StemCell Therapy

WATERTOWN, Mass. and RESEARCH TRIANGLE PARK, N.C., Nov. 07, 2019 (GLOBE NEWSWIRE) -- SQZ Biotechnologies (SQZ), and Asklepios BioPharmaceutical, Inc. (AskBio), announced a research collaboration to create tolerizing antigen carriers (TACs) containing AAV (adeno-associated virus) components to solve one of gene therapys biggest challenges the barrier to treatment posed by patients immune systems generating neutralizing antibodies toward therapeutic AAVs. SQZ and AskBio will combine their proprietary cell and gene therapy platform technologies to open the door to new treatment paradigms with potential impact across many genetic diseases.

Gene therapies utilizing AAV vectors can be transformative for patients with genetic diseases, but neutralizing antibodies can prevent large populations of patients from benefitting from AAV gene therapies. Patients immune systems develop neutralizing antibodies after receiving their first dose of AAV, or they can be pre-existing. This collaboration will strive to give these patients access to novel therapeutics and enable them to take multiple or repetitive doses to gain the full, durable benefit these treatments can provide. Expanding patient eligibility and allowing repeat treatment could change the future of how products are developed and significantly impact the long-term health of millions in need.

This is a tremendous opportunity to bring together the power of both cell and gene therapy for patients. AskBio has been an innovative leader in gene therapy and shares our patient-centric philosophy. By working together and leveraging the potential of both our platforms, we hope to bring more effective, more durable treatments to patients suffering from devastating rare genetic disorders, said Armon Sharei, PhD, founder and chief executive officer of SQZ Biotech.

The collaboration between SQZ and AskBio will evaluate the administration of SQZ TACs and AskBios gene therapies to potentially address AAV immunogenicity. SQZ is a pioneer in cell therapy, and the companys knowledge and expertise, as well as their advance capabilities in manufacturing, are critical to this collaborations approach to synergizing cell and gene therapies. Preclinical data from SQZ has demonstrated that SQZ TACs specifically inhibit undesired immune responses in multiple contexts, including AAV models. As a leader in the AAV field, AskBio brings expertise in AAV technology, capsid design, clinical processes and manufacturing that would allow for application of these novel methods to overcome immunogenicity. The two companies have a shared goal to increase world-wide access of transformative therapeutics.

R. Jude Samulski, PhD, chief scientific officer and co-founder of AskBio, noted, AskBio is firmly committed to improving the lives of underserved patients, such as those suffering from Pompe, Huntingtons and various neuromuscular and central nervous system diseases. Addressing AAV immunogenicity is essential to the future of gene therapy as it is one of the most significant limiting factors plaguing the gene therapy space today. SQZs pioneering approach to tolerance could offer a solution to this problem. Our collaboration with SQZ is exemplary of our goal to broadly explore potential redosing of AAV gene therapies, added Sheila Mikhail, chief executive officer and co-founder of AskBio. We are thrilled to be working with SQZ and are hopeful that this initial research collaboration utilizing two of the most promising therapeutic modalities currently available, cell and gene therapy, will ultimately provide options to improve patients immune response to gene therapy.

About AskBioFounded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy platform company dedicated to improving the lives of children and adults with genetic disorders. AskBios gene therapy platform includes an industry-leading proprietary cell line manufacturing process known as Pro10 and an extensive AAV capsid library. Based in Research Triangle Park, N.C., the company has generated hundreds of proprietary third-generation gene vectors, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production, chimeric vectors, and self-complementary DNA. AskBio maintains a portfolio of clinical programs across a range neurodegenerative and neuromuscular indications with a current pipeline that includes therapeutics for Pompe disease, Limb Girdle Muscular Dystrophy and congestive heart failure as well as out-license clinical indications for Hemophilia (Chatham Therapeutics acquired by Takeda) and Duchenne Muscular Dystrophy (Bamboo Therapeutics acquired by Pfizer). For more information, visit http://www.askbio.com.

About SQZ BiotechSQZ Biotech is a privately held, clinical-stage company creating innovative treatments by transforming cells into sophisticated therapeutics. Using its proprietary platform, SQZ has the unique ability to precision engineer virtually any cell type and deliver multiple materials, potentially resulting in powerful, multifunctional cell therapies for a range of diseases with an initial focus on cancer and autoimmune disease. The companys initial applications leverage SQZs ability to generate target-specific immune responses, both in activation for the treatment of solid tumors, and immune suppression for the treatment of immune reactions and diseases. For more information please visit http://www.sqzbiotech.com.

About SQZ TACsSQZ tolerizing antigen carriers (TACs) are being developed to induce tolerance to aberrant or unwanted immune activity. TACs are developed from red blood cells (RBCs) SQZd with target-specific antigens and piggyback on the natural process of RBC destruction in the body, also known as eryptosis. A process moderated by our liver and spleen, eryptosis causes macrophages to take up aged or senescent RBCs. When our bodies process RBCs for destruction, their components are presented in a tolerogenic manner, reminding our immune systems not to attack our own red blood cells. SQZ TACs drive targeted antigensthrough this powerful natural mechanism, specifically tolerizing the immune system, potentially stopping undesired immune responses.

AskBio Contacts: Mark Rosenbergmark@trueparallel.com919-412-7378

Roger Friedensen, APRroger@trueparallel.com919-349-3206

SQZ Contacts:Rebecca CohenSenior Manager, Corporate Relationsrebecca.cohen@sqzbiotech.com617-758-8672 ext. 728

Cait Williamson, PhDLifeSci Public Relations cait@lifescipublicrelations.com646-751-4366

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SQZ Biotech and AskBio Announce Research Collaboration to Create Immune Tolerization Products for AAV Gene Therapies - GlobeNewswire

Cell therapy CDMO Cognate Bioservices will add plasmid DNA and viral vector capabilities through the acquisition of Swedish manufacturer Cobra Biologics.

Memphis, Tennessee-based contract development and manufacturing organization (CDMO) Cognate has entered into an agreement to acquire Cobra for an undisclosed fee, led by existing Cognate investor EW Healthcare Partners.

The deal adds to Cognates presence in the regenerative medicine space by bringing on board plasmid DNA and viral vector manufacturing capacity and expertise, complementing its own autologous and allogeneic cell-based and cell-mediated gene therapy capabilities.

Image: iStock/Good_Stock

According to Cognate, the deal will create a fully integrated cell and gene therapy CDMO providing more scalable solutions to its clients.

This acquisition is central to Cognates strategy to build on its existing offerings and create an enterprise platform for life cycle management of cell and gene therapy products, accelerating the availability of new technologies to patients that need them most, said Cognate CEO J. Kelly Ganjei.

The combined Cognate-Cobra expertise, infrastructure, and geographical footprint immediately positions both businesses to better respond to current and future market needs more quickly, effectively, and comprehensively.

The transaction is subject to receipt of approval for the US authorities under the Hart-Scott-Rodino Antitrust Improvements Act of 1976.

Peter Coleman, CEO of Cobra Biologics, told Bioprocess Insider the two companies will operate as different business units and he will continue to be involved with Cobra following the acquisition.

There will be elements of integration, in particular linking the technical capabilities together, but the intention is to operate as two separate business units each with its own specialization and track record.

Cognate operates an 80,000 square-foot site in Memphis, originally built in 2017 for autologous cell therapy manufacturing but now produces various cell types for customers clinical projects. Our largest phase of clinical grade production was an autologous product for phase III clinical trials, mostly manufactured in Memphis with products shipped to more than 80 clinical sites in four countries, the company states.

Cobra brings to the table two GMP approved facilities: an advanced therapy medicinal product (ATMP) production site in northwest UK offering DNA and viral vector services, and an ATMP production site in Matfors, Sweden offering DNA and microbiota services.

Last month, Cobra inked a deal to manufacture the adeno-associated viral vector (AAV) for Nordic gene therapy company Combigenes epilepsy candidate CG01 from its facility in the UK.

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Cognate buys Cobra to boost gene therapy CDMO - Bioprocess Insider - BioProcess Insider

A team of scientists from City University of Hong Kong (CityU) and the Karolinska Institute has created a novel protein that can increase the target accuracy in genome editing. Their findings are published in the journal Proceedings of the National Academy of Sciences (PNAS).Meet CRISPRThe gene editing technology Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cas9 looks set to revolutionize modern medicine, agriculture, and synthetic biology.The ability to edit the genome in vivo offers the potential to develop novel gene therapies for diseases that currently lack viable treatment options. Several clinical trials are underway exploring the utility of CRISPR technology in treating specific cancers, blood disorders and eye diseases.CRISPR-Cas9 as a gene editing tool is superior over other techniques due to its ease of use. In traditional gene therapy, additional copies of the "normal" gene are introduced into cells. Using CRISPR technology, this isnt necessary; CRISPR-Cas9 enters the cell and "repairs" the problematic gene by removing it or correcting it to restore normal physiological function.

There are different components to the CRISPR mechanism. Cas9 is the enzyme that flags and locates the problematic DNA throughout the genome, acting in a "hunting" fashion. However, the precision of Cas9 cannot always be established, and occasionally modifications of DNA at unintended places can occur. If CRISPR is to be utilized to repair faulty genes in patients, potential off-target genome editing could have serious adverse effects.

There are currently two versions of the Cas9 enzyme commonly adopted in CRISPR research: SpCas9 (Cas9 nuclease from the bacteria Streptococcus pyogenes) and SaCas9 (Cas9 nuclease from Staphylococcus aureus). Both of these enzymes are limited in that they possess a certain level of imprecision.

Thus, scientists have endeavored to develop variants of both enzymes, with the aim being to increase their precision and reduce off-target effects. The issue with SpCas9 is that the modified variants are often too large to "fit" in the delivery system adopted for inserting gene therapies into patients, known as adeno-associated viral (AAV) vectors.SaCas9 is advantageous over SpCas9 in that it can be easily packaged into the AAV vectors for delivering gene-editing contents in vivo. However, at present, there is no SaCas9 variant that possesses high accuracy in genome-wide editing. Until now.Now meet SaCas9-HFIn the new study published in Proceedings of the National Academy of Sciences (PNAS), a research team led by Zheng Zongli, Assistant Professor of Department of Biomedical Sciences at CityU and the Ming Wai Lau Centre for Reparative Medicine of the Karolinska Institute in Hong Kong, and Shi Jiahai, Assistant Professor of Department of Biomedical Sciences at CityU, has successfully engineered SaCas9-HF, a CRISPR Cas9 variant which has demonstrated high accuracy in genome-wide targeting in human cells without compromising on-target efficiency.In the study, the scientists conducted an extensive evaluation of 24 targeted human genetic locations comparing the original (known as wild-type) SaCas9, and the new variant, SaCas9-HF. They discovered that for targets with highly similar sequences in the genome (and therefore often disposed to off-target editing by wild-type Cas9), SaCas9-HF decreased the off-target activity by ~90%. When assessing targets that had relatively less off-targeting editing by wild-type SaCas9, the SaCas9-HF enzyme produced little to no detectable off-target effects.

"Our development of this new SaCas9 provides an alternative to the wild-type Cas9 toolbox, where highly precise genome editing is needed. It will be particularly useful for future gene therapy using AAV vectors to deliver genome editing 'drug' in vivo and would be compatible with the latest 'prime editing' CRISPR platform, which can 'search-and-replace' the targeted genes," said Dr Zheng.Reference: Tan et al. 2019. Rationally engineered Staphylococcus aureus Cas9 nucleases with high genome-wide specificity. Proceedings of the National Aacademy of Sciences (PNAS). DOI: https://doi.org/10.1073/pnas.1906843116

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A Highly Precise Cas9 Enzyme, SaCas9-HF, Is Added to the CRISPR Toolbox - Technology Networks

7-Nov-2019

Design and Build

Canadian cleanroom builder to work in the US on a new 1,600-sq-ft site of ISO Class 7

The New Jersey Innovation Institute is located in Newark, in the United States

Mecart, the Canadian cleanroom specialist, has been selected by the US-based New Jersey Innovation Institute (NJII) for a new GMP cleanroom at its Cell and Gene Therapy Development Center. The NJII is based in Newark, US.

Commenting on the project with Mecart, Dr Haro Hartounian, NJII Senior Executive Director, Biotechnology and Pharmaceutical Innovation, said: We were excited to meet with the Mecart team, travel to Quebec City to visit with their leadership, and review the project in detail with them. It has been a great experience thus far and we know that we picked the best partner for this critical project.

The project represents a new GMP cleanroom of approximately 1,600 square feet of ISO 7 space developed specifically for advanced cell and gene therapy processing and manufacturing operations.

Its an honour to work with such an innovative company that focuses on local collaboration with its technological resources, like this new cleanroom, said Patrice Genois, General Manager of Mecart and Vice President of PolR.

The NJII is an NJIT corporation that applies the intellectual and technological resources of the states science and technology university to challenges identified by industry partners.

Upon completion, the new GMP site will also serve as a training facility and a venue for collaborating with local manufacturing.

Charles Lipeles, Vice President of US Operations, commented: When NJII approached Mecart, they were clear that lead time was crucial as was a very tight specification for their state-of-the-art GMP cleanroom. They had very aggressive goals, made more challenging when dealing with an institution with government ties, but we were ready for the challenge. We are excited to work with the NJII and NJIT teams and help them exceed their goals with this new suite of cleanrooms."

Construction is planned to begin later this year and will be completed in Q1 of 2020.

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NJ Innovation Institute chooses Mecart for cell and gene therapy centre - Cleanroom Technology

Registrational Trial for Wiskott-Aldrich Syndrome Met Key Primary and Secondary Endpoints at Three Years; Data from Integrated Analysis Reinforce Treatment Benefits of Gene Therapy and Durability of Effect in Additional Patients

Similar Profiles Reported Between Cryopreserved and Fresh Formulations of OTL-101, Further Supporting Upcoming Regulatory Filing and Broad Patient Availability

BOSTON and LONDON, Nov. 06, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced the upcoming presentation of registrational data from multiple programs at the 61st American Society of Hematology (ASH) Annual Meeting in Orlando, FL.

Investigators will describe ongoing clinical progress for two lead development programs in the companys primary immune deficiencies portfolio: OTL-103, an investigational gene therapy in development for the treatment of Wiskott-Aldrich syndrome (WAS) at theSan Raffaele-Telethon Institute for Gene Therapy(SR-Tiget) inMilan, Italy; and OTL-101, an investigational gene therapy in development for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).

In addition, investigators will deliver an oral presentation featuring updated data from the ongoing proof-of-concept study of OTL-203, an investigational gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) atSR-Tiget.

This growing body of positive data, from dozens of patients across multiple diseases, provides a solid foundation as we advance each program toward its next phase of development, including upcoming regulatory submissions for ADA-SCID and WAS, saidMark Rothera, president and chief executive officer ofOrchard Therapeutics. We now have two supportive data sets one from our OTL-101 program in ADA-SCID and one from our OTL-200 program in metachromatic leukodystrophy that demonstrate cryopreserved formulations are engrafting as expected, similar to the fresh formulation. This supports our strategy for making these therapies, if approved, broadly available to patients in need throughout the world.

We are extremely pleased with our continued clinical progress, including the duration of benefits seen in our WAS trial, which is the longest published follow-up of hematopoietic stem cell gene therapy durability to date using lentiviral vector transduction, said Bobby Gaspar, M.D., Ph.D., chief scientific officer of OrchardTherapeutics. The totality of these data underscores the broad applicability of our gene therapy platform approach and the opportunity we have to deliver potentially curative treatments for a variety of devastating and rare genetic disorders.

Full presentation details are below:

Poster Presentation Details

Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010Publication Number: 3346Session: 801. Gene Therapy and Transfer: Poster IIDate and time:Sunday, December 8, 6:00-8:00pm ET

This presentation includes results from an integrated analysis of 17 patients treated with OTL-103 for the treatment of WAS, including the complete data set for the eight patients from the registrational study and nine who received OTL-103 as part of an expanded access program (EAP). Participants have been followed for a median of three years.

In the eight-patient registrational trial, investigators reported that the study achieved its key primary and secondary endpoints at three years, including the elimination of severe bleeding episodes and a significant reduction in the frequency of moderate bleeding episodes. Successful engraftment was observed within three months, leading to an increase in WAS protein expression and a vector copy number that has been maintained for up to eight years. Nine months post-administration, all patients stopped receiving platelet transfusions, and no severe bleeding events were reported. A significant reduction in the rate of severe infections was also observed and all patients were able to stop immunoglobin replacement therapy (IgRT), suggesting a complete reconstitution of immune function with durability of effect of up to eight years of follow-up post-gene therapy.

Similar clinical results were seen in the integrated analysis of 17 patients and overall survival was 94% (16/17). One death occurred among the EAP cohort that was considered by the investigator to be unrelated to OTL-103.

Across the original and integrated data sets, there were no adverse events considered to be related to OTL-103, including no evidence of oncogenesis, replication competent lentivirus or abnormal clonal proliferation. Clinical benefit was also attained in patients older than five years of age, a group considered at higher risk when treated with allogeneic hematopoietic stem cell transplantation (HSCT).

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded CohortPublication Number: 3345Session: 801. Gene Therapy and Transfer: Poster IIDate and time: Sunday, December 8, 6:00-8:00pm ET

This presentation details the safety and efficacy of OTL-101 in 30 individuals with ADA-SCID, treated with either fresh (n=20) or cryopreserved (n=10) formulations. Patients were followed for a median of 24 months (range 12-24 months overall and 12-18 months for patients treated with the cryopreserved formulation), and results were compared with a historical cohort of 26 ADA-SCID patients treated with allogeneic hematopoietic stem cell transplantation (HSCT), including HSCT both with, and without, a matched related donor.

Results showed engraftment of genetically modified hematopoietic stem cells in 29 of 30 OTL-101 patients by six to eight months, which persisted through follow-up in both studies. Analysis of both the vector copy number in granulocytes (a measure of engraftment) and T-cell reconstitution (a relevant measure of immune recovery) showed consistent performance across the fresh and cryopreserved-treated patients.

In the OTL-101 treated patients, overall survival was 30/30 (100%) and event-free survival was 29/30 (97%). One of the 30 patients restarted treatment with enzyme replacement therapy (ERT) and subsequently withdrew from the study and received a rescue HSCT. In the historical control population, 42% of HSCT patients required re-initiation of ERT, rescue HSCT or other intervention, or died. As expected, there was no incidence of graft versus host disease in the OTL-101 group, compared with eight patients who received HSCT.

Eighteen of 20 patients (90%) in the fresh formulation study stopped immunoglobin replacement therapy (IgRT) after two years, compared to 52% of HSCT patients. Of the seven patients treated with the cryopreserved formulation with 18 months of follow-up, five had discontinued IgRT (71%), which is comparable to the 18-month data for patients treated with the fresh formulation.

Oral Presentation Details

Extensive Metabolic Correction of Hurler Disease by Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II TrialPublication Number: 607Session: 801. Gene Therapy and Transfer: Gene Therapies for Non-Malignant DisordersDate and time:Monday, December 9, 7:00am ET

Investigators will present updated analyses from the ongoing proof-of-concept trial of OTL-203 for mucopolysaccharidosis type I (MPS-I).

About ADA-SCID and OTL-101Severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID) is a rare, life-threatening, inherited disease of the immune system caused by mutations in the ADA gene resulting in a lack of, or minimal, immune system development.1-4The first symptoms of ADA-SCID typically manifest during infancy with recurrent severe bacterial, viral and fungal infections and overall failure to thrive, and without treatment the condition can be fatal within the first two years of life. The incidence of ADA-SCID is currently estimated to be one in 500,000 live births inthe United Statesand between one in 200,000 and one in 1 million inEurope.3OTL-101 is an autologous,ex vivo,hematopoietic stem cell-based gene therapy for the treatment of patients diagnosed with ADA-SCID being investigated in multiple clinical trials inthe United StatesandEurope, including a registrational trial at theUniversity of California, Los Angeles(UCLA). OTL-101 has received orphan drug designation from theU.S. Food and Drug Administration(FDA) and the European Medicines Agency (EMA) for the treatment of ADA-SCID, and Breakthrough Therapy Designation from theFDA.

About WAS and OTL-103Wiskott-Aldrich Syndrome (WAS) is a life-threatening inherited immune disorder characterized by autoimmunity and abnormal platelet function and manifests with recurrent, severe infections and severe bleeding episodes, which are the leading causes of death in this disease. Without treatment, the median survival for WAS patients is 14 years of age. Treatment with stem cell transplant carries significant risk of mortality and morbidities. OTL-103 is anex vivo,autologous, hematopoietic stem cell-based gene therapy developed for the treatment of WAS that Orchard acquired from GSK in April 2018 and has been developed at theSan Raffaele-Telethon Institute for Gene Therapy(SR-Tiget) inMilan, Italy. The global incidence of WAS is estimated to be about 100-260 births per year, with a global prevalence of 2,900-4,700 patients.

About MPS-I and OTL-203Mucopolysaccharidosis type I (MPS-I) is a rare inherited neurometabolic disease caused by a deficiency of the IDUA (alpha-L-iduronidase) lysosomal enzyme required to break down glycosaminoglycans (also known as GAGs or mucopolysaccharides). The accumulation of GAGs across multiple organ systems results in the symptoms of MPS-I including neurocognitive impairment, skeletal deformity, loss of vision and hearing, hydrocephalus, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births.5There are three subtypes of MPS-I; approximately 60 percent of MPS-I patients have the severe Hurler subtype and, when untreated, these patients rarely live past the age of 10.IdTreatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is anex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by theSan Raffaele-Telethon Institute for Gene TherapyinMilan, Italy.

About Orchard Orchard Therapeuticsis a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.

Orchards portfolio ofex vivo, autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis, a gammaretroviral vector-based gene therapy and the first such treatment approved by theEuropean Medicines Agencyfor severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. Strimvelis, as well as the programs in MLD, WAS and TDT were acquired by Orchard from GSK inApril 2018and originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene TherapyinMilan, Italyinitiated in 2010.

Orchard currently has offices in the UK and the U.S., including London, San Francisco and Boston.

Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, intends, projects, and future or similar expressions that are intended to identify forward-looking statements.Forward-looking statements include express or implied statements relating to, among other things, the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including the product candidate or candidates referred to in this release, the timing of announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, including any cryopreserved formulations of such product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved.Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards annual report on Form 20-F for the year endedDecember 31, 2018as filed with theU.S. Securities and Exchange Commission(SEC) onMarch 22, 2019, as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

1Orphanet. SCID due to ADA deficiency.2Whitmore KV, Gaspar HB. Front Immunol. 2016;7:314.3Kwan A, et al. JAMA. 2014;312:729-738.4Sauer AV, et al. Front Immunol. 2012;3:265. 5Beck et al. The Natural History of MPS I: Global Perspectives from the MPS I Registry. Genetics in Medicine 2014, 16(10), 759.

Contacts

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

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

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Orchard Therapeutics to Present New Registrational Data of Investigational Gene Therapies at the 61st American Society of Hematology Annual Meeting -...

St. Jude Childrens Research Hospital and the National Institutes of Health to present updated MB-107 clinical data for the treatment of X-linked severe combined immunodeficiency

Fred Hutchinson Cancer Research Center to present overview of ongoing MB-106 Phase 1/2 clinical trial

NEW YORK, Nov. 06, 2019 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, announced today that updated Phase 1/2 clinical data for MB-107 lentiviral gene therapy for X-linked severe combined immunodeficiency (XSCID) have been selected for oral and poster presentations at the 61st American Society of Hematology (ASH) Annual Meeting. ASH will be held December 7-10, 2019, at the Orange County Convention Center in Orlando, FL.

MB-107 is currently being assessed in two Phase 1/2 clinical trials for XSCID: the first in newly diagnosed infants under the age of two at St. Jude Childrens Research Hospital, UCSF Benioff Childrens Hospital and Seattle Childrens Hospital and the second in patients over the age of two who have received prior hematopoietic stem cell transplantation at the National Institutes of Health. Positive Phase 1/2 clinical data from the trial for infants under the age of two were published in the New England Journal of Medicine in April 2019 and positive Phase 1/2 clinical data from the trial in patients over the age of two were published in Science Translational Medicine in April 2016. The U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to MB-107 for the treatment of XSCID in August 2019.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, We are extremely pleased that additional clinical data on MB-107, a lentiviral gene therapy for the treatment of XSCID, will be presented in oral and poster sessions at the 2019 ASH Annual Meeting. The curative potential of MB-107 based on previously announced compelling Phase 1/2 data is impressive, and we look forward to working with St. Jude and NIH to advance the development of this important treatment option.

Details of the MB-107 presentations are as follows.

Oral Presentation:Title: Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined ImmunodeficiencySession: 801. Gene Therapy and Transfer: Gene Therapies for Non-Malignant DisordersAbstract Number: 608Date and Time: Monday, December 9, 2019, 7:15 a.m. ET Location: Orange County Convention Center, Valencia BC (W415BC)Presenter: Harry Malech, M.D., Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD, USA

Poster Presentation:Title: Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical StudySession: 801. Gene Therapy and Transfer: Poster IAbstract Number: 2058Date and Time: Saturday, December 7, 2019, 5:30-7:30 p.m. ETLocation: Orange County Convention Center, Hall BPresenter: Ewelina Mamcarz, M.D., Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Childrens Research Hospital, Memphis, TN, USA

In addition, Mustangs collaborator Fred Hutchinson Cancer Research Center will present a poster about the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 CD20-targeted CAR T for high-risk B-cell non-Hodgkin lymphomas.

Details of the MB-106 presentation are as follows.

Poster Presentation:Title: CD20 Targeted CAR-T for High-Risk B-Cell Non-Hodgkin LymphomasSession: 704. Immunotherapies: Poster IIAbstract Number: 3235 Date and Time: Sunday, December 8, 2019, 6-8 p.m. ETLocation: Orange County Convention Center, Hall BPresenter: Mazyar Shadman, M.D., M.P.H., Fred Hutchinson Cancer Research Center, Seattle, WA, USA

Copies of the abstracts can be viewed online through the ASH website at http://www.hematology.org.

About Mustang BioMustang Bio, Inc. (Mustang) is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T and CRISPR/Cas9-enhanced CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission. Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking Statements This press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 940-0135tplohoros@6degreespr.com

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Mustang Bio Announces MB-107 Lentiviral Gene Therapy and MB-106 CD20-Targeted CAR T Data Selected for Presentations at 61st American Society of...

UniQure has started screening patients for its phase 1/2 trial of gene therapy AMT-130 for Huntingtons disease, and says it hopes to start treating the first subject in late 2019 or early 2020.

The start of the trial will give the Dutch biotech a second gene therapy in clinical trials to go along with AMT-061 (etranacogene dezaparvovec), its one-shot therapy for haemophilia B which is in phase 3 testing.

Huntingtons disease is a rare, devastating neurodegenerative genetic disorder that affects motor function and causes severe cognitive decline, eventually leading to total physical and mental deterioration.

The disease is caused by a mutation in the gene coding for huntingtin which causes the formation of an abnormally long and unstable form of the protein that is chopped up by cellular repair mechanisms into smaller, toxic fragments.

AMT-130 consists of an adeno-associated virus (AAV) vector carrying a micro-RNA that is designed to switch off the huntingtin gene and prevent it from producing the mutant form of the protein.

In annual models, a single dose of AMT-130 was shown to reduce huntingtin levels, initially in deep structures of the brain like the striatum that are affected first by the disease and spreading to higher structures such as the cerebral cortex that come into play later in the course of Huntingtons.

The phase 1/2 trial will be conducted in around 26 patients at several clinical sites, who will be treated either with a single dose of the gene therapy directly into the striatum or an imitation procedure with no drug.

The main outcome measures will be safety and the persistence of AMT-130 in the brain, but the trial will look at clinical outcomes including motor, cognition, and behavioural function over a five-year period. First results should be available in 2022.

Other companies notably Wave Life Sciences/Takeda and Ionis/Roche are developing antisense drugs to switch off production of huntingtin, but these would require continuous dosing in order to be effective.

The announcement was made in UniQures third-quarter results update, at which it also said it had completed enrolment of 62 patients into its HOPE-B trial of haemophilia B therapy AMT-061, setting it on course for a readout in 2020 and possible filing in early 2021.

In July, UniQure reported phase 2b results with AMT-061 showing that it could restore Factor IX levels into the normal range for two out of three subjects.

UniQure was the first company to launch a gene therapy onto the market in Europe, introducing Glybera (alipogene tiparvovec) for familial lipoprotein lipase deficiency (LPLD) in 2012, but the product was a commercial flop and was withdrawn from sale in 2017.

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UniQure presses go on Huntington's gene therapy trial - - pharmaphorum

It's a very early report, from just two patients, only a few months after treatment. But UMass Medical School Dean Terence Flotte this week shared at a conference what could be landmark news about a terrible genetic disease: Two young patients with Tay-Sachs disease showed no ill effects from a new gene therapy that aims to correct the defect at the heart of the disease.

One of them, treated at just 7 months, has appeared to stabilize instead of following the typical quick slide toward death by age 4.

"It seems right now that she's not degenerating," Flotte said. "But I would say it's too early to say that definitively."

Tay-Sachs is a fatal disorder that tends to affect babies of Eastern-European Jewish ancestry, along with other ethnicities including Cajun and Irish. They usually seem to develop normally for the first few months, but as the disease kills off their nerve cells, they lose the ability to move or breathe on their own.

Flotte says the brain MRI of the baby treated at 7 months looks encouraging, and a clinical trial in more than a dozen patients is expected to begin soon.

Edited highlights of our conversation follow.

You've just presented at a gene therapy conference. What did you report?

We reported the first two patients ever treated with gene therapy for Tay-Sachs disease two infants treated at UMass Memorial Medical Center. What we presented was that these two patients were both treated safely. The vector[the engineered virus that delivered the genetic fix] was administered directly into the brain.

We saw bio-activity, which basically means that we partially restored the enzyme that is missing in Tay-Sachs disease. And the patients were able to tolerate that safely. Also, in one of the cases, with the patient treated early in the course of the disease, we've seen some stabilization of the patient's condition.

What do you mean by stabilization?

One of the patients was treated at 2-1/2 years of age, and that patient had really advanced disease. And we've seen the biochemical effect, but really no clinical effect.

The second patient was treated between 6 and 7 months of age, and in that patient, it appears, although it's still very early, that the patient may be having some continued preservation of her ability to sit up and control her muscles. She's basically seeming to have a more gradual progression at the current time, really being stable at a time point when we might be expecting her to lose some of these developmental milestones.

The best way to explain it is that if a normal infant begins to sit up at around six months of age, Tay-Sachs babies do that, but then they tend to lose the ability to sit up some time between 10 months of age and maybe 15 months of age. The last time we assessed the patient, at 10 months of age (and she's now close to 12 months of age), she seems to not be losing any of the strength required to sit up. We have her older siblings for comparison, and it's encouraging that she seems to be progressing less than they did. We also saw some encouraging signs on her brain MRI.

It seems right now that she's not degenerating. But I would say it's too early to say that definitively. If you think about the progression of development as the slope of a line, the line is flat at this point. It's not going up or going down. The next assessment will be very important, to see whether she's continuing to be flat, which would be a major benefit, or whether she's regressing but just a little bit more slowly.

When you say flat, she's also not advancing as a typical child would?

That is right. It looks like preservation of function rather than gaining. But her oldest sibling died before his third birthday. So considering how fast these patients can decline, a preservation or stabilization could be very important.

It's important to note, too, that we are just at the very beginning. The first patient got the vector injected just into the fluid around the brain, the cerebro-spinal fluid, not into the brain tissue. The second patient got a portion of it injected into the thalamus, which projects out to the entire brain tissue. It's kind of the relay center of the brain, and it can actually ship enzyme out all over the brain.

No one's ever tried that in a humans before, so that was really an important milestone, that intra-thalamic injection. As the trials progress, a larger dose will be injected into the thalamus.

Why has there never been an injection into the thalamus in humans before? What's the challenge?

One challenge is that it is a completely irreplaceable structure. Effectively, all motor and sensory function relays through the thalamus. So if you were to have bleeding or injury to the thalamus, it could cause a stroke or a persistent pain syndrome. So it is somewhat risky. On the other hand, when you're dealing with the infantile form of Tay-Sachs, it's so tragic that it warrants a rather risky approach.

It's been done many times in animals, but this was the first time doing it in patients.

What's next? A full clinical trial?

Yes, Axovant has licensed the program. This first program was done all at UMass Medical School and UMass Memorial Medical Center, and the program is now licensed to Axovant, and they are planning in the near future to do a Phase 2 trial, which we will still be involved in.

It will entail increasing the proportion of the vector injected into the thalamus, so that we will get to the exact proportional dose that was used to correct all of the different animal models that have been treated: a mouse, a sheep and a cat model.

UMassMed Magazine has more on the school's Tay-Sachs gene therapy work here.

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Early Report: Baby Treated With Gene Therapy For Deadly Tay-Sachs Disease Appears To Stabilize - WBUR

PTC Therapeutics gene therapy candidate PTC-AADC (formerly AGIL-AADC) provided clinically meaningful and sustained improvements in motor, cognitive, and language milestones in children with aromaticl-amino acid decarboxylase (AADC) deficiency up to five years following the one-time treatment, trial analyses show.

A single dose of PTC-AADC delivered into the brain lowered the number of oculogyric crises (involuntary upward eye movement) and recovered childrens weight, as well as improved their ability to sit, walk, and talk over a five-year period.

PTC Therapeutics will request marketing approval soon, with plans to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration later this year.

The new findings were shared at the 48th Annual Meeting of the Child Neurology Society (CNS), held recently inCharlotte, NC. Data were presented in two posters titled AGIL-AADC gene therapy results in sustained improvements in motor and developmental milestones through 5 years in children with AADC deficiency (page S136), and Safety and Improved Efficacy Outcomes in Children With AADC Deficiency Treated with AGIL-AADC Gene Therapy: Results From Three Clinical Trials (page S148).

We are excited to see the transformational effects in AADC deficiency patients in this long-term study as patients with severe AADC deficiency never achieve the ability to sit, walk or talk, Stuart Peltz, PhD, PTC Therapeutics CEO, said in a news release.

We are on track to submit a BLA to the FDA by the end of the year and are proud to be on the verge of bringing the first commercial treatment for AADC deficiency patients which is in line with our mission of bringing clinically differentiated treatments to patients with rare disorders, he added.

PTC-AADC is an investigationalgene therapy designed to deliver a healthy copy of the DDCgene the faulty gene in patients with AADC deficiency to nerve cells. The goal is to restore the production of AADC enzyme which is missing because of this genetic defect and counter the symptoms caused by this deficiency.

A working copy of DDC is passed on to cells through an adeno-associated virus that is modified to be non-infectious.

The gene therapy is injected via a surgical procedure into an area of the brain called the putamen. This region is crucial for producing chemical messengers (neurotransmitters) such as dopamine and serotonin, which are involved in movement control but fail to be produced in patients with the disease.

In one of its presentations, PTC Therapeutics provided the most extensive study of PTC-AADCs efficacy and safety to date. It conducted a joint analysis of three open-label clinical trials, which together enrolled 26 children with AADC deficiency, ranging in age from 21 months to 8.5 years.

At the beginning of these studies, children had no full head control and were unable to sit, stand, or walk. They were given a single dose of PTC-AADC (total dose, 1.81011 vector genomes, vg) which was injected into the patients putamen during a single surgery session.

One year after treatment, the patients mean body weight had increased from 12.0 kg to 15.2 kg, and there was a reduction in the frequency of involuntary upward eye movements characteristic of the disease (oculogyric crises).

Dyskinesia (uncontrolled erratic movements) was a common adverse event, affecting 23 of 26 patients, but most events were mild or moderate in severity, and all cases had resolved within 10 months from dosing.

In addition to failing to reach key developmental milestones, such as walking and talking, children with AADC deficiency can experience severe symptoms that affect their everyday lives. These symptoms can include episodes of oculogyric crises, which can last for minutes or hours and involve sustained upward movement of the eyes, involuntary movements of the neck, tongue protrusions and jaw spasms, which can be very distressing for patients and their families, said Claudio Santos, MD, senior vice president of global medical affairs at PTC Therapeutics.

The post-treatment data presented at CNS confirm reductions in the number of patients experiencing oculogyric crises, suggesting that this gene therapy treatment has the potential to make a real difference in the lives of patients with AADC deficiency, he added.

A second analysis demonstrated that PTC-AADCs benefits can hold up to five years after treatment, the longest data available for any investigational therapy for AADC deficiency.

The findings came from the latest follow-up data of two open-label clinical studies: AADC-1601 (NCT02926066), a trial in which patients were enrolled under individual compassionate use consents, and AADC-010 (NCT01395641).

Together, the studies enrolled 18 patients who were 21 months to 8.5 years old. None had full head control or could sit unassisted or stand. In this update, all patients had two years of follow-up data, and eight of these patients had five years of post-treatment data.

Prior results shared by PTC Therapeutics showed that at two years, eight patients (44%) had achieved full head control, six (35%) were able to sit unassisted, and three (17%) could stand without support. Among the eight patients followed for five or more years, four (50%) had full head control, four (50%) could sit unassisted, and two (25%) could stand without support.

The latest results continue to support these meaningful improvements in motor, cognitive, and language skills, and importantly, show that effects from a single dose of PTC-AADC can last at least five years post-treatment.

In addition, all treated patients continued to demonstrate sustained production of dopamine in the body, one of the neurotransmitters missing in patients with AADC deficiency.

No new safety signals were observed during these long-term evaluations.

Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.

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AADC Improvements Sustained with Gene Therapy PTC-AADC, Data Say - AADC News

The U.S. National Institutes of Health (NIH) wants to cure HIV and sickle cell disease with gene therapies, and will invest $100 million over the next four years towards that goal, the agency announced today (Oct. 23).

For this effort, the NIH will partner with The Bill & Melinda Gates Foundation, which will also invest $100 million.

Critically, the partnership aims to make the therapies affordable and accessible to people around the world, particularly in developing countries, where the burden of these diseases is greatest.

"This is a very bold goal, but we have decided to go big," Dr. Francis Collins, director of the NIH, said in a news conference today.

The effort aims to have the therapies ready for testing in clinical trials in the U.S. and sub-Saharan Africa within the next seven to 10 years.

Related: 10 Amazing Things Scientists Just Did with CRISPR

The majority of the 38 million people with HIV live in developing countries, with two-thirds living in Sub-Saharan Africa. For sickle cell disease, the majority of cases also occur in Sub-Saharan Africa.

The NIH has been trying to find a cure for HIV for "decades and decades," said Dr. Anthony Fauci, director of The National Institute of Allergy and Infectious Diseases. Although current treatments with antiretroviral therapy (ART) are effective at suppressing the virus in the body, they are not a cure, and must be taken everyday. What's more, there are millions of people with HIV who don't have access to ART treatment.

Although scientists are working to develop gene-based cures for HIV, these approaches are often costly and would be difficult to implement on a large scale, Fauci said. For example, some of these therapies take cells out of a patient's body and then re-infuse them, an expensive and time-consuming intervention.

For this reason, the new collaboration will focus on developing cures that use "in vivo" approaches, meaning they happen inside the body, Fauci said. One example of this could be to remove the gene for the CCR5 receptor, which HIV uses to get inside cells. Another idea is to excise the HIV "proviral" DNA that has copied itself into the human genome and lurks in the body even after years of treatment.

Similarly, for sickle cell disease, the goal would be to develop an in vivo therapy that could repair the genetic mutation that causes the disease. This would require a gene-based delivery system that could selectively target the mutation.

"Beating these diseases will take new thinking and long-term commitment. I'm very pleased to see the innovative collaboration announced today, which has a chance to help tackle two of Africa's greatest public health challenges," Matshidiso Rebecca Moeti, the World Health Organization's Regional Director for Africa, said in a statement.

Still, much work would be needed to make sure these therapies are safe and effective.

"It is very clear we have a ways to go, which is why this is a 10 year effort to try and take that promise and turn it into a reality," Collins said.

Earlier this year, the Trump Administration announced a plan to end the HIV epidemic in the U.S. in 10 years.

Originally published on Live Science.

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Can Gene Therapy Cure HIV? US Gov't. Is Banking $100 Million On It. - Livescience.com

The National Institutes of Health (NIH) announced a partnership with the Bill and Melinda Gates Foundation on Wednesday to fund the development of targeted cures for HIV and sickle cell disease with a view to helping people in developing countries using gene therapy. With most of the populations affected by each disease residing in sub-Saharan Africa, treatments are being sought with regional conditions in mind.

The NIH and the Gates Foundation are investing $100 million in the initiative to develop low-cost gene therapies. The announcement follows President Donald Trump's pledge in his 2019 State of the Union address that the United States would eradicate HIV within the next decade. The Trump administration has also tried to draw more attention to sickle cell disease (SCD) in the past few years, according to a press release from NIH.

Sickle cell disease is a blood disorder that can cause anything from mild pain to heart failure. Human immunodeficiency virus (HIV) is a communicable disease that, if left untreated, wipes out the immune system. People with SCD inherit the disease from their parents, whereas HIV is acquired through blood contamination with certain bodily fluids of an infected person. While the mechanisms of transmission are different, both diseases are carried in the genome of infected individuals. Globally, both diseases also disproportionately impact individuals in lower-income communitiesand scientists believe that both could be combatted with gene-based treatments.

The past few years have seen unprecedented strides toward cures for these two diseases using gene therapy, which the NIH defines as experimental technique wherein doctors insert genes into a patient's cells so their body can more effectively resist a disease. It can include inserting a healthy variant of a gene to replace the unhealthy copy that causes a disease, or placing an entirely new gene in the body to fight the disease.

"Dramatic advances in genetics over the last decade have made effective gene-based treatments a reality... Yet these breakthroughs are largely inaccessible to most of the world by virtue of the complexity and cost of treatment requirements, which currently limit their administration to hospitals in wealthy countries," the press release states. The new initiative will focus on developing treatments that can be delivered in "low-resource settings."

Speaking on the initiative's viability, Dr. Ronald Mitsuyasu, a professor of medicine in hematology-oncology at the University of California, Los Angeles with more than 25 years of experience in HIV clinical trials research, told Newsweek that this sort of solution has been attempted in the past, but gene therapy hasn't yet proved successful in treating HIV.

"There have been several attempts to use gene therapy for HIV by either incorporating genes that suppress HIV genes, producing decoys for various viruses required processes needed for viral replication, or substituting inactive genes for functional genes of HIV," he said.

But those living in developing countries have not had as many chances to benefit from these solutions as those living in places like the U.S., according to the press release.

"SCD and HIV are major burdens on health in low-resource communities around the world," the press release read. "Approximately 95% of the 38 million people living with HIV globally are in the developing world, with 67% in sub-Saharan Africa, half of whom are living untreated. Fifteen million babies will be born with SCD globally over the next 30 years, with about 75% of those births occurring in sub-Saharan Africa."

Further, the prediction indicates that three-quarters of those infants will be born into low-income countries and communities. Between 50 and 90 percent of babies born with the disease in sub-Saharan African countries will die before the age of five, according to the release.

So, the NIH and the Gates Foundation's initiative aims to identify potential cures for both diseases as well as partner with groups in Africa to identify candidates on whom these new cures can be tested.

We are losing too much of Africa's future to sickle cell disease and HIV. Beating these diseases will take new thinking and long-term commitment. I'm very pleased to see the innovative collaboration announced today, which has a chance to help tackle two of Africa's greatest public health challenges." Matshidiso Rebecca Moeti, M.B.B.S., the World Health Organization's regional director for Africa said of the initiative.

Mitsuyasu said he agreed that continued investigation into gene-based cures would eventually yield worthwhile results. "I personally believe that it should be possible to ultimately develop a gene therapy approach to overcome ... HIV," Mitsuyasu said. "Continued scientific developments in the field of gene therapy will eventually allow for the conquest of most genetic and viral gene integrated diseases."

See more here:
Gates Foundation, NIH Bet on Gene Therapy To Bring Cheap HIV and Sickle Cell Cures to Sub-Saharan Africa - Newsweek

Gene therapy is a promising therapeutic procedure for genetic disorders or diseases in which defective genes are corrected, replaced, or inactivated.

In the case of adrenoleukodystrophy (ALD) a genetic disorder caused by mutations in the ABCD1 gene that damages the myelin sheath around nerve cells gene therapy may benefit patients prior to the onset, or during the early stages, of the disease by stopping the progression of demyelination. However, the therapy cannot be beneficial after the disease has worsened significantly.

Gene therapy works by introducing the correct gene sequence into cells. Since genetic material cannot enter the cell on its own, the correct gene sequence needs to be delivered using a vector. This vector can be a modified virus that has been engineered to remove its pathogenic genetic material so that it cannot cause disease, but is still able to transfer the correct gene sequence to the host cell.

The vector can be directly injected into the patients body or into host cells grown in the laboratory and then transplanted back into the patient. Upon successful viral transfer, the host cell should be able to produce the functional protein.

In ALD, the clinician first takes out the patients own stem cells (autologous) and then inserts the correctABCD1 gene sequence into these cells using a viral vector in the laboratory. The corrected stem cells that are able to produce the functional ALD protein are then implanted back into the patients body so they may develop into nerve cells in the brain. Since the patients own cells are being used, there are fewer risks than when donor stem cells are used.

Lenti-D,an investigational gene therapy developed by Bluebird Biois currently being studied in a Phase 2/3 clinical trial (NCT01896102) in the U.S., the U.K., and France. The study aims to evaluate the safety and effectiveness of Lenti-D in boys, up to 17 years old who havecerebral adrenoleukodystrophy (CALD). Based on the preliminary data from this study,the U.S. Food and Drug Administration (FDA)designated Lenti-D a breakthrough therapy for the treatment of CALD in May 2018.

A Phase 1/2 clinical trial (NCT02559830) is recruiting patients with ALD at the Shenzhen Second Peoples Hospital in Guangdong, China. The study aims to assess the safety and effectiveness of transplanting patient-derived bone marrow stem cells, which have been genetically-corrected using a lentiviral vector, for the treatment of ALD.

Another Phase 1/2 clinical trial (NCT03727555) at the Shenzhen Geno-Immune Medical Institute also in Guangdong, China is recruiting 10 patients with ALD. The study aims to evaluate the safety and effectiveness of a lentiviral vector carrying the healthy ABCD1 gene (TYF-ABCD1) injected directly into the patients brain for the treatment of ALD.

***

Adrenoleukodystrophy News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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zge has a MSc. in Molecular Genetics from the University of Leicester and a PhD in Developmental Biology from Queen Mary University of London. She worked as a Post-doctoral Research Associate at the University of Leicester for six years in the field of Behavioural Neurology before moving into science communication. She worked as the Research Communication Officer at a London based charity for almost two years.

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Gene Therapy - Adrenoleukodystrophy News

Sigmund Freud never uttered the word neuroscience. Neither did Santiago Ramn y Cajal. It was biophysicist Francis Schmitt who grafted neuro with science in 1962 when he established the Neurosciences Research Program at MIT. The new moniker was intended to encompass a merging of relevant neuro disciplines, ranging as far afield as physiology, psychology, immunology, physics and chemistry.

Brains and behaviors have been scrutinized for millennia. But as psychology blogger Vaughn Bell has pointed out, the 1960s marked a shift in perspective. Neuroscience was the formal name given by Schmitt. But the period represented the beginnings of a neuroculture, that put brain science on a pedestal even leading to the familiar meme proclaiming my brain made me do it. One example was rooted in pharmaceutical companies development of psychiatric drugs that resulted in their investing millions both into divining the neurochemistry of experience and into massive marketing campaigns that linked brain functions to the psyche, Bell notes.

The field received an adrenaline boost precisely 50 years ago with the founding of the Society for Neuroscience, allowing Schmitts collaborative vision to be globally shared. SFNs first annual meeting in 1971 drew 1,395 attendees to Washington, D.C. This years wrapped up on October 23, bringing more than 27,500 to Chicagoand the annual numbers have occasionally topped 30,000. SFN now boasts 37,000 members from more than 95 countries.

Anything to do with the topic brain found a place among the more than 14,500 abstracts of unpublished work presented in 2019 on themes ranging from the mechanisms of sleep to cocaine craving. But the society has had to adapt its U.S.-based get-together this year to respond to a world of closing national borders.

Some members were unable to get visas to enter the U.S., in part because of the U.S. travel ban, which includes broad restrictions on visits from Iran,Libya,Syria,Yemen,Somalia,North KoreaandVenezuela. In response, SFN initiated a program called Science Knows No Borders in which would-be attendees had a PDF printed out and posted or else PowerPoints and a recorded talk proferred in their absence. An Iranian doctoral student, Shahrzad Ghazisaedi, from University of Toronto, a neuropathic pain researcher, was one among about a dozen people who took advantage of the program (not all of them necessarily subject to the travel ban). Her poster entitled Sex specific DNA methylation pattern in spinal cord and periaqueductal gray (PAG) before and after peripheral injury could be seen Monday afternoon by attendees during a session entitled Central Nervous System Mechanisms in Pain.

For those who actually were able to make it, a range of topics caught the eye: research on nervous system immune cells implicated in a range of disorders, a gene therapy for converting the brains support cells to neurons for treating Alzheimers, a prosthetic forearm that provides a sense of touch and synchronization of brain waves between two people holding hands. Also, a group of scientists got together to start planning a test in humans to determine which of two theories of consciousness is more likely to be correct.

Another theme that stood out was the challenge of working with miniaturized brain facsimiles, called organoids, that show promise of more faithfully replicating what goes on in the human organ than a mouse brain can. Organoids, though, are too close to a Mary Shelley creation for some people. At the conference, members of the Green Neuroscience Laboratory in San Diego called for a research moratorium on organoid tissue implanted into mice or other animals, a technique already in use. In an abstract for their talk, they ventured that the technology is perilously close to crossing an ethical Rubicon in which organoids may experience sentient activity and behavior. The group advocates that methods should be developed to ascertain whether any given organoid has the ability to sense and react to its surroundings.

At a press conference of scientists who grow the five-millimeter-diameter mini brains, ethical debate was welcomed, but the researchers also tried to place their work in context. Paola Arlotta from Harvard showed a video of organoids, at most the size of small peasnothing resembled an imagined brain-in-a-dish. The brain bits are also difficult to work withchallenging to grow reproducibly and their cells do not mature to become an exact replica of human cells, but instead end up with a confused identity. Researchers think the kinks can be worked out, but, even then, that may not pave the way for growing full-sized organs.

Most scientists are not interested in figuring out how to grow a human brain in a dish, says Arnold Kriegstein, of the University of California, San Francisco. They are more interested in a particular disease mechanism or a certain process they want to study. And that really requires a reductionist system. It's too complicated to study an intact human brain. What you really want are the important elements, which you can dissect and delve into in great detail in the laboratory.

Everyone agreed that discussion about mini-brain ethics is warranted. But as far as existential threats, tiny tissue nuggets run amok may not be at the top of a list that includes antibiotic resistance, climate change and self-driving cars engineered with internals that produce a loss of control that resembles a wayward 737 MAX.

Read the original here:
Society for Neuroscience at 50 Delves into Mini Brains, Gene Therapy, Prosthetics and All Else Related to Our Three-Pound Wonder - Scientific American

Dive Brief:

The EMA's green light for Bluebird's manufacturing removes a final hurdle standing in the way of marketing the gene therapy, which costs $1.8 million per patient.

A requirement from European authorities to narrow drug product specifications for Zynteglo forced the company to delay the gene therapy's launch later than when Wall Street analysts had expected.

In a statement, Apceth said it's ready for the challenge to bring Bluebird's treatment to market. Between 2,000 and 3,000 patients in the European Union would be eligible under the conditions approved by regulators for Zynteglo's use.

Bluebird has cautioned investors to take a long view of the new treatment's prospects, and to expect a slow start. In addition to winning approval for the new manufacturing specifications, Bluebird has to navigate through healthcare systems that aren't used to paying large sums for a one-time treatment.

In hopes to alleviating those problems, the company has offered an installment plan that would require later payments only if the treatment continues to benefit patients. The hope is that Zynteglo saves healthcare dollars by sparing beta-thalassemia patients the need for regular blood transfusions and the complications that can go along with them.

Patients with the blood disorder carry a genetic mutation that hinders the body from effectively producing the crucial oxygen-carrying protein hemoglobin. As a result, they often require transfusions every two to five weeks to fight anemia.

"This is one step along the commercial journey as we advance our ongoing launch and market access activities on a country-by-country basis," said Alison Finger, Bluebird's chief commercial officer, in the company's statement on the EMA's nod.

In a September company presentation, Bluebird said it wants to make sure to "get the model right" as it looks toward future gene therapies it's developing in its pipeline. The company is initially planning to offer Zynteglo through treatment centers in Germany, Italy, the U.K. and France, with a drug manufacturing facility in Munich.

Read more:
Bluebird gets European green light for gene therapy production - BioPharma Dive

A gene therapy for a rare blood disorder has shown what the manufacturer calls the first evidence of long-term improvement associated with the disease.

New York-based Rocket Pharmaceuticals said Thursday that it had presented long-term follow-up data from the Phase I/II study of RP-L102, its gene therapy for Fanconi anemia, at the annual congress of the European Society of Cell and Gene Therapy in Barcelona, Spain. The company said it represented the first evidence of long-term improvement and stabilization in blood counts and durable mosaicism among patients who received the therapy without the use of the conditioning regimens normally used for allogeneic stem cell transplants, which the company calls Process A.

Shares of Rocket were up slightly on the Nasdaq following the news. RP-L102 is a lentiviral vector-based gene therapy. Most other gene therapies in development, and both of the currently marketed ones Spark Therapeutics Luxturna (voretigene neparvovec-rzyl) and Novartis Zolgensma (onasemnogene abeparvovec-xioi) are adeno-associated viral vector-based.

According to the data, representing four of nine patients, there were improved blood counts and long-term bone marrow mitomycin C (MMC) resistance, thereby indicating durable phenotypic correction. The data met or exceeded a 10 percent threshold that the company said the Food and Drug Administration and European Medicines Agency had agreed to for its upcoming Phase II registration study, for which it plans to start enrolling patients by the end of the year.

FA is a rare, genetic bone marrow failure disorder, half of whose patients are diagnosed before the age of 10, while about 10 percent of patients are diagnosed as adults, according to the National Organization for Rare Disorders. It is often associated with progressive deficiency of production of red and white blood cells and platelets in the bone marrow and can eventually lead to certain solid and liquid tumor cancers. It occurs in 1-in-136,000 births and is more common among Ashkenazi Jews, Spanish Roma and black South Africans.

These results indicate the feasibility of engraftment in FA patients using autologous, gene corrected [hematopoietic stem cells] in the absence of any conditioning regimen, said Dr. Juan Bueren, scientific director of the FA gene therapy program at Spains Center for Energy, Environmental and Technological Research, in a statement. This indicates the potential of this therapeutic approach as a definitive hematologic treatment, while avoiding the burdensome side effects associated with allogeneic transplant, including the risk of post-transplant mortality and a substantially higher risk of head and neck cancer.

Photo: virusowy, Getty Images

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Rocket's gene therapy shows long-term efficacy in rare blood disorder - MedCity News

In Junedata on three subjects in a phase II trial of Krystal Biotechsepidermolysis bullosa gene therapy sent the group's share price up 41%. Today a final update from the trial, including results from two new patients, left investors unmoved.

This might have something to do with the updated data showing that two lesions that had been successfully treated with Krystals therapy at 90 days had reopened a month later, raising questions about whether the treatment works in the long term.

The two new patients with severe recessive dystrophic epidermolysis bullosa (RDEB) had beenadded to the trial in June 2019, after the initial data release(Krystal plays down dropout to claim a mid-stage win, June 25, 2019). Bercolagene telserpavec or B-Vec for short, formerly called KB103, was administered to one wound on each patient every other day for two weeks, or until the wound closed completely. The other wound was treated with a placebo gel.

This differs slightly from the earlier phase II patients, who had B-Vec administered to two wounds each and placebo to a third.In its press release Krystal trumpeted that, of all 10 wounds treated in the phase I and phase II trials, nine had healed at 90 days.

The unhealed 90-day lesion was a chronic wound, reported to have been open for over four years, in one of the patients in the first phase II cohort. It was still only 42% closed at 90 days following the initial administration of B-Vec. The wound was re-dosed with B-Vec approximately three and a half months later, and healed within a week of this second dose.

Reopening

But the company glossed over the fact that, at four months, two of the healed B-Vec-treated wounds in cohort 1 had reopened: at 120 daysa lesion on one patients back had returned to only 77% closure, and another patient had a lesion on their left upper arm return to 85% closure.

Moreover, one of the placebo-treated wounds that had not healed at 90 days did heal at 120. At the four-month point, across both phase II cohorts, the success rates are 63% for B-Vec versus 20% for placebo not quite emphatic as the earlier 90% versus 14%.

One question iswhich time point is the more important? On clinicaltrials.gov the primary endpoint cutoff is listed as wound healing at 24 weeks a completely different point, and one that has not yet been reached, despite Krystals statement that this would be the final update from the phase II trial.

EB is a cyclical disease. Wounds open, close and reopen in the natural course of the condition even without treatment, so it can be tricky to show a drugs effect. Krystal intends to move B-Vec into phase III, and investors might want to take careful note of the time point regulators pick for the primary endpoint.

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Gene therapys duration is less than Krystal clear - Vantage

During the third-quarter financial call, Vasant Narasimhan, CEO of Novartis, noted that questions from European and Japanese regulators regarding chemistry, manufacturing and controls (CMC) were behind expected decision dates on Zolgensma (onasemnogene abeparvovec) being pushed back into 2020.

At present, the company expects to receive opinions from the European Medicines Agency (EMA) and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) in the first quarter and the first half of 2020, respectively.

Narasimhan revealed few other details regarding the questions, only that there were an extensive set of questions with respect to manufacturing, to which it had submitted responses. Reuters stated that he also confirmed that the decision delay was not due to the revelation of data manipulation in August.

Despite the setback on potential approval date, the company was able to confirm that the product had achieved US sales of $160m (143m), arriving higher than analyst predictions of $98m (88m).

When questioned on the patient numbers this related to, on paid programs, Narasimhan confirmed that approximately 100 patients had been treated though other patients had received the gene therapy through treatment in clinical trials.

Once approved in elsewhere in the world, Narasimhan predicted such number could increase rapidly: I think in some countries in Europe, as well in the Middle East, there could be very strong demand coming very quickly after approval.

He cited pent-up demand as a reason that sales would increase quickly, and also pointed to early access programs being made available in France, Portugal and Germany as another positive long-term sign for the product.

The company will need to see substantial return on the product, after investing $8.7bn in the AveXis acquisition to gain access to the technology.

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Novartis gene therapy held up by manufacturing questions - BioPharma-Reporter.com

Cobra Moradian, Fatemeh Rahbarizadeh

Department of Medical Biotechnology, Faculty of Medical Sciences, Tarbiat Modares University, Tehran, Iran

Correspondence: Fatemeh RahbarizadehDepartment of Medical Biotechnology, Faculty of Medical Sciences, Tarbiat Modares University, Jalal AleAhmad Highway, Tehran 14115-111, IranTel +98 21 82883884Fax +98 21 82884555Email rahbarif@modares.ac.ir

Background: Breast cancer stem cells (BCSCs) are cells with a higher ability to metastasis and resistance to conventional treatments. They have a phenotype of (CD44high/CD24low) and the unlimited ability for proliferation. Development of strategies to target the BCSC population may lead to the establishment of more effective cancer therapies. Pseudomonas exotoxin A (PE) is a potent cytotoxic protein. CXCR1 promoter provides BCSC and HER2 specificity on transcription level. 5UTR of the basic fibroblast growth factor-2 (bFGF 5UTR) provides tumor specificity on translation level. Here, we utilized a mutant form of PE encoding DNA PE38, CXCR1 promoter and bFGF 5UTR to target BCSCs.Methods: The stemness of SK-BR-3, MDA-MB-231 and MCF10A cell lines were evaluated based on the expression of the CD44high/CD24low stem cell signature and the ability to form mammospheres. Then, the cell lines were transfected with constructs encoding luciferase/PE38 under the control of the CMV/CXCR1 promoter with or without bFGF 5UTR. Luciferase protein expression was evaluated using dual-luciferase reporter assay. PE38 transcript expression was measured by real-time PCR, and the cytotoxic effect of PE38 protein expression was determined by MTT assay.Results: The percentage of CD44high/CD24low population did not correlate to mammosphere forming efficiency (MFE). Given that the percentage of CD44 high/CD24 low is not a conclusive BCSC profile, we based our work on the mammosphere assay. However, in comparison with MCF10A, the two tumorigenic cell lines had higher MFE, probably due to their higher BCSC content. Reporter assay and real-time PCR results demonstrated that CXCR1 promoter combined with bFGF 5UTR increased BCSC-specific gene expression. Meanwhile, tightly regulated expression of PE38 using these two gene regulatory elements resulted in high levels of cell death in the two tumorigenic cell lines while having little toxicity toward normal MCF10A.Conclusion: Our data show that PE38, CXCR1 promoter and bFGF 5UTR in combination can be considered as a promising tool for killer gene therapy of breast cancer.

Keywords: breast cancer stem cell, PE38, CXCR1 promoter, bFGF-2, HER2, mammosphere

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Targeted Toxin Gene Therapy Of Breast Cancer Stem Cells Using CXCR1 Pr | OTT - Dove Medical Press

PTC Therapeutics has presented new long-term outcome data from its investigational gene therapy, PTC-AADC, in patients living with aromatic L-amino acid decarboxylase (AADC) deficiency.

The one-time gene therapy was found to give patients the ability to sit, walk, and talk, from data representative of up to five years of follow up post-treatment.

The new analysis evaluated outcomes of 26 patients with AADC deficiency across three separate clinical trials, and found that 12 months post-treatment with PTC-AADC, patients mean body weight had increased from 12.0 kg to 15.2 kg, and the frequency of oculogyric crises (involuntary upward eye movement) was reduced.

The data, presented at the Child Neurology Society 48th Annual Meeting, is a result of the most comprehensive analysis of patients treated with PTC-AADC to date.

Unfortunately, there are currently no approved therapies that address the underlying cause, and as such patients with severe AADC deficiency have a high risk of death during childhood.

The company is excited to see the transformational effects in AADC deficiency patients in this long-term study as patients with severe AADC deficiency never achieve the ability to sit, walk or talk, said Stuart Peltz, chief executive officer of PTC Therapeutics.

He also announced that PTC is on track to submit a biologics license application (BLA) to the FDA by the end of the year and are proud to be on the verge of bringing the first commercial treatment for AADC deficiency patients, which is in line with our mission of bringing clinically differentiated treatments to patients with rare disorders.

AADC deficiency is a rare genetic condition caused by a mutation in the dopa decarboxylase (DDC) gene, resulting in a lack of functioning AADC enzyme, which is responsible for the final step in the synthesis of key neurotransmitters dopamine and serotonin.

It results in delays or failure to reach developmental milestones such as head control, sitting, standing, walking, or talking, low muscle tone, severe, seizure-like episodes involving involuntary eye movement, autonomic abnormalities, and the need for life-long care.

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Investigational gene therapy shows long-term success in AADC - PharmaTimes

The Centre for Process Innovation (CPI) has announced it has overcome a significant bottleneck in the development of gene therapies.

CPI said its CRD IUK project, which was launched in partnership with Cobra Biologics and GE Healthcare Life Sciences, has been successful in its aim to develop a scalable, cost-effective purification process for adeno-associated viruses (AAVs).

AAVs are a vital technology for the delivery of gene therapies into patients. By transporting genetic material into patient cells, AAVs are able to provide a cure for otherwise untreatable diseases.

However, manufacturers are currently hindered by the low efficiency of AAVs production, which slows down the overall development timescale of gene therapies, ultimately increasing the cost for payers in healthcare systems.

The CRD IUK project was funded by a 570K grant from Innovate UK and focused on optimising an AAV purification process using GE Healthcare Life Sciences Fibro chromatography material. The material is based on electrospun cellulose nanofibers that contain different chromatography functionalities, overcoming the limitations of existing chromatographic supports.

Whilst the technology was understood to be highly effective for purification of biomolecules, the CRD IUK project extended the technologys effectiveness to AAVs. After assessing the technology, a multistep purification process was developed for AAV purification.

Daniel Smith, chief scientific officer, Cobra Biologics, said:We are delighted to have been part of this collaboration working to develop robust processes for use in the development of gene therapies. This project has provided a scalable, cost-effective fibre-based chromatography method for production of AAVs that will greatly enhance development of innovative new treatments.

John Liddell, chief technologist, CPI, said: Gene therapies have the potential to be transformative for disease areas with unmet clinical need, and effective manufacturing processes are crucial for reaching the time and cost points necessary for achieving commercialisation. This was the second Innovate UK-funded project related to viral vectors for CPI and therefore further enhances the Catapult centres ability to support growth of this emerging sector, which has been confirmed in subsequent gene therapy projects.

Oliver Hardick, business leader, Puridify, GE Healthcare Life Sciences, added: This has been an excellent collaboration with Cobra Biologics and CPI. Together, we have made a big step forward in the production of viral vectors to be used in gene therapies. The success of the project will significantly reduce the cost and time associated with development and manufacturing of AAVs, helping to accelerate delivery of gene therapy products to market.

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Industry collaboration overcomes significant bottleneck for gene therapy production - EPM Magazine