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Archive for the ‘Gene therapy’ Category

Orchard Therapeutics’ gene therapy Strimvelis linked to a leukemia case – FiercePharma

Tuesday, November 3rd, 2020

The potential of gene therapies as cures for some hard-to-treat genetic diseases can be very attractive. But one such product is now suspected of causing a serious safety problem.

Friday, Orchard Therapeutics said its Strimvelis treatment, approved by European authorities in 2016 to treatthe rare inherited condition ADA-SCID, has been linked to a patients leukemia.

Preliminary findings suggest this diagnosis may be attributable to an insertional event related to treatment with Strimvelis, the company said. Its now investigating whether theres indeed a causal relationship.

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Since its 2016 EU approvalwhen it was owned by original developer GlaxoSmithKlineonly 16 patients have been treated with Stimvelis. The patient who developed leukemia had apparently been treated under a GSK compassionate use program in 2016.

No more patients will get the therapy before the investigation is complete, Orchard said. The drug was never approved in the U.S.

ADA-SCIDis a condition marked by a mutation in the gene making the adenosine deaminase (ADA) enzyme, which is essential for maintaining normal white blood cells. ADA-SCID patients, with a dysfunctional immune system, have less than two years to live without effective intervention.

RELATED:Orchard licenses gene therapy tech from GSK

Strimvelis, originally developed by GSK and bought by Orchard in 2018, offers an option for patients who cant find a matched stem cell donor. It works by editing the patients own hematopoietic stem cells with the functional ADA gene. The cells arethen transferred back into the patient's bone marrow to mature and produce the normal ADA protein.

The therapy uses a gammaretrovirus as the vector to carry the gene. Problem is, retrovirus can incorporate their own genetic information into the human genome, causing unintended changes that can give rise to cancer. It is a known risk factor of gammaretroviral vector-based gene therapy and has been described as one of the important potential risks for Strimvelis in its EU approval.

Besides Strimvelis, Orchard is also developing OTL-101, which uses a lentivirus to insert a functional copy of the ADA gene into a patients cells. The drug is currently undergoing a registrational trial and has won breakthrough and orphan drug designations from the FDA.

All the gene therapy candidates in Orchards pipeline use lentiviral vectors that have been specifically designed to avoid insertional oncogenesis after administration, Orchard said, adding that no dangerous gene insertion has been reported around lentiviral vector-based stem cell gene therapy in any indication.

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Gene therapy may be able to restore vision – Genetic Literacy Project

Tuesday, November 3rd, 2020

In a normal eye, opsins are expressed by the rod and cone photoreceptors in the retina. When activated by light, the photoreceptors pulse and send a signal through other retinal neurons, the optic nerve, and on to neurons in the brain.

A variety of common eye diseases, including age-related macular degeneration and retinitis pigmentosa, damage the photoreceptors, impairing vision. But while the photoreceptors may no longer fully function, other retinal neurons (bipolar cells) remain intact. The team identified a way for bipolar cells to take on some of the work of damaged photoreceptors.

The beauty of our strategy is its simplicity, said Samarendra Mohanty, PhD, Nanoscope founder and corresponding author of a report on the mouse study that appears today in Nature Gene Therapy. Bipolar cells are downstream from the photoreceptors, so when the MCO1 opsin gene is added to bipolar cells in a retina with nonfunctioning photoreceptors, light sensitivity is restored.

The strategy could overcome challenges plagued by other approaches to retinal regeneration, according to the researchers. Gene replacement therapy has thus far worked principally in rare diseases that leave photoreceptors intact.

Under a best-case scenario, the therapy could help patients achieve 20/60 vision, according to the researchers; however, no one knows how the restored vision will compare to normal vision.

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Avantor Discusses Cell & Gene Therapy Production Trends Through the Lens of COVID-19 – PRNewswire

Tuesday, November 3rd, 2020

Avantor executives joined other industry leaders at virtual Cell & Gene Therapy Bioprocessing & Commercialization Conference

Panels discussed critical topics including process efficiency and scaling solutions

RADNOR, Pa., Nov. 2, 2020 /PRNewswire/ --Executives from Avantor Inc. (NYSE: AVTR), a leading global provider of mission-critical products and services to customers in the life sciences, advanced technologies and applied materials industries, recently provided expert insight at the Cell & Gene Therapy Bioprocessing & Commercialization Virtual Conference. Cell and gene therapy (C>) are two of the most revolutionary applications driving the biopharmaceutical industry.

In separate panel discussions with other global industry leaders, Dr. Ger Brophy, EVP, Biopharma Production at Avantor and Claudia Berrn, SVP, Business Development and Commercial Operations at Avantor, each addressed novel considerations and challenges facing the biopharmaceutical industry, including the impact of COVID-19. They highlighted innovation to help drive the creation and commercialization of life-changing, personalized C> treatments.

In a panel on the evolution of C> and the path toward scalability and manufacturability of these treatments, Dr. Brophy gave his perspective on this growing need for the bioprocessing industry.

"Genuine progress is being made in the long-standing battle to effectively treat and control disease, and cell & gene therapies will only continue to unlock new frontiers in medicine," said Dr. Brophy. "We're beginning to see more clearly the issues that need to be addressed, and know that if products are to scale and become more accessible to patients worldwide, there needs to be efficiency in operations. There is an absolute requirement for automation, both to reduce variability and to generate process efficiencies. At Avantor, we are ready to make the essential materials and technologies available to companies leading the charge and treating patients. Innovation and agility are central to how we're partnering with the industry to resolve these issues."

On a separate panel, Ms. Berrn highlighted how the industry is navigating the challenges and impact of the global pandemic, from the fragmentation of supply chains to clinical trial disruption.

"In this critical moment for the industry and the world, it is more important than ever for leaders in the cell & gene therapy community to come together to strategize and share ideas," said Ms. Berrn. "Avantor is actively working with the world's leading pharmaceutical and biotechnology companies to accelerate the production of novel treatments. Collectively, our goal is to mitigate any challenges in the process of taking a groundbreaking treatment from its initial scientific discovery to delivery in treating patients."

Avantor provides products for biopharma production workflows, including cell and gene therapy offering. Search 'Avantor biopharma' in your browser.

About AvantorAvantor, a Fortune 500 company, is a leading global provider of mission-critical products and services to customers in the biopharma, healthcare, education & government, and advanced technologies & applied materials industries. Our portfolio is used in virtually every stage of the most important research, development and production activities in the industries we serve. One of our greatest strengths comes from having a global infrastructure that is strategically located to support the needs of our customers. Our global footprint enables us to serve more than 225,000 customer locations and gives us extensive access to research laboratories and scientists in more than 180 countries.We set science in motion to create a better world. For information, visit avantorsciences.com and find us on LinkedIn, Twitter and Facebook.

Robert DonohoeSenior Director, Corporate CommunicationsAvantorM: 484-688-4730[emailprotected]

Source: Avantor and Financial News

SOURCE Avantor and Financial News

http://avantorsciences.com

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Euro Convergence: Early consultation critical in overcoming gene therapy hurdles – Regulatory Focus

Tuesday, November 3rd, 2020

When it comes to advancing gene therapy products in the United States and Europe, the key is early consultation with regulators and identification of potential problems, experts said at the Euro Convergence 2020 virtual meeting.

It is better to do it earlier and establish a collaborative and educational approach to discussions, advised Mridula Shukla, director of global regulatory affairs at Arcutis Biotherapeutics in Palo Alto, Calif. Patrick Celis, PhD, head of the scientific secretariat of the Committee for Advanced Therapies at the European Medicines Agency (EMA), echoed that advice.

Please talk to EMA and National Authorities early and frequently so that we can help you as much as possible to get these innovative products to the patients as soon as possible, Celis said.

There are currently more than 10 gene therapy products approved globally and momentum has been building in this area since around 2015, Shukla said.

Gene therapy seems to have potential as it offers the possibility of an actual cure instead of chronic treatment for rare diseases, hereditary diseases and cancer, Shukla said.

Despite the promise of the therapies, more than half of gene therapy clinical trials are in phase 1. Shukla said this is due to a combination of factors including the complexity of the therapies, the rare indications, the high price of the therapies and reimbursement issues, unique manufacturing and supply chain challenges and controversies surrounding the ethnics of the techniques.

There is guidance from the US Food and Drug Administration (FDA) to assist developers. In July 2018, FDA issued draft guidance on Human Gene Therapy for Rare Diseases, which was finalized in January 2020. The document offers advice on some common manufacturing challenges, including encouraging companies to establish critical process parameters (CPP) early on and establish and qualify a potency assay prior to conducting clinical trials.

The FDA recommends communication with its Office of Tissues and Advanced Therapies early in product development, even before submission of an investigational new drug (IND) application. The agency also offers an Initial Targeted Engagement for Regulatory Advice on CBER Products (INTERACT) meeting, which can be used to discuss specific product issues, Shukla said.

Overall, the recommended approach is to front-load as much development as possible, preferably prior to Phase 1, and to hold early interactions and frequent interactions with FDA or EMA, Shukla said.

In the US, there are also multiple options for expedited review of products, including:

These expedited processes are similar to programs available for advanced therapy medicinal products (ATMPs) in Europe, such as the priority medicines (PRIME) scheme, which offers early consultation and the potential for accelerated assessment.

Since 2009, the EMA has approved 17 ATMPs, of which 10 are gene therapy products, Celis said.

The common issues that come up with ATMP applications include a lack of experience with commercial manufacturing processes, finding a relevant potency assay, ensuring product consistency and challenges with consistent starting materials, according to Celis. Other problems that can lead to delays in authorization include a lack of randomization in trial design, an indication that doesnt reflect the patients in the trial, and limited data on safety and durability of response.

The nature of gene therapies and the small number of patients in clinical trials mean that sponsors often have difficulty showing long-term safety and efficacy in a robust patient population. While regulators dont expect the same level of data as with other traditional medicines, there is a need for early planning of post-authorization studies, Celis said.

The EMA is asking for a detailed post-authorization plan that can include follow-up of patients in the clinical trials, post-marketing trials, and post-authorization registry-based studies.

If a sponsor is using a registry, Celis said it is better to go with an established disease registry and to contact the registry early to find out what data is collected. Additionally, registries may not be a good basis for safety reporting, Celis said.

There are support mechanisms in place for ATMP developers including scientific advice, a meeting with the EMAs Innovation Task Force, and the PRIME program for medicines addressing unmet needs.

We can help, Celis said. Make sure that you make best use of what we can do.

For sponsors looking to market gene therapies globally, Celis said that the EMA and FDA are in frequent contact and are seeing many of the same issues as they review products. He encouraged developers to make use of this interaction between European and US regulators either formally, through parallel scientific advice, or informally.

Euro Convergence 2020

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ELEVECTA for AAV gene therapy vector production – Bioprocess Insider – BioProcess Insider

Tuesday, November 3rd, 2020

Pharmaceutical companies with gene therapies in their pipeline need to establish scalable production processes that can keep up with the increasing demand in terms of volume, quality and robustness, says CEVEC.

Gene therapies are seen as one of the most promising approach to treat life-threatening diseases with no therapeutic options and high medical needs. In addition, gene therapies are now advancing also into indications with larger numbers of patients, including Alzheimers and Parkinsons, thereby dramatically increasing the demand for viral vectors. As a result, pharmaceutical companies with gene therapies in their pipeline need to establish scalable production processes that can keep up with the increasing demand in terms of volume, quality and robustness.

CEVEC Pharmaceuticals, a German biotechnology company focusing on viral vector technologies, has developed and commercially launched ELEVECTA, a unique technology that enables fully scalable AAV manufacturing providing unprecedented process robustness and consistency in vector yield and quality.

One of the major bottlenecks in AAV vector production is volumes. Standard technologies have proven to be very hard or impossible to scale, as they are often based on adherent cell lines or require transient transfections or helper viruses.

While suspension cell lines are getting more and more common now, most processes still rely on transient transfection to bring the different AAV genes into the producer cells. The typical transient transfection process is complex and requires three different GMP-grade plasmids driving up the production costs. This is where ELEVECTA stableAAV producer cell lines come into play.

Figure 1: ELEVECTA, a revolutionary technology that enables fully scalable, stable viral vector manufacturing with unprecedented process robustness and consistency in vector yield and quality.

Based on a stable human suspension cell line, ELEVECTA enables full scalability in all types and formats of suspension bioreactors, from laboratory to commercial scale. ELEVECTA Producer Cell Lines reach titers of 1*1014 vg/l and more during a typical single-use suspension bioreactor run. CEVEC has now successfully scaled-up from 15 ml to 50 L bioreactors in a single step without any productivity loss, demonstrating the scalability and robustness of the process.

ELEVECTA Producer Cells contain all components required for AAV stably integrated into the cell. There is no need for cGMP plasmids, special transfection reagents and difficult-to-scale-up transfection protocols. Just one cell line which, after propagation and expansion to the desired cell titers, produces AAV vectors at high yields a process that resembles well-established production methods for recombinant proteins and monoclonal antibodies.

Figure 2: Cryo TEM picture from AAV particles derived from ELEVECTA Producer Cell Lines. The viral particles display an approximate size of 22 nm. Full particles are indicated by a black arrow, empty particles by a white arrow

AAV vectors can have different surface structures, so called serotypes, which in nature allow the virus to target specific cells in a body. For gene therapy applications viral serotypes can further be engineered to improve their tissue-specificity and safety. Every ELEVECTA producer cell line is custom-made and employs a specific natural or engineered capsid gene and the therapeutic gene of interest.

With this newly introduced technology, CEVEC is the leading technology provider for stable AAV producer cell lines. Its inherent scalability, process robustness and consistency in yield and quality provide the features that are needed to pave the way for the success of modern gene therapies in a broad range of indications.

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Novartis Expands Presence in Gene Therapy With Acquisition – Yahoo Finance

Tuesday, November 3rd, 2020

- By Barry Cohen

Novartis AG (NYSE:NVS) is expanding its gene therapy footprint by acquiring a venture capital-backed company focusing on treating vision loss that can lead to blindness.

The Swiss pharmaceutical giant will pay shareholders of Vedere Bio $150 million upfront, which could be boosted by $130 million more if the Cambridge, Massachusetts-based company's programs achieve several development milestones.

Novartis has made huge strides aimed at beefing up its gene therapy program. In 2018, the company bought AveXis for $8.7 billion, inheriting its spinal muscular atrophy therapy Zolgensma, and this year established partnerships with Dyno Therapeutics and Sangamo Therapeutics Inc. (NASDAQ:SGMO).

Vedere is trying to take naturally occurring, light-sensing proteins and use a special gene therapy method to inject them into a certain part of the eye. Once there, they will be sent to retinal cells that are still healthy to help improve the vision of patients, according to an article in BioPharma Dive.

Novartis thinks this approach has much broader uses. The company said the proteins, in conjunction with Vedere's delivery tools, could "vastly expand" the number of treatable patients with vision loss from photoreceptor death.

Investors should keep in mind that the Vedere treatments are still in the pre-clinical stage so a great deal of development still needs to be done. Working with the acquisition's scientists, Novartis is preparing to promptly begin human testing.

There could be an even bigger population the Vedere platform could address, beyond inherited eye disorders. That would be a new way to treat geographic atrophy, one of the more advanced forms of an age-related vision loss called dry age-related macular degeneration. The American Academy of Ophthalmology notes an estimated 15 million people in North America have AMD, the vast majority with the dry form.

Story continues

Following the close of the deal, Vedere Bio II will operate as a wholly independent entity from Novartis and Vedere Bio.

Novartis decided to get out of one area of the eye-care business when it spun off Alcon Inc. (NYSE:ALC) last year. The company said it wanted to concentrate on treating diseases of the eye, not eye-care products.

Novartis' eye drugs include Xidra, treating dry eye disease, and Beovu, which was recently approved for the wet form of AMD. Its third eye drug and the biggest producer is Lucentis, which had sales of $515 million in the third quarter. Xidra and Beovu combined to bring in revenue of about $151 million. Novartis' biggest competitor is the Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) drug Eylea, a multibillion-dollar annual seller.

The Novartis faithful are hoping for better days ahead. At about $90, the company's shares are pretty much where they were five years ago. On the plus side, its dividend yields nearly 4%.

Despite the stock being stuck in neutral, Wall Street seems to think Novartis is a good investment, with three recent buy ratings and just one hold. Analysts give it an average target price of more than $106, according to TipRanks

UBS analyst Laura Sutcliffe last month upgraded the stock to buy from hold, calling its valuation "compelling." She thinks new products in the pipeline will boost the company's annual earnings growth to a healthy 9%.

Disclosure: The author has no positions in any of the stocks mentioned in this article.

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Retinal Disorders Treatment Market: Advancements in Gene-therapy and Stem-cell Therapy to Bolster Market Growth – BioSpace

Tuesday, November 3rd, 2020

Retinal Disorders Treatment Market: Introduction

According to the report, the global retinal disorders treatment market was valued at US$ 9.18 Bn in 2019 and is projected to expand at a CAGR of ~7% from 2020 to 2030. Macular degeneration is of two types: wet age-related macular degeneration and dry age-related macular degeneration. Diabetic retinopathy is one of the common diabetic eye disorders characterized by damaged blood vessels in the retina. Damaged blood vessels and nerves lead to vision impairment, blurring of vision, and eye hemorrhage. If left untreated, it could lead to retinal detachment and blindness. In terms of indication, the global retinal disorders treatment market has been classified into macular degeneration, diabetic retinopathy, diabetic macular edema, and others. The macular degeneration segment has been bifurcated into dry macular degeneration and wet macular degeneration. Based on therapeutic class, the global retinal disorders treatment market has been categorized into anti-VEGF agents and others. In terms of dosage form, the global retinal disorders treatment market has been divided into gels, eye solutions, capsules & tablets, eye drops, and ointments.

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Based on distribution channel, the global retinal disorders treatment market has been segregated into hospital pharmacies, retail pharmacies, and online sales. Rise in prevalence of retinal disorders due to increase in geriatric patient population boosts the growth of the global retinal disorders treatment market. The U.S. dominated the global retinal disorders treatment market in 2019, and the trend is anticipated to continue during the forecast period. Well-established healthcare infrastructure and early adoption of advanced technologies are the factors expected to fuel the growth of the market in the region. Moreover, rise in prevalence of various types of retinal disorder leads to increase in demand for treatment.

China is likely to be a highly lucrative market for retinal disorders treatment during the forecast period. Diagnosis and treatment rates have increased due to a rise in disposable income and health awareness. This has led to an increase in the adoption of macular degeneration drugs

Rise in Prevalence of Retinal Disorders Due to Increase in Geriatric Patient Population to Drive Global Market

Age is a prominent risk factor for age-related macular degeneration. The risk of developing advanced age-related macular degeneration increases from 2% in people aged between 50 and 59 to nearly 30% for those over 75. The prevalence of other retinal disorders was 93 million people with diabetic retinopathy, 21 million people diabetic macular edema and 28 million people with vision-threatening diabetic retinopathy. Increase in R&D activities, rise in the number of patients suffering from diseases, and rapid expansion of healthcare and biopharmaceutical industries in developed and developing countries are projected to boost advancements in therapies in the AMD treatment market during the forecast period. For instance, Lucentis and Eylea accounted for 2.8% of total pharmaceutical sales in Canada in 2017.

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Macular Degeneration to Dominate Global Market

In terms of indication, the global retinal disorders treatment market has been divided into macular degeneration, diabetic retinopathy, diabetic macular edema, and others. Macular degeneration has been bifurcated into dry macular degeneration and wet macular degeneration. The macular degeneration segment dominated the market in terms of revenue in 2019. The rise in prevalence of macular degeneration is anticipated to drive the segment during the forecast period. For instance, the number of people living with macular degeneration is expected to reach 196 million globally by 2020 and increase to 288 million by 2040.

Anti-VEGF Agents to be Main Therapeutic Class

Based on therapeutic class, the global retinal disorders treatment market has been categorized into anti-VEGF agents and others. The anti-VEGF agents dominated the global retinal disorders treatment market in 2019. Major market products such as Avastin and Eylea are included in the anti-VEGF drug class. Increase in demand for these products in the treatment of retinal disorders and strong product pipeline are likely to drive the segment. However, the others segment, which includes anti-inflammatory drugs, is projected to expand at the highest CAGR from 2020 to 2030. The increase in the use of anti-inflammatory drugs for pain relief is anticipated to augment the segment.

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Eye Solutions to be Preferred Dosage Form

In terms of dosage form, the global retinal disorders treatment market has been divided into gels, eye solutions, capsules & tablets, eye drops, and ointments. The eye solutions segment dominated the global retinal disorders treatment market in 2019. However, the eye drops segment is expected to expand at the highest CAGR during the forecast period. The segment is likely to grow at a rapid pace due to increase in demand for eye drops for treatment of retinal diseases in emerging countries such have China, India, and Brazil.

Retail Pharmacies to Emerge as Major Distribution Channel

Based on distribution channel, the global retinal disorders treatment market has been segregated into hospital pharmacies, retail pharmacies, and online sales. The retail pharmacies segment dominated the market in terms of revenue in 2019 due to wide network, ease of access, and diverse product offerings, including prescription and OTC ophthalmic drugs. However, the shift toward the use of electronic payment modes is projected to boost the growth of the online sale segment during the forecast period.

U.S. to Dominate Global Market

The global retinal disorders treatment market has been segmented into five major regions/country: the U.S., Europe, China, Russia, and Rest of the World. The U.S. dominated the global market in 2019, followed by Europe. The U.S. accounted for a major share of the global retinal disorders treatment market in 2019. Well-developed healthcare infrastructure, high healthcare expenditure, and adoption of branded drugs to treat retinal disorder disorders are the key factors attributed to the countrys significant share of the global market.

The retinal disorders treatment market in China is anticipated to expand at a high CAGR from 2020 to 2030. There have been significant unmet medical needs in the region. Furthermore, healthcare expenditure is increasing in developing markets. Key players are making investments to establish their operations in China. This, in turn, is projected to augment the market in the country.

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Competition Landscape

Regeneron Pharmaceuticals, Inc., F. Hoffmann-La Roche Ltd., and Novartis AG are the three major companies operating in the global retinal disorders treatment market. The global retinal disorders treatment market is fragmented in terms of number of players. Key players in the global market include Allergan plc, Bayer AG, F. Hoffmann-La Roche Ltd., Graybug Vision, Inc., Kubota Pharmaceutical Holdings Co., Ltd., Novartis AG, Pfizer, Inc., Regeneron Pharmaceuticals, Inc., Santen Pharmaceutical Co., Ltd., and Takeda Pharmaceutical Company Limited. New product development through robust R&D activities and mergers & acquisitions are key strategies adopted by these players to gain a competitive advantage in the global retinal disorders treatment market.

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Decibel Therapeutics Announces Exclusive Licensing Agreements for Hearing Loss Gene Therapy Technology | DNA RNA and Cells | News Channels -…

Tuesday, November 3rd, 2020

DetailsCategory: DNA RNA and CellsPublished on Monday, 02 November 2020 14:44Hits: 321

BOSTON, MA, USA I November 02, 2020 IDecibel Therapeutics, a clinical-stage biotechnology company developing novel restorative gene therapeutics to treat hearing loss and balance disorders, today announced exclusive license agreements with the University of Florida (UF) and the University of California, San Francisco (UCSF) for an adeno-associated virus (AAV) gene therapy technology designed to restore hearing to individuals with profound, congenital hearing loss caused by mutations in the otoferlin gene.

Otoferlin is a protein present in the inner hair cells of the cochlea that is critical for the communication between sensory cells of the inner ear and the auditory nerve by regulating release of neurotransmitters. People born with biallelic mutations in the otoferlin gene have profound hearing loss because this signal between the ear and the brain is lost. Decibel aims to restore functional otoferlin using gene therapy. A principal challenge is the size of the otoferlin gene, which is too large for the packaging capacity of AAV vectors. To overcome this challenge, Dr. William Hauswirth (UF), Dr. Omar Akil (UCSF), and collaborators employed a dual-vector approach to deliver the gene in two separate AAV vectors. This approach resulted in expression of the complete otoferlin gene, restored the signaling connection between the ear and the brain, and rescued normal hearing in a deaf, otoferlin-deficient mouse model.1

The dual-AAV gene therapy approach has shown significant promise in preclinical research as a method to deliver a large gene to the ear, said William Hauswirth, Ph.D., Professor of Ophthalmology at University of Florida. We were able to cure deafness in a mouse model and look forward to the potential of this technology in the development of a gene therapy to restore hearing in human patients.

The AAV-mediated gene therapy technology is jointly owned by UF and UCSF, and Decibel has secured exclusive licenses to the rights of the two universities in the intellectual property. The technology has been incorporated as a key component of Decibels lead gene therapy program, DB-OTO, for treatment of individuals lacking otoferlin.

Decibels precision gene therapeutic, DB-OTO, also incorporates proprietary, cell-specific regulatory control that restricts expression of the human otoferlin sequence to hair cells of the inner ear, thereby avoiding off-target expression. Decibel is developing DB-OTO in collaboration with Regeneron Pharmaceuticals.

There is a significant unmet need for therapeutics that can restore hearing in individuals with congenital, profound hearing loss. We believe AAV-mediated gene therapy is a modality well-suited to the ear, which could potentially have a major impact for patients, said Laurence Reid, Ph.D., Chief Executive Officer of Decibel. These important agreements support our goal to further advance our lead developmental gene therapy program, DB-OTO.

Terms of the agreement have not been disclosed.

1Akil et al. (2019) Proceedings of the National Academy of Sciences. https://doi.org/10.1073/pnas.1817537116

About Decibel Therapeutics

Decibel Therapeutics is a clinical-stage biotechnology company focused on developing treatments that restore functional hair cells within the inner ear to treat disorders of hearing and balance. Leveraging industry-leading single-cell genomics capabilities and proprietary gene therapy technologies, Decibel has established the worlds first comprehensive research, discovery and drug development platform aimed at restoring hearing and balance function. Decibels pipeline, including its lead gene therapy program (DB-OTO) to treat congenital monogenic deafness and its ototoxicity prevention program, is designed to deliver on our vision of a world in which the privileges of hearing and balance are available to all. For more information about Decibel Therapeutics, please visit http://www.decibeltx.com or follow @DecibelTx.

SOURCE: Decibel Therapeutics

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Novartis buys a new gene therapy for vision loss, and this is one preclinical venture that didn’t come cheap – Endpoints News

Tuesday, November 3rd, 2020

Big Pharma execs usually dont just fade away. Sometimes, they jump to the money side of the biotech business, like Chris Viehbacher and Olivier Brandicourt. Some stick with a batch of mentoring board seats. Joe Jimenez not only plans to do both, hes also going biotech entrepreneur and starting his own play in style.

A week ago, Jimenez and his close partner Mark Fishman, who got to know each other well during their respective time as CEO and early-stage NIBR chief at Novartis, filed papers with the SEC noting that their Aditum Bio Fund I had raised the $133,040,000 they had targeted more than a year ago.

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Sloan Kettering Institute, Bluebird Bio in court trial over gene therapy deal – Crain’s Chicago Business

Tuesday, November 3rd, 2020

Then, Girondi claimed, Sloan Kettering mothballed his work to favor Bluebird, whose chief executivehad a prior business relationship with the cancer center's boss.

Girondis rage has been fueled by Bluebirds trajectory since then: its thalassemia treatment was approved last year by the European Union and at $1.8 million per patient will be among the most expensive.

In my neighborhood, theyd have gotten ball batted for similar behavior, said Girondi, a self-described former street tough from the South Side of Chicago whosbeen using such talk to describe his adversaries for years.

Now, Girondi is finally getting his day in court. Having survived years of legal challenges, which have portrayed his case as absurd and Girondi himself as erratic and ill-tempered, his trial began on Thursday.Errant is seeking hundreds of millions of dollars in damages, according to court filings.

The trial promises a rare glimpse into the not uncommonly messy marriage of medical researchers and for-profit companies, and it will showcase a slew of revealingdocuments and emails that have emerged in the court file, including one that Girondis lawyers described in court as the smoking gun.

Written in June 2010 by Nick Leschly, then interim president of Genetix Pharmaceuticals, which was renamed Bluebird Bio a few months later, the emailsaid:Pat Girondineed to shut him down.

The recipient of the email, another Genetix executive, responded by saying they need to be nice, suck up, etc. to Girondi, so they can review valuable data from a Sloan Kettering scientist with whom Errant was collaborating.

Both Sloan Kettering and Bluebird deny Errants allegations.

Sloan Kettering is vigorously defending itself in court, said Jorge Lopez, executive vice president and general counsel for Memorial Sloan Kettering Cancer Center, in a statement. We also disagree with EGTs characterizations of the case and of the Courts rulings. He declined further comment.

In court filings, Sloan Kettering has argued that Girondis company, called EGT for short,wasnt tricked but rather was short of money and repeatedly failed to meet obligations outlined in its deal.

The evidence shows that EGTs case is the fantasy of a defunct company and its founder that refuse to accept responsibility for their own failure, an attorney for Sloan Kettering Institute wrotein a July 8, 2019 filing.

Bluebirds attorney, Jeffrey Eilender, said the court record has disproved Errantsclaim of a conspiracy between Sloan Kettering Institute, referred to as SKI in court filings, and Bluebird, as well as an allegation that Bluebird gleaned secrets from Girondis company.

None of the evidence relied upon by EGT shows a material issue as to the ultimate fact: none suggests in any way that there was an agreement between Bluebird and SKI to defraud EGT, Bluebirds lawyers wrotein a July 8, 2019 court filing. In fact, EGT does not even explain how or why the facts it cites are relevant here (they are not); it just throws everything at the wall to see if it sticks.

As for Leschlys email saying that Girondi needed to be shut down, Eilender said it referred to Girondi,not the company. Why? Because with all due respect to Mr. Girondi, hes a nudnik, Eilender told the court at January 2019 hearing, explaining Girondi had becomea nuisance.

Leschly wasnt available for an interview, but he has previously expressed his opinions about Girondi and his firm.

Errant is toothless and the guy behind it is completely insane, truly, Leschly wrote in a 2012 email to an investment analyst.

Thalassemia is an inherited blood disorder in which the body doesnt produce enough hemoglobin, the substance in red blood cells that carriesoxygen. Moderate and severe, or beta,cases require frequent blood transfusions and can result in early death.

In 1992, Rocco Girondi was diagnosed with a more severe form of the blood disorder. He was two years old. The next year, Girondi retired from what he describes as a lucrative trading career to devote himself to finding a cure.

Girondi, 62, isnt your typical biotech entrepreneur. A high school dropout, he was listed as one of America's most eligible bachelors in Playgirl magazine in 1988 and appeared on the Oprah Winfrey Show in an episode on male chauvinists. (A copy of the show wasnt readily available; Girondi said he had defended both a mans and a woman's right to work, but believed one should stay home if they have children).A 1987 article about him in Chicago Magazine is entitled Fonzie Gets Rich. He left Chicago decades ago for Italy, where he occasionally performs in concert, playing blues and rock andthe occasional Italian ballad. But hestill speaks in the blunt, sometimes salty, manner of the Chicago neighborhood where he grew up.

He had some money at a time when few others showed interest in gene therapy. By 2000, Girondi began providing financial support to researchers at Sloan Kettering, including Dr. Michel Sadelain, who had brought thalassemia under control in mice, according to Errants complaint. Those researchers had developed a method to replace defective genes in thalassemia patients with a healthy copy. The plan used a modified virus known as a vector to deliver the genetic material into the cells.

At that time, gene therapy was relatively new and scarred by missteps, including a patient who had died after undergoing treatment. In 2005, Sloan Kettering granted a license to develop Sadelains potential gene therapy treatment to the only interested party, Errant Gene Therapeutics, according to the complaint.

Progress was slow and hampered by delays, according to Girondi and his lawyers. But by 2010, Errant had manufactured enough of the medicine to start clinical trials, his lawyers say in court papers.

Girondi said the relationship between his company and Sloan Kettering changedsoon after Craig Thompsons hiring as president and chief executive officer of Memorial Sloan Kettering Medical Centerwas announced in August 2010. By that fall, he said it was clear his company was on the outs.

It ended very strangely, Girondi said. I think thats the best way to say it.

Sadelain didnt respond to messages seeking comment.

Thompson, 67, had previously worked at the University of Pennsylvania, where he had been director of the Abramson Cancer Center. He also co-founded a company called Agios Pharmaceuticals in 2007with the goal of unlocking a new field of discovery in cellular metabolism.

Agiosreceived an infusion of $33 million from several venture capital firms in 2008 including Third Rock Ventures, where Leschly the future Bluebird CEO -- was a partner. Leschly also served as Agioss interim chief business officer,according to a 2010 Bluebird press release. A few months later, when Thompson began his job at Sloan Kettering, he was listed as being on Agioss scientific advisory board.

In September, 2010, Sloan Kettering asked Errant for physical possession of the vector to complete a study which it said was necessary to move forward with clinical trials, according to Errants lawyers. Errant delivered the vector and never got it back, the lawyers said.

Sloan Kettering said in court documents that by 2010, Errant had defaulted on its obligations, and that following arbitration and a new deal the following year, all rights granted to Errant in the 2005 deal reverted to Sloan Kettering.

Thompson started his job at Sloan Kettering in November, 2010. That same month,Sloan Ketteringmet with Bluebird and gave them a technical demonstration on Errants vector, sharing confidential information that served as a preludeto a more formal agreement the next year, according to Errants lawyers.

In November 2010, Bluebirds board of directors in 2010 weighedthe pros and cons of collaborating withSloan Kettering, according to Errants court filings. Among the positives? Eliminates the most threatening competitor, according to thepresentation, which is part of the court record.

Andrew Maslow, a former Sloan Kettering executive, said in an interview thathe made the decision to pursue a collaboration with Bluebird, not Thompson. One reason was the improving landscape for gene therapy, and another was Bluebirds capabilities, he said.

These guys are the real thing. They are totally capable, he said. They were just the opposite of Pat.

While Errant squabbled with Sloan Kettering, Bluebird continued to move toward commercialization of its treatment.

Bluebirds treatment for transfusion-dependent thalassemia patients, Zynteglo, was approved by the European Union last year, and the company plans to apply for U.S. approval in 2021. But thats just the beginning. The gene therapy is also intended foruseas a treatment for sickle cell disease(SCD). It could ultimately generate $1 billion a year in annual sales, according to Bloomberg Intelligence.

These therapies have the potential to transform the lives of patients with thalassemia and SCD,said Marc Engelsgjerd, a biotech analyst at Bloomberg Intelligence, who called the treatments groundbeaking.

Leschly, for one, has already benefited. He has pocketed roughly $78 million from stock sales since the company went public in 2013, according to data compiled by Bloomberg. Girondi and Sadelain have been left to contemplate what might have been.

We could have gotten an incredible product and a Nobel Prize, Sadelain said, in a May 2015 phone call that Girondi recorded, also part of the court file. And right now, we have nothing.

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Sloan Kettering Institute, Bluebird Bio in court trial over gene therapy deal - Crain's Chicago Business

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Analysis of Recent Cell and Gene Therapy Innovations: 2020 Competitive Landscape Report – CD-19 is the Most Common Target Antigen for Allogeneic CAR-T…

Tuesday, November 3rd, 2020

DUBLIN, Oct. 28, 2020 /PRNewswire/ -- The "Competitive Landscape Analysis of Recent Cell and Gene Therapy Innovations" report has been added to ResearchAndMarkets.com's offering.

This research identifies some of the key developments across CAR-T cell therapies and provides insights across technological, IP, and investment landscapes. The study also provides an analysis of the competitive landscape while highlighting the key growth opportunities within the CAR-T cell therapy platform.

Key Topics Covered:

1.0 Executive Summary1.1 Research Focus: Emerging Technologies Enabling chimeric antigen receptor (CAR) T-cell Therapies1.2 Analysis Framework: The Author's Core Value1.3 Research Methodology: Five Steps Toward Success1.4 Key Findings of Technology Breakthrough Driving Sepsis Diagnosis

2.0 Technology Snapshot2.1 Rising Pace of Cell and Gene Therapy Approvals2.2 Regulatory and Ethical Perspectives on Gene Therapy2.3 Rising Demand for Precision Medicine Strategies2.4 Manufacturing Continues to be the Key Bottle Neck2.5 II Generation Chimeric Antigen Receptors Likely to Dominate the Cell Therapy Landscape in the Future2.6 CD-19 is the Most Common Target Antigen for Allogeneic CAR-T Therapies

3.0 Emerging Patent Landscape3.1 Steady Increase in Patent Grants for CAR-T Cell Therapies3.2 University of Pennsylvania and Novartis Lead the Patent Landscape for CAR-T Cell Therapies3.3 China and the US Lead the Patent Landscape for CAR-T Cell Therapies3.4 Snapshot of Key Patent Grants: Novartis3.5 Snapshot of Key Patent Grants: Cellectis and BlueBird Bio

4.0 Analysis of the Investment Landscape4.1 Key M&A Trends Across the Global Life Sciences Sector4.2 Gene Therapy - Venture Capital Funding Assessment4.3 Gene Therapy - Big Pharma In-licensing Deals Assessment4.4 Strategic Insights: Cell Therapies and Gene Therapies, Viral Vector CMOS

5. Analysis of the Competitive Landscape5.1 Allogene Therapeutics5.2 Precision BioSciences Inc.5.3 CRISPR Therapeutics AG5.4 Cellectis S.A.5.5 Celyad5.6 Bristol-Myers Squibb (BMS)5.7 Gilead5.8 Novartis5.9 BlueBird Bio5.10 Summary of the Scoring Methodology5.11 Competitive Analysis of CAR-T Participants

6.0 CAR-T Cell Therapies: Growth Opportunity Universe6.1 Growth Opportunity: CAR-T for Solid tumors, 2020

7.0 Industry Influencers

For more information about this report visit https://www.researchandmarkets.com/r/dgf6f3

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

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SOURCE Research and Markets

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Precision Medicines That Are Tailored and Off-the-Rack – Genetic Engineering & Biotechnology News

Tuesday, November 3rd, 2020

Precision medicines, such as cell therapies, remain expensive to manufacture and hard to access by patients. For example, Kymriah, the first chimeric antigen receptor (CAR) T-cell treatment approved in the United States, can have price tags as high as $475,000. Unfortunately, precision medicines are expensive to develop and manufacture, and the costs are ultimately borne by taxpayers and patients, according to The State of Personalized/Precision Medicine a report issued last year by GlobalData.

Today, companies are developing new models to lower the costs of manufacturing and bring drugs to more patients. Among them are companies developing new business models and services, innovative equipment for on-site manufacturing in hospitals, and improved formulation technology.

A key challenge for companies is scaling up the delivery of precision medicines, notes Janel Firestein, partner and life sciences industry leader at Clarkston Consulting. Companies supplying precision medicines are harvesting material for patients in a hospital or clinic, and then freezing or shipping it fresh to a contract manufacturing organization (CMO), contract development and manufacturing organization (CDMO), or other manufacturing entity.

What were seeing with a lot of our clients leveraging contract manufacturers is theyre contracting for specific slots, she says. They have x number of slots per week or month, and the scalability of that is hard.

Precision medicines are manufactured in small batches in accordance with genetic, environmental, and lifestyle factors, that is, for patients in subpopulations that meet certain well-defined criteria. (The subset of precision medicines known as personalized medicines are even more specific; that is, they are developed uniquely for each individual patient.) If a patient doesnt pass prescreening at the scheduled time, Firestein warns, the manufacturing slot for the patients treatment is lost unless the manufacturer can find another eligible patient.

Conversely, if the company is working across multiple CMOs in different countries, it needs to schedule slots in a predictable way. You need to know which slots are open, Firestein points out. You need to leverage automation and artificial intelligence to give a manufacturing view to physicians at the patient hub, so they know which dates are available and can ensure the patients cells are viable upon receipt at the manufacturing plant.

Orgenesis is among the companies turning to localization to deliver precision medicines to patients. The companys CEO and director, Vered Caplan, is a serial entrepreneur and among the top 20 inspirational leaders in advanced medicine listed in The Medicine Makers Power List 2020. Caplan has developed a point-of-care business model for hospitals that combines technological and biological development with a business strategy.

We see that centralized processing is very costly, she explains. It can be a solution for companies working in clinical trials, butonce you get to marketit is not feasible for large numbers of patients.

The companys Cell & Gene Therapy Biotech Platform incorporates the following elements: POCare Therapeutics, a pipeline of licensed cell and gene therapies (CGTs); POCare Technologies, a suite of proprietary and in-licensed technologies; and POCare Network, a collaborative, international ecosystem of research institutes and hospitals. This platform, the company asserts, is about decentralization, enabling precision medicines to be prepared on-site at hospitals.

The platform automates the production of precision medicines by validating closed box processes to reduce cleanroom footprints once the product gets to market. Caplan works to develop and commercialize drugs that can be licensed for use by hospitals in the Orgenesis network.

What we do is offer a low-cost supply platform with processing and regulatory solutions that are validated in a harmonized fashion, she details. Essentially, we take responsibility for R&D. Our hospitals are partners, and because were working in a network, the economic burden isnt high, and we can supply the therapy at a reasonable cost.

The Orgenesis approach doesnt follow the usual approach, which involves a hospital research center licensing its drug to a pharmaceutical company, which then pays the center for clinical trials. Instead, Orgenesis works in partnership with a partner hospital throughout the commercialization process. Production of the final product is automated and supplied via an on-site point-of-care processing unitreducing the complex logistics involved in transporting cells.

Fujifilm Diosynth Biotechnologies, a global CDMO, is developing a new platform to streamline the development of adeno-associated viruses (AAVs) for gene therapies. There are three methods to make AAVs, says Steve Pincus, PhD, the companys head of science and innovation. Two of the methods use viral vectors, and a third uses plasmids.

People using the latter need a source of cells and plasmids, he notes. Unfortunately, there are few licensable cell lines and few plasmid manufacturers. Consequently, as Pincus points out, If you want to manufacture your GMP plasmids at one of these, you have to wait 6 to 12 months to get in the queue.

Fujifilm wanted to tackle these problems, so it decided to license five different Rep-Cap plasmids, an adenovirus helper plasmid, and a human embryonic kidney 293 (HEK293) cell line for AAV production by plasmid transfection from Oxford Genetics. Pincus explains that by licensing these technologies, the company means to offer an HEK293 master cell bank that is well characterized and stocks GMP-grade Rep-Cap and helper plasmids, so that people can come and use those readily available reagents without having to wait 6 to 12 months, and so that the clients pay only for what they need.

To support the production of AAVs, Pincus and his team are developing specialized upstream and downstream processes. They are also developing in-process analytics for common problems in the AAV manufacturing space, such as measuring empty and full virus capsids.

Earlier this year, on September 8, Lonza announced that in a project at Sheba Medical Center in Israel, the first cancer patient received a CAR T-cell therapy that had been manufactured using the companys Cocoon platform. Cocoon is another model for distributed manufacturinga closed, automated piece of equipment for manufacturing cell therapies at the scale of a single patient, with a custom cassette that incorporates all the media, agents, and other consumables.

When you look at the way cell therapies are manufactured, one of the costs is cleanroom space, says Matthew Hewitt, PhD, head of clinical development and personalized medicine at Lonza. A cleanroom suite graded class B for air quality is noticeably more expensive than one graded class C, and the size of the room also matters. If you move to a closed or functionally closed automated platform like the Cocoon that has integrated cell culture, then you can move to cheaper cleanroom space, Hewitt asserts. or you can increase the manufacturing density in your existing cleanroom to use the space more efficiently.

Hewitt divides CAR T-cell manufacturing into a seven-step process: 1) collecting a patient sample; 2) preparing the sample for manufacturing; 3) activating the cells; 4) modifying (transducing) the cells; 5) expanding cell populations as needed for dosing; 6) washing, harvesting, and formulating the cells; and 7) dosing the patient. According to Hewitt, the steps currently automated by Cocoon include activation, transduction, and washing/harvesting/formulation. Additional automation features, he says, will debut in the coming months. Later this year, the company will begin beta testing automatic magnetic cell separation. Next year, the company plans to incorporate automated sample preparation into the Cocoons cassette.

Speaking on the future of manufacturing for precision medicine, Hewitt says he sees a role for both distributed and centralized models. Lonzas centralized facility in Houston, TX, for example, can offer standardized and well-controlled conditions, as well as an experienced team, for process development and early-stage activities.

Once you get to later stages, he points out, manufacturing needs to be moved toward the point of care to mitigate any issues with logistics. He adds that as cell therapies become more common, building enough space to process patient therapies at a centralized facility becomes increasingly impractical. Even if your centralized location served 50,000 patients a year, he says, the logistics would be a heroic endeavor.

Gene and cell therapies dont have much going on in terms of formulation, says Maria Croyle, PhD, professor of molecular pharmaceutics and drug delivery at the University of Texas at Austin. The formulation side needs to catch up.

She argues that even though precision medicines are often formulated just by adding glycerol to the cells, preparing precision medicines to dose the patient is often a complex process. When I talk about these therapies to my students, she relates, I explain that you need to thaw them out and do complicated dilutions. Its not as simple as adding 5 mL to a flask.

Precision medicines are often stored on-site in ultra-low-temperature (80C) freezers, devices that are, Croyle notes, expensive to run. The costs are often passed onto the patient. In addition, preparing the medications often involves lengthy dilution processes. Any of these medications that arent used within a couple of hours must be discarded, pushing costs yet higher.

Although some companies are moving to freeze-drying as a way to preserve living viruses and cells, preserving a live virus can take 48 to 72 hours. I had no idea until I talked to industry how much freezer dryers were a power drain, she recalls. They use a lot of electricity for 72-plus hours, and thats added to the cost of the drug.

Croyle has developed a method for stabilizing live viruses inspired by the film Jurassic Park, which depicted the recovery of dinosaur DNA from amber. She has three patents on a peelable film, inspired by amber, into which gene therapy or vaccine products can be suspended and dried within hours. You can mix them by 8 am, peel them by 3 pm, and package them to be sent off, she asserts. Its very simple and space savingits just a flat envelope with a strip of film, and it can be used in a variety of ways.

Film-packaged doses, she says, can be rehydrated to produce nasal-sprayable vaccines or injectable gene therapy solutions, or they can be placed under the tongue and upper cheek, where dissolution of the film surface releases the vaccine, activating an immune response. To commercialize the technology, she has founded Jurata Thin Film. The company is named after a mythical Lithuanian goddess who lived in an amber castle under the sea.

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BRIEFNovasep and Handl sign gene therapy product development and manufacturing deal – The Pharma Letter

Tuesday, November 3rd, 2020

Belgian companies Novasep and Handl Therapeutics have signed an agreement for the development and manufacturing of an adeno associated virus (AAV)-based gene therapy drug candidate for the treatment of neurodegenerative diseases.

Handl is developing multiple therapeutic AAV-based programs in collaboration with the Katholieke Universiteit Leuven, with the Center for Applied Medical Research of the University of Navarra, Spain, and with the Biomedical Neuroscience Institute of the University of Chile. The company is currently conducting invesrigational new drug (IND)-enabling pre-clinical studies.

Under the terms of the agreement, Novasep will develop and manufacture AAV vectors designed for these programs and will supply drug substance and drug product to support Handl Therapeutics preclinical and clinical studies.

Michael Linden, co-founder and head of research and development at Handl, said: We are excited to engage with Novasep to develop GMP manufacture capabilities for our novel gene therapies and are happy to have identified an outstanding partner right on our doorstep here in Belgium.

Cedric Volanti, Novaseps president of biopharma solutions, said: This new agreement recognizes Novaseps expertise in the field of viral vectors and will contribute to the important development of the cell and gene therapy market in Belgium.

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BRIEFNovasep and Handl sign gene therapy product development and manufacturing deal - The Pharma Letter

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Scout Bio Advances Novel Gene Therapy for the Treatment of Feline Diabetes – BioSpace

Tuesday, November 3rd, 2020

PHILADELPHIA, Oct. 28, 2020 (GLOBE NEWSWIRE) -- Scout Bio, a biotechnology company focused on revolutionizing pet medicine by delivering a pipeline of one-time therapeutics for major chronic pet health conditions, today announced it has initiated two pilot clinical studies to demonstrate effectiveness of an AAV expressing a GLP-1 analog (SB-009) in treating diabetic felines.

Scout Bio envisions two potential therapeutic applications for SB-009. First, to replace daily insulin injections with a single injection of SB-009 to treat feline diabetes and second to significantly increase the percentage of cats entering remission when SB-009 is given with insulin.

The two initiated pilot clinical studies will investigate each of these two potential treatment paradigms.

Mark Heffernan, Ph.D., Chief Executive Officer of Scout Bio remarked, This one-time injectable therapy for feline diabetes has the potential to provide a convenient single treatment that is an alternative to the burden of twice daily insulin injections. We believe SB-009 has the potential to be a blockbuster product for animal health and that our pipeline of gene therapy products for pets will disrupt and grow major markets.

SB-009 was developed under a collaboration between scientists at Scout Bio and the University of Pennsylvanias Gene Therapy Program, where the protein was engineered to improve potency, circulating accumulation kinetics and manufacturability. The design of SB-009 makes the clinical dose both affordable and commercially attractive.

Matthew Wilson, VP Product Discovery and External Innovation said, This is a further example of our strong collaborative relationship with UPenns Gene Therapy Program and Scouts internal capabilities of executing preclinical research to rapidly identify highly potent AAV gene therapies. In less than 12 months after initiating a discovery program, we are now in a position to transition into patients.

Scout Bio has conducted robust preclinical studies with various GLP-1 constructs in rodents and healthy cats. Key findings include:

Dr. Anne Traas, Scout Bios Chief Development Officer reflected, Owners can be devastated to learn their pet has this life-threatening disease and unfortunately, many are unable to give twice daily insulin injections and have to make the difficult choice to euthanize their beloved pet. A one-time safe and efficacious therapy, given by a veterinarian, that eliminates the need for insulin and worry of hypoglycemia, would greatly improve the current treatment paradigm and result in an improved quality of life for diabetic cats and their owners.

Feline diabetes, a severe disease lacking recent innovation, remains a major challenge for veterinarians and owners to safely and effectively manage. Diabetes in most cats is similar to type 2 diabetes in people. Insulin resistance, caused by factors such as obesity, leads to Beta-cell disfunction (the cells that produce insulin). Cats become insulin dependent when blood sugar levels rise, commonly 3-10 times normal, leading to the development of clinical signs which can seriously and negatively impact both the owner and the cat. The most common signs are increased drinking, increased urination and weight loss despite ravenous appetite.

About SB-009 SB-009 is a recombinant AAV gene therapy viral vector utilizing a novel capsid expressing an engineered feline GLP-1 agonist for the treatment of feline diabetes. GLP-1 has been shown to be a safe and highly efficacious molecule in the treatment of humans with type 2 diabetes and SB-009 is the first gene-therapy delivered GLP-1 to be studied in clinical studies in cats with a view to treat the disease.

The expressed feline GLP-1 analog protein functions by stimulating the beta-cells in the pancreas to produce more insulin and may also have an effect in decreasing insulin resistance. GLP-1 receptor agonists do not decrease glucose levels in animals with normal blood glucose, so there is expected to be a very low, or no risk of hypoglycemia.

About Feline DiabetesMost diabetic cats appear to have disease similar to human type 2 diabetes, which is primarily defined as a combined problem with insulin production by the beta-cells in the pancreas, as well as a decrease in the sensitivity to the normal action of insulin (insulin resistance). In cats, one of the most common factors contributing to insulin resistance is obesity which reduces insulin sensitivity.

Lack of insulin production and decreased sensitivity to insulin causes the glucose (sugar) in the blood to become very high leading to the clinical signs. Very high levels of blood glucose also hurt the beta-cells in the pancreas, leading to further reductions in insulin production.

Substantial progress has been made in the treatment of human type 2 diabetes, even in the early stages of the disease. However, insulin therapy remains the only FDA-approved treatment for diabetes in cats.

About Current Treatment | Feline DiabetesCurrent therapy aims to replace the insulin that the cats body no longer makes by injecting insulin twice daily. Giving insulin in the right amounts may bring the blood sugar levels down. If the blood glucose can be brought under control for the majority of a 24-hour period each day, then the clinical signs will be reduced to manageable levels. Too much insulin can cause the blood sugar to drop to dangerous levels (hypoglycemia), so there has to be a careful balance made between maintaining ideal blood glucose levels andadministering too much insulinwhich may result in life threatening low blood sugar levels.

Often owners find the prospect of administering injections to their cats daunting and the strict regimen of twice daily injections and feedings can be difficult to fit into a busy lifestyle. Unfortunately, not treating the cats is simply not a viable option and usually results in a rapid decline in physical health. Even with insulin treatment, some cats diabetes is not well controlled, resulting in the continuation of clinical signs and/or euthanasia.

About Diabetic RemissionGood control of blood glucose may also allow the beta-cells to rest. That rest may increase their capacity to regain some of their insulin-secreting ability. Insulin administration helps to decrease and control the excess blood glucose levels and complement whatever insulin producing ability the cat has left. In some cases, cats regain enough function to allow the insulin injections to stop. This is called diabetic remission. A cat is determined to be in remission when blood sugar is normal and there is complete correction of clinical signs once insulin has been discontinued.

About Scout BioScout Bio is a biotechnology company focused on revolutionizing pet medicine by delivering a pipeline of one-time therapeutics for major chronic pet health conditions. Scout Bios therapeutics are designed to induce long-term expression of therapeutic proteins in pet patients using AAV vector technology. Scout Bio has an exclusive research and development collaboration with the University of Pennsylvanias Gene Therapy Program. Scout Bios innovative partnerships build on a 20-year history with AAV leaders and is complemented by Scout Bios global leaders in gene therapy research and development. Scout Bio is a private company headquartered in Philadelphia, Pennsylvania. For more information, see http://www.scoutbio.coFor further information, please contact:

Investors:Sarah McCabeStern Investor Relations, Inc.212-362-1200sarah.mccabe@sternir.com

Media:Fran Gaconnier214.417.4142Fran.gaconnier@scoutbio.co

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Scout Bio Advances Novel Gene Therapy for the Treatment of Feline Diabetes - BioSpace

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Gene Therapy Market is projected to be worth USD 10 Billion by 2030, growing at an annualized rate of over 40%, claims Roots Analysis – PRnews Leader

Tuesday, November 3rd, 2020

The success of the approved gene therapies has led to an upward surge in the interest of biopharmaceutical developers in this field, resulting in a significant boost in clinical research initiatives and several high value acquisitions

Roots Analysis has announced the addition of Gene Therapy Market (3rd Edition), 2019-2030 report to its list of offerings.

Encouraging clinical results across various metabolic, hematological and ophthalmic disorders have inspired research groups across the world to focus their efforts on the development of novel gene editing therapies. In fact, the gene therapy pipeline has evolved significantly over the past few years, with three products being approved in 2019 alone; namely Beperminogene perplasmid (AnGes), ZOLGENSMA (AveXis) and ZYNTEGLO (bluebird bio). Further, there are multiple pipeline candidates in mid to late-stage (phase II and above) trials that are anticipated to enter the market over the next 5-10 years.

To order this 550+ page report, which features 190+ figures and 355+ tables, please visit this link

Key Market Insights

Around 470 gene therapies are currently under developmentNearly 45% of pipeline drugs are in the clinical phase, while rest are in the preclinical / discovery stage. Gene augmented therapies presently represent 66% of the total number of such interventions that are in the pipeline. It is worth mentioning that majority of such product candidates are being developed as in vivo gene therapies.

More than 30% of clinical stage pipeline therapies are being designed for treating oncological disordersConsidering the overall pipeline, over 20% of product candidates are being developed to treat various types of cancers, followed by those intended for the treatment of metabolic (15%) and ophthalmic disorders (12%). It is also worth highlighting that adenovirus vectors are presently the preferred vehicles used for the delivery of anticancer gene therapies.

Over 60% of gene therapy developers are based in North AmericaOf the 110 companies developing gene therapies in the abovementioned region, 64 are start-ups, 26 are mid-sized players, while 18 are large and very large companies. Further, within this region, most of the developers are based in the US, which has emerged as a key R&D hub for advanced therapeutic products.

More than 31,000 patents have been filed / published related to gene therapies, since 2016Of these, 17% of patent applications / patents were related to gene editing therapies, while the remaining were associated with gene therapies. Leading assignees, in terms of the size of intellectual property portfolio, include (industry players) Genentech, GSK, Sangamo Therapeutics, Bayer and Novartis, (non-industry players) University of California, Massachusetts Institute of Technology, Harvard College, Stanford University and University of Pennsylvania.

USD 16.5 billion has been invested by both private and public investors, since 2014Around USD 3.3 billion was raised through venture capital financing, representing 20% of the total capital raised by industry players till June 2019. Further, there have been 28 IPOs, accounting for more than USD 2.2 billion in financing of gene therapy related initiatives. These companies have also raised significant capital in secondary offerings.

30+ mergers / acquisitions have been established between 2014 and 2019Examples of high value acquisitions reported in recent past include the acquisition of AveXis by Novartis (2018, USD 8,700 million) and Bioverativ by Sanofi (2018, USD 11,600 million).

North America and Europe are anticipated to capture over 85% of market share by 2030With a promising development pipeline and encouraging clinical results, the market is anticipated to witness an annualized growth rate of over 40% during the next decade. In addition to North America and Europe, the market in China / broader Asia Pacific region is also anticipated to grow at a relatively faster rate.

To request a sample copy / brochure of this report, please visit this link

Key Questions Answered

The USD 10 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom gene therapies are likely to be the most promising treatment options for genetic disorders. The report includes detailed transcripts of discussions held with the following experts:

The research covers brief profiles, featuring an overview of the therapy, current development status and clinical results. Each profile includes information on therapeutic indication, targeted gene, route of administration, special designations, mechanism of action, dosage, patent portfolio, technology portfolio, clinical trials and recent developments (if available).

For additional details, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

or email [emailprotected]

Contact:Gaurav Chaudhary+1 (415) 800 3415+44 (122) 391 1091[emailprotected]

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Gene Therapy Market is projected to be worth USD 10 Billion by 2030, growing at an annualized rate of over 40%, claims Roots Analysis - PRnews Leader

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Foundational research shows early gene therapy prevents Angelman syndrome – BioWorld Online

Tuesday, November 3rd, 2020

Scientists working at the University of North Carolina, Chapel Hill reported in the Oct. 21, 2020, issue of Nature on the successful development of a one-time specific sequence-directed gene therapy approach using the combination of AAV with CRISPR technology that successfully prevented the presentation of Angelman syndrome throughout the lifetime of a mouse model.

Lifelong gene therapy has held promise for decades now as one of the only approaches that could possibly address many neurodevelopmental genetic disorders. But even after decades of research, gene therapy still possesses significant risks due to untoward random genomic insertions of vectors that could ultimately cause other genetic disorders.

Meanwhile, it has been known for decades now that adeno-associated virus (AAV) is a particularly powerful potential gene therapy vector because AAV integrates into the genome so well. However, the integration of AAV has always been random and so it inherently comes with significant risk.

This is the first time that a treatment for Angelman syndrome has been shown to correct this neurodevelopmental disorder.

Principal investigator, Mark Zylka, professor of Cell Biology and Physiology in the Neuroscience Center, University of North Carolina, Chapel Hill, told BioWorld Science, "The key really from what we can tell is going early in treatment. So for the animals that have the disorder we can identify them with genotyping. If you catch it early, you can treat them one time and it lasts forever as far as we can tell.

That longevity, he said, "contrasts with treatments that are in development using antisense technologies that usually have to be injected every 4 months or so, which is not ideal for a pediatric disorder that will last a lifetime."

Angelman syndrome is caused by loss of function of the maternal Ube3a allele, while the paternal allele is normally silenced by a very long antisense noncoding RNA known as Ube3a-ATS. Previously in a 2011 Nature publication Zylka and collaborators demonstrated that a class of drugs called topoisomerase inhibitors could reactivate the paternal allele by interfering with Ube3a-ATS. So Zylka knew that if the paternal copy of Ube3a can be turned on, this will provide the possibility of treating the condition.

Topoisomerase inhibitors, which include chemotherapy agents such as irinotecan and doxorubicin, are not a therapeutic option for Angelman syndrome due to their broad-spectrum nature and toxicity. But with the development of CRISPR combined with AAV, the researchers have now developed a tool to precisely hone in on specific regions of the genome.

First, the team screened 250 different RNA guided CRISPR/Cas9 constructs in cell culture until they identified the best one (Spjw33) reactivating the Ube3a-ATS allele. These clones had the good fortune to target Snord115 genes within the large Ube3a-ATS locus. The Snord genes are functionally redundant, with over 100 of them present in both mice and humans.

Ultimately the CRISPR/Cas9 with the cloned RNA guide was used to a specific region of the DNA, where DNA was inserted into the Snord115 gene of the Ube3a-ATS locus. The inserted DNA possessed a polyadenylation signal that caused the premature termination of the Ube3a-ATS noncoding RNA such that it no longer silenced the paternal expression of Ube3a.

With the Ube3a now made in the mouse, it fully developed and no longer presented with any phenotypes resembling Angelman syndrome throughout the life of the animal.

In short, instead of deleting the gene, this approach disrupted the Ube3a-ATS gene by stopping its full production prematurely. Only a small nonfunctioning part of the noncoding RNA was still produced in treated animals.

Earlier is better

The broad implications are that the study proves that Angelman syndrome can be treated and possibly prevented, if it is done early enough.

Previous studies showed that if turning on the paternal copy later, even within just a few days after birth in a mouse, this approach does not prevent Angelman syndrome.

Zylka said, "It is like with a building. You want to make sure the foundation is done correctly. Tons of time is put into the foundation. If there is a problem with the foundation, then when building on top of it, it is very hard and next to impossible to go back and fix the foundation. When the brain is developing, it is the initial foundation upon which the brain is built that is critical and you cannot really go back and fix it. So this study now shows that you can fix the problem if you catch it early enough by administering just a single treatment."

One encouraging result was the lack of gene therapy occurring in the mother. The team injected the vector into the fetus, but no gene therapy was detectable in the mother's liver and brain. Instead, the gene therapy was restricted to only the fetus. This was remarkable and very important since AAV is well known to particularly target the liver.

The technology to identify fetuses with the mutation that causes Angelman syndrome is already available and currently used in hospitals around the world. Techniques like amniocentesis, chorionic villus sampling, and even newer noninvasive technologies involving taking extra blood from the mom can now detect fetal DNA and cells to find out if there are any Angelman syndrome mutations.

However, there has not been a strong incentive to look for Angelman syndrome given that there are no therapeutic options at this point.

Zylka hopes to ultimately test the approach in the clinic. But first-time gene therapy technologies are often only given one shot in clinical trials and safety is of primary concern. So, extensive further research will be necessary to not throw away his shot (Wolter, J.M. et al. Nature 2020, Advanced publication).

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Foundational research shows early gene therapy prevents Angelman syndrome - BioWorld Online

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Sarepta, continuing its gene therapy push, helps launch a startup – BioPharma Dive

Sunday, October 25th, 2020

Dive Brief:

Sarepta is best known for its RNA technology platform, which has led to two approved though also controversial drugs for Duchenne muscular dystrophy.

Yet, Sareptais also deeply invested in gene therapy, having developed an extensive list of more than two dozen experimental treatments, six of which have reached human testing.

Rare diseases have been an early target in this rapidly growing field. The two gene therapies approved in the U.S., Roche's Luxturna and Novartis' Zolgensma, are respectively used to treat an uncommon form of blindness and a muscle disease that occurs in about 1 in every 10,000 births.

Sarepta'stherapies target a wide variety of rare diseases, including Duchennemuscular dystrophy, Pompe disease, and types of Limb-girdle muscular dystrophy. Taking a stake in AavantiBio, with its work in Friedreich'sataxia, could expand Sarepta'sreach even further. The disease affects approximately 1 in every 40,000 people, according to the National Organization for Rare Disorders, which would equate to around 8,200 patients in the U.S.

"Our equity participation in AavantiBio serves our strategy to build our gene therapy engine through targeted investment in potentially life-enhancing therapies,"Doug Ingram, Sarepta'sCEO, said in a statement Thursday.

AavantiBio joins a couple large, powerful companies in the hunt for a gene therapy to treat Friedreich's ataxia. Pfizer and Novartis are each working on their own programs.

Outside of gene therapy, Reata Pharmaceuticals disclosed last year positive datafrom a study that tested an oral drug, known as omaveloxolone,in patients with Friedreich's ataxia. Reata said it intends to file the drug for approval based on those results.

AavantiBiowill be headquartered in the greater Boston area, putting it close by Cambridge, Massachusetts-based Sarepta. Cumbo, along with his role as CEO, will take one of AavantiBio'seight board of directors seats. Cumbohas, during the span of his career, helped launch 11 specialty products across multiple drug companies, including Sarepta, Gilead and Vertex, where he built a sales team for the hepatitis C drug Incivek.

Co-founders Byrne and Corti will be on the board too, alongside two independent directors and representatives from the investor group of Perceptive, Bain and RA.Louise Rodino-Klapac, senior vice president of gene therapy for Sarepta, will serve as a board observer.

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Resurgence of gene therapy has dramatically altered the the biomedicine revolution – Genetic Literacy Project

Sunday, October 25th, 2020

Some technologies that have emerged and altered the landscape in recent years include immunotherapy, CRISPR-Cas9 gene editing, and chimeric antigen receptor (CAR) T-cell therapies. Now, another platform technology is maturing from the research laboratory to commercial viability. In 2017, the U.S. Food and Drug Administration (FDA) approved the first directly administered gene therapy for mutations of a specific, disease-related gene. That product, Luxturna, marketed by Spark Therapeutics, delivers a functioning copy of the RPE65 gene to retinal cells using an adeno-associated virus (AAV) as a vector to treat a genetic form of blindness.

This advance has injected new energy into biotech startups seeking to capitalize on gene therapy.

As the field matures, gene therapy companies will eventually need to find a way to sell once-in-a-lifetime cures in a market built around chronic therapies. In some ways, its been easier for our European counterparts and other single-payer economies, [research analyst Mani] Foroohar comments. [They] can rest more assured that the financial benefits of reducing future hospitalizations due to expensive future illness will be captured by the same payor thats providing the upfront outlay.

Just as biotechnology has transformed every aspect of our healthcare system over the last 40 years, gene therapy will challenge, disrupt, and overturn our healthcare pricing and reimbursement paradigms as it becomes an increasingly common and routine treatment approach.

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Resurgence of gene therapy has dramatically altered the the biomedicine revolution - Genetic Literacy Project

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SparingVision Nabs 44.5 Million to Support Gene Therapy, Adds New CEO – BioSpace

Sunday, October 25th, 2020

Paris-based SparingVision, a genomic medicine company focused on ocular diseases, raised 44.5 million (approximately $52.2 million) in a financing round. Funds will be used to advance the development of the companys treatment for a genetic eye disorder that can lead to vision loss.

SparingVision is developing SPVN06 for the mutation-agnostic treatment of retinitis pigmentosa, the most common inherited retinal degeneration that affects about two million people globally. There is currently no approved treatment to treat all genetic forms of this rare retinal disease that leads to blindness, the company said. According to SparingVision, SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy consisting of one neurotrophic factor and one oxidative stress reducing enzyme which, acting synergistically, aim to slow or stop the degeneration of photoreceptors. Loss of photoreceptors leads to blindness in retinitis pigmentosa. In June, the European Commission granted Orphan Drug designation to SPVN06

In addition to advancing its gene therapy treatment, funds from the financing round will be used to support SparingVisions GMP activities, including the manufacturing of a first clinical batch of the product, as well as regulatory activities. Funds will also be used to begin human trials of the gene therapy, which are set to begin in 2021.

Gene therapy has already been approved by regulatory to approve a type of genetic blindness. Spark Therapeutics, now a division of Roche, won regulatory approval for Luxturna (voretigene neparvovec), a gene therapy for a rare, genetic form of blindness. Luxturna is approved for the treatment of pediatric and adult patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. The disease can lead to vision loss and may cause complete blindness in certain patients. The approval marked the first time the U.S. Food and Drug Administration approved a directly administered gene therapy that targets a disease caused by mutations in a specific gene.

In addition to developing its gene therapy for retinitis pigmentosa, SparingVision said it intends to establish a toehold in the United States and will expand its management team.

The financing round was led by 4BIO Capital and UPMC Enterprises. It was supported by Jeito Capital and Ysios Capital. Current investors Bpifrance and Foundation Fighting Blindness also participated in the round. Torreya Capital, LLC served as exclusive placement agent for the offering.

In addition to the financing, Stphane Boissel, who currently serves as chairman of the board of directors, was named chief executive officer of the company. He takes over from Florence Allouche, a cofounder of the company. Boissel, who previously served as head of corporate strategy at Sangamo Therapeutics, said the support SparingVision received in the financing round demonstrates the excitement about the potential of SPVN06.

With its singular mutation-agnostic approach, SPVN06 could have a much broader commercial potential than most gene therapy products for RP currently in development and will be used as an anchor to build an economically-viable portfolio of therapies in the field of ophthalmology. Our shareholders, both new and existing, are all long-term, strategic and patient-centric investors that share our vision and we are excited to be working with them to achieve our goals, Boissel said in a statement.

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SparingVision Nabs 44.5 Million to Support Gene Therapy, Adds New CEO - BioSpace

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Sana CEO reveals details about stealthy gene therapy startup that has raised more than $700M – GeekWire

Sunday, October 25th, 2020

Luke Timmerman interviews Sana Biotechnology CEO Steve Harr at the GeekWire Summit this week.

Sana Biotechnology CEO Steve Harr shed more light on one of most secretive, heavily funded startups in Seattle and the global biotech industry detailing its plans to create tools that replace and repair human body cells, with the potential to treat various diseases and create new medicines.

Harr spoke with biotechnology journalist Luke Timmerman, founder of The Timmerman Report, this week at the GeekWire Summit. Sana raised more than $700 million this summer in one of the largest venture financing deals in the life sciences industry and one of the biggest rounds on record in Seattle.

Founded in 2019, the 250-person company has an ambitious goal of both repairing cells in the body (gene therapy) and also replacing damaged cells (cell therapy). Its led by several former executives from Juno Therapeutics, another Seattle biotech company that went public in 2014 and sold to Celgene for $9 billion in 2018.

Sana has kept a relatively low profile since launching. It is competing with much larger entities that have deeper pockets and more robust logistics capabilities. But Harr said a startup such as Sana has a key differentiator.

We have one competitive advantage: we can make faster and better decisions, he said. We get there because we have better people, we have greater focus, and we have better communication.

Read on for key takeaways from the conversation.

How Sana started:Harr and his former colleagues at Juno learned a lot about engineering cells and manipulating genes during their startup journey. Juno was among a handful of U.S. companies making cutting-edge cancer immunotherapy treatments.

But they also knew there was more opportunity in a nascent industry of gene and cell therapy.

We wanted to build the transformative or winning company of this next era, of the next 20 years, Harr said. To do that, we had to break the model of what biotech is, which is typically taking an idea and figuring out where to apply it best.

Sana instead is trying to build the platform that can engineer cells and fix them, much like building a computer.

There are a whole host of component parts that go into it, Harr said. We have to aggregate the right technologies.

Harr said too many biotech companies sell solutions in search of problems. He likened it to someone showing up with a tiny screwdriver and looking for a loose screw to fix. Harr sees Sana more as a toolbox that can help build the right medicine for the right patient.

Sana is targeting various disease areas, including cancer, diabetes, genetic disorders, and more. They are relatively diverse, but there are some really fundamental underlying platform and strategy principles that drive each of those, Harr said.

Sanas secret sauce:One key focus for the company is reimagining the delivery system for these therapies how to get DNA, RNA, proteins, etc. into a cell. Ultimately at the core, what were trying to do is really improve delivery, and really figure out how to hide cells from the immune system, Harr said.

Hiding re-engineered or replaced cells from ones immune system is important to prevent the possibility of the body rejecting the new cells.

Harr also talked about delivering therapy via injection, with the body becoming the bioreactor. Its similar to technology built by Moderna and others. You deliver the tools to enable your body to make its own medicine, Harr said.

Manufacturing:Sana is also aiming to innovate how gene and cell therapies are produced and distributed at scale. They are typically expensive Timmerman said CAR T-cell immunotherapies for cancer ran in the $300,000-to-$400,000 range per patient. Figuring out manufacturing costs at scale and making it less than current alternative methods of care for patients will be key to the strength of Sanas business. Harr added that you have to do it in a way thats constructive for the system.

Headcount: Sana employs 250 people spread across offices in Seattle, the Bay Area, and Cambridge, Mass. Having three outposts helps the company attract the best talent, Harr said. One advantage to raising so much capital is being able to hire the best folks. Last month Sana added top scientists Ed Rebar and Terry Fry to the executive team.

If you really hire one of the true world leaders in something, it is pretty amazing how quickly teams form around them, Harr noted.

Money matters: Having more than $700 million in the bank helps Sana in various other ways. Harr said a lot of biotech companies often run what amount to experiments to justify raising more capital. We have the privilege of running experiments to find truth as fast as you want to, he said. And then we want to have the balance sheet, technologies, and people to be able to grapple with whatever the truth is.

Timeline: Harr said the company is on track with its original strategy but does not plan on selling medicine in the next two years. It will progress with multiple medicines in parallel, not one at a time, Harr said.

Leadership advice:During the pandemic and remote work, Harr said hes started to reach out to four-to-six people at Sana each week that he wouldnt normally talk with. He holds half-hour meetings to chat about what they are working on, and what leadership could do to make their life or job better. I found that to be just such an invigorating way to learn whats going on, he said.

[The full interview with Harr, and other GeekWire Summit sessions, are available on-demand exclusively to attendees of the virtual event.Learn more and register here.]

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Sana CEO reveals details about stealthy gene therapy startup that has raised more than $700M - GeekWire

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