certified in Psychiatry and also holds an MBA dual degree from Columbia University and London Business School.

About Apic Bio

Apic Bio is a gene therapy company focused on developing novel treatment options for rare, undertreated neurological and liver diseases. The Company's lead program is an adeno-associated (AAV)-based gene therapy for the treatment of the copper zinc superoxide dismutase 1 (SOD1) ALS, a genetic form of the disease. Preclinical studies of additional genetic forms of ALS (C9Orf72) and alpha-1 antitrypsin deficiency (Alpha-1) are ongoing. The Company is also advancing discovery programs for two undisclosed CNS indications that leverage its proprietary silence and replace THRIVE platform. The Company is backed by leading and disease-centric investors, including Morningside Ventures, ALS Investment Fund, and The Alpha-1 Project (TAP). For more information please visit http://www.apic-bio.com.

More:
Apic Bio Announces Appointment of Jorge A. Quiroz, MD, MBA, as Executive Vice President and Chief Medical Officer - Business Wire

The addition of cryogenic freezer systems firm MVE Biological Solutions will boost Cryoports position in the temperature-controlled cell and gene therapy logistics space.

Cryoport, a temperature-controlled supply chain services firm for the life sciences, has agree to buy MVE Biological Systems for $320 million with the deal expected to close by the end of the year.

MVE is a subsidiary of Chart Industries and will bring Cryoport a greater presence in the cell and gene therapy services space, broadening its portfolio to include manufactured vacuum insulated products and cryogenic freezer systems. The firm has three manufacturing sites two in the US and one in China and more than 300 customers, including Cryoport itself.

Image: iStock/Zhanna Hapanovich

According to Jefferies analyst Brandon Couillard, MVE has an estimated 55% share of the vacuum insulated products and cryogenic equipment market and the acquisition will bolster Cryoports strategy of becoming the top life sciences supply chain solutions provider, further augmenting its exposure to the rapidly growing cell & gene therapy space.

The deal comes days after Cryoport announced it is buying French logistics firm CRYOPDP for 49 million ($58 million).

Cryoport has fundamentally transformed the company via the CRYOPDP and MVE deals, Couillard said in a note, adding the acquisition spree solidifies Cryoports status as one of the best ways to play the coming cell and gene therapy boom.

Speaking on the deal, Jerrell Shelton, CEO of Cryoport, said: The acquisition of MVE Biological Solutions represents an important step in carrying out Cryoports mission. It further entrenches us in the cell and gene therapy supply chain ecosystem at a time when cell and gene therapies are experiencing rapid and sustained growth, and with an even more exciting growth story ahead.

He added: Bringing MVE Biological Solutions under the Cryoport umbrella, which will include Cryoport Systems, Cryogene, and the recently announced agreement to acquire CRYOPDP is expected to increase our revenue run rate to over$160 millionand to be immediately accretive.

Read the original post:
Cryoport strikes second acquisition in a week - Bioprocess Insider - BioProcess Insider

KUALA LUMPUR, Malaysia, Aug. 26, 2020 /PRNewswire/ --Sunway University's Professor Jeff Tan Kuan Onn of the Department of Biological Sciences and Professor Poh Chit Laa from the Centre for Virus and Vaccine Research, along with their research collaborators from Harvard Medical School's Center for Stem Cell Therapeutics and Imaging (USA) as well as University of Tennessee Health Science Centre (USA) have completed a study that has demonstrated the efficacy of molecular gene therapy as a new strategy for cancer treatment.

Professor Jeff Tan Kuan Onn

The research could potentially contribute to shorter treatment time for cancers, reduce treatment costs and minimise the adverse effects of current chemo-drugs in cancer patients such as susceptibilities toward microbial infections, hair-loss and other side effects of chemo-drugs that drastically affect the quality of life of cancer patients undergoing therapy.

Principal Investigator Professor Jeff Tan explained, "Currently, chemo-drugs are relatively ineffective against cancer cells that have developed drug-resistance resulting in the need for high doses of chemo-drugs or a combination of chemo-drugs to be administered to patients with cancer cells. Chemo-drug resistant cancer cells also can spread quickly and that drastically reduce the survival rate of cancer patients".

"Our research utilises molecular gene therapy which is the introduction of genetic materials into cancer cells to promote the sensitivity of cancer cells to chemo-drugs. By genetically engineering the cancer cells, we find that we can induce the cancer cells to produce activated pro-death and tumour suppressor proteins that cause cell death and growth arrests in cancer cells. The weakened cancer cells can then be killed relatively easily by the administration of chemo-drugs in smaller doses. Ultimately, the research could contribute to increasing the survival rates of cancer patients undergoing cancer treatments," he added.

Co-Investigator Professor Poh Chit Laa said that the effectiveness of the strategy has been demonstrated in mice implanted with human breast cancer cells. "In the mice that weretreated with the gene therapy, the tumours obtained from the treated mice showed significant tumour cell death and the tumours were 20 times smaller and 32 times lighter in volume and weight, respectively, when compared to the tumours obtained from the untreated mice. The results indicated that the gene therapy was able to shrink the tumours significantly, even without treatment with chemo-drugs. Small doses of market-available anti-cancer drugs could then be used to kill the cancer cells effectively. We hope to see our research contribute to better survival rates of cancer patients, and minimise the side-affects associated with anti-cancer drugs," said Professor Poh.

"We are currently working on investigations to optimise the delivery of the gene therapy and anti-cancer drugs to human tumours with hopes that this will result in tangible clinical outcomes," said Professor Jeff Tan.

The research project was recently published in the peer-review Journal of Cancer Research and Clinical Oncology. Collaborators for the research include Lee Yong Hoi, Pang Siew Wai and Samson Eugin Simon from the Department of Biological Sciences, Sunway University; Esther Revai Lechtich and Khalid Shah, of the Center for Stem Cell Therapeutics and Imaging, Brigham and Women's Hospital, Harvard Medical School (USA); Suriyan Ponnusamy and Ramesh Narayanan from the Department of Medicine, Centre of Cancer Drug Discovery, College of Medicine, University of Tennessee Health Science Centre (USA).

Story continues

The research is a result of a collaboration agreement between Harvard Medical School and Sunway University aimed at developing new cancer therapies targeting drug resistant cancer cells. In 2016, Professor Jeff Tan visited Harvard University on the Jeffrey Cheah Travel Grant which enabled him to better understand how cancer research projects are conducted as well as examining experimental models used to study cancer biology at Harvard University, Massachusetts General Hospital (MGH), a hospital affiliated with Harvard Medical School, and the Dana-Farber Cancer Institute.

To read the jointly published article: https://link.springer.com/article/10.1007/s00432-020-03231-9

Photo - https://photos.prnasia.com/prnh/20200825/2898392-1

SOURCE Sunway University

Visit link:
Sunway University's Collaboration with Harvard Medical School Makes Advances in Potential Cancer Treatment using Gene Therapy - Yahoo Finance