Category Archives: Genetic Therapy

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We are indeed living in interesting times and in many ways, thats a good thing. Take the automotive industry, for example. Technology is changing a rapid pace, and when it settles, it will dramatically change the way we drive. In 2030, our concept of car will likely be unrecognizable to drivers from 1980. The biggest changes are coming from power systems and artificial intelligence. AI will bring autonomous tech to our cars, making self-driving vehicles a reality. But the power systems changes will hit us first. In fact, electric-drive vehicles are already on our roads, and electric vehicle (EV) companies are proliferating rapidly. For the moment, there are several roads to potential success in the EV market. Companies are working to position themselves as leaders in battery tech, or electric power trains, or to maximize their range and performance per charge. Its a fact-paced industry environment, offering both opportunity and excitement for investors. Smart investors will look for companies capable of meeting scaling demands, once they have settled on marketable models. Investment firm Morgan Stanley has been watching the EV industry, seeking out innovative new design and production companies that are positioning themselves for gains as the market matures. The firms automotive analyst, Adam Jonas, has selected two stocks that investors should seriously consider buying into, saying As we survey the EV/battery startup landscape, we are prioritizing highly differentiated technology and/or business models with a path to scale at a reasonable level of risk. Opening up the TipRanks database, weve pulled up the details on both of Jonas picks to see whether they could be a good fit for your portfolio. Fisker (FSR) First up, Fisker, is based in Southern California, the epicenter of so much of our ground-breaking tech industries. Fiskers focus is on solid-state battery tech, a growing alternative to the lithium-ion batteries that most EVs depend on. While more expensive that the older lithium-based systems, solid state batteries are safer and offer higher energy densities. Fisker has been busy patenting its moves into solid-state batteries, a sound strategy to lock in its advances in this field. For EVs, solid-state batteries offer faster charging times, longer range per charge, and potentially lower battery weight all important factors in vehicle performance. Every car company needs a flagship model, and Fisker has the Ocean an EV SUV with a mid-range price ($37,499) and a long-range power system (up to 300 miles). The vehicle features stylish design and room mounted solar panels to supplement the charging system, and is scheduled to enter serial production for the markets in 2022. The stylish design reflects the sensibilities of the companys founder, Henrik Fisker, known for his work on the BMW Z8 and the Aston Martin DB9. Fisker entered the public markets through a SPAC merger agreement last fall. Since completing the SPAC transaction on October 29, shares in FSR are up 112%. Morgan Stanleys Jonas is impressed by this company, describing the value proposition of Fisker as design, time to market, clean sheet user experience and management expertise, and saying that the 4Q22 launch schedule for the Ocean is likely to be met. Fisker is specifically targeting the personal owned/passenger car business as opposed to commercial oriented end markets, where emotive design and user experience matter more. Additionally, the company wants to create an all-digital experience from the website to the app to the HMI in the car and continued customer engagement through its flexible lease product, Jonas added. In line with his upbeat outlook on the company (and the car), Jonas rates Fisker an Overweight (i.e. Buy), and sets a $27 price target suggesting an upside of 42% for the coming year. (To watch Jonas track record, click here) Turning to the TipRanks data, weve found that Wall Streets analysts hold a range of views on Fisker. The stock has a Moderate Buy analyst consensus rating, based on 7 reviews, including 4 Buys, 2 Holds, and 1 Sell. Shares are currently priced at $18.99, and the $21.20 average price target implies a one-year upside of ~12%. (See FSR stock analysis on TipRanks) QuantumScape (QS) Where Fisker is working on solid-state batteries in the context of vehicle production, QuantumScape is setting itself up as a leader in EV battery technology and a potential supplier of the next generation of battery and power systems for the EV market. QuantumScape designs and builds solid-state lithium-metal batteries, the highest energy density battery system currently available. The key advantages of the technology are in safety, lifespan, and charging times. Solid-state batteries are non-flammable; they last longer than lithium-ion batteries, with less capacity loss at the anode interface; and their composition allows faster charging, of 15 minutes or less to reach 80% capacity. QuantumScape is betting that these advantages will outweigh the technologys current higher cost, and create a new standard in EV power systems. The companys strongest tie to the EV production field is its connection with Volkswagen. The German auto giant put $100 million into QuantumScape in 2018, and an additional $200 million in 2020. The two companies are using their partnership to prepare for mass-scale development and production of solid-state batteries. Like Fisker, QuantumScape went public through a SPAC agreement late last year. The agreement, which closed on November 27, put the QS ticker in the public markets where it promptly surged above $130 per share. While the stock has since slipped, it remains up 47% from its NYSE opening. For Morgan Stanleys Jonas, involvement in QS stock comes with high risk, but also high potential reward. In fact, the analyst calls it, "The Biotech of Battery Development." "We believe their solid state technology addresses a very big impediment in battery science (energy density) that, if successful, can create extremely high value to a wide range of customers in the auto industry and beyond. The risks of moving from a single layer cell to a production car are high, but we think these are balanced by the commercial potential and the role of Volkswagen to help underwrite the early manufacturing ramp," Jonas explained. Noting that QS is a stock for the long haul, Jonas rates the shares an Overweight (i.e. Buy), and his $70 price target indicates confidence in an upside of 28% for one-year time horizon. Granted, not everyone is as enthusiastic about QS as Morgan Stanly. QS's Hold consensus rating is based on an even split between Buy, Hold, and Sell reviews. The shares are priced at $54.64 and their recent appreciation has pushed them well above the $46.67 average price target. (See QS stock analysis on TipRanks) To find good ideas for EV stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights. Disclaimer: The opinions expressed in this article are solely those of the featured analyst. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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Global Gene Therapy Partnering Deals Terms and Agreements Directory 2010-2020: Company A-Z, Deal Value, Phase of Development, Deal Type, and Therapy...

New York, Feb. 10, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Europe Cell and Gene Therapy Market - Industry Outlook and Forecast 2021-2026" - https://www.reportlinker.com/p06021776/?utm_source=GNW

The global cell and gene therapy market is observing significant mergers and acquisition activities, product sales, and new market authorizations. In 2026, the market is expected to grow almost four times more than the current value, with new product approvals expected annually. Although initial product approvals have been for relatively small patient groups, the significant pipeline of cell & gene therapy studies for diseases such as hemophilia and various forms of blindness will significantly expand. In addition, the Europe market is witnessing steady growth due to the increased availability of funds from several public and private institutes. There is increased support from regulatory bodies for product approvals and fast-track product designations, which encourage vendors to manufacture products at a fast rate. Moreover, with over 237 regenerative medicines companies headquartered in Europe, the region is seen as the favorite destination for cell and gene therapy manufacturing.

The following factors are likely to contribute to the growth of the Europe cell and gene therapy market during the forecast period: CMOs Offering Vector Manufacturing Services for Cell and Gene Therapy Companies Robust Cell & Gene Therapies in the Pipeline Increase in Strategic Acquisitions Regulatory Support for Cell and Gene Therapy Products

The study considers the present scenario of the Europe cell and gene therapy market and its market dynamics for the period 2020?2026. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market.

Europe Cell and Gene Therapy Market Segmentation The Europe cell and gene therapy market research report includes a detailed segmentation by product, end-user, application, geography. A high potential to treat several chronic diseases, which cannot be effectively treated/cured through conventional methods otherwise, is propelling the growth of gene therapies. Gene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields. The number of clinical trials investigating gene therapies is increasing in Europe, despite the limited number of products that have successfully reached the market. However, gene therapies show slow progress and promising prospect in terms of treatments. High support from regulatory bodies to commercialize these products and make them affordable to patients is another important factor contributing the market growth.

Delivering cell and gene therapies requires specialized facilities, capabilities, and clinician skills. Therefore, manufacturers are working in tandem with chosen treatment centers (hospitals) to establish the protocols and procedures necessary to receive the product and therapies. While cell therapies represent a paradigm shift in the treatment of several incurable, chronic diseases, with durable responses and long-term disease control measures, hospitals appear an ideal location to carry out these procedures. Hospitals are growing at a significant rate due to the increasing target population in Europe. Tier-I hospitals are proving to be sought-after network partners for cell and gene therapy developers. They tend to be in major population centers, have adequate financial and personnel resources, and value the prestige that comes with being the first movers in an innovative treatment area.

Oncology accounted for a share of over 30% in 2020. While cancer treatments have evolved and undergone massive developments in recent years, it continues to be one of the deadliest diseases confronted by humans. Traditional cancer therapies have a curative effect in the short term; however, they have side effects, thereby decreasing the patients quality of life. Cell and gene therapies for certain types of cancers have been promising results. The chimeric antigen receptor- (CAR-) T cell therapy is one of the most recent innovative immunotherapies and is rapidly evolving. CAR-T cell therapies are developing rapidly, and many clinical trials have been established on a global scale, which has high commercial potential for the treatment of cancer. Immunotherapies based on CAR-T cells go one step further, engineering the T cells themselves to enhance the natural immune response against a specific tumor antigen. CAR-T clinical trials have shown high remission rates, up to 94%, in severe forms of blood cancer, thereby increasing the market growth.

Product Cell Therapies Gene Therapies End-user Hospitals Cancer Care Centers Wound Care Centers Others Application Oncology Dermatology Musculoskeletal Others

INSIGHTS BY GEOGRAPHY Germany, France, the UK, Italy, and Spain play a significant role in the Europe cell and gene therapy market. Clinical trials and the number of manufacturing facilities are increasing slowly in the European region. The region has become a major R&D destination for several vendors as the funding for cell & gene therapies is increasing. Europe has supported collaborative efforts in gene transfer and gene therapy research. In addition, the target patient population is increasing across Europe; there were an estimated 3.9 million new cases of cancer and 1.9 million cancer deaths in Europe in 2018. In addition, the prevalence surveys in the UK and Denmark indicate that there are 34 people with one or more wounds per 1,000 people. Favorable government support in terms of product approvals, reimbursement and coverage, and high R&D funding to academic institutes that are involved in the development of cell and gene therapies are expected to boosting the market in Europe.

Geography Europe o UK o Germany o France o Italy o Spain o Switzerland o Netherlands

INSIGHTS BY VENDORS Novartis, Spark Therapeutics, Amgen, Gilead Sciences, and Organogenesis are the leading players in the Europe cell and gene therapy market. The market offers tremendous growth opportunities for existing and future/emerging players on account of the presence of a large pool of target patient population with chronic diseases such as cancer, wound disorders, diabetic foot ulcer, CVDs, and other genetic disorders. Recent approvals have prompted an unprecedented expansion among vendors. While a few vendors are opting for in-house production of cell and gene therapies, a substantial number of vendors are preferring third-party service providers, including CMOs.

Prominent Vendors Novartis Spark Therapeutics Amgen Gilead Sciences Organogenesis

Other Prominent Vendors APAC Biotech AVITA Medical bluebird bio CHIESI Farmaceutici CollPlant CO.DON Human Stem Cells Institute PJSC (HSCI) Medipost NuVasive Nipro Orchard Therapeutics RMS Regenerative Medical System Orthocell Osiris Therapeutics Sibino GeneTech Shanghai Sunway Biotech Takeda Pharmaceutical Company Terumo Vericel

Emerging Investigational Vendors In Europe Adaptimmune Therapeutics AgenTus Therapeutics Autolus Cellecits Celyad CombiGene EUKARS Freeline Therapeutics Innoskel PsiOxus Therapeutics Ltd SparingVision uniQure

KEY QUESTIONS ANSWERED 1. What is the Europe cell and gene therapy market size and growth rate during the forecast period? 2. What are the factors driving the demand for CAR-T therapy in the European region? 3. How are strategic acquisitions aiding in market growth of cell and gene therapy products? 4. Which segments are expected to generate the highest revenues during the forecast period? 5. Who are the leading vendors in the European cell and gene therapy market?Read the full report: https://www.reportlinker.com/p06021776/?utm_source=GNW

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The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 20212026 - GlobeNewswire

ALISO VIEJO, Calif.--(BUSINESS WIRE)--As gene therapies and editing technologies rapidly advance, it is more urgent than ever to provide updates and information to the rare disease community on how these technologies can be applied across multiple diseases. Global Genes, a leading rare disease patient advocacy organization, is pleased to announce they will be publishing a multimedia series, titled Platforms of Hope: Advances in Gene Therapy and Gene Editing, throughout 2021 regarding upcoming data announcements and information on gene therapy and editing technology advances with thought leaders from the National Center for Advancing Translational Sciences (NCATS), the NIH Common Funds Somatic Cell Genome Editing (SCGE) program and other leading voices in these fields.

In addition to ongoing coverage through Global Genes videos, online publication RARE Daily and RARECast podcast, the organization will be publishing a special report at the end of 2021 on gene therapy and gene editing innovation. This will address a wide range of topics, with a focus on efforts to accelerate the translation of discoveries into genetic medicines that benefit patients with rare diseases.

With more than 7,000 rare diseases, there is an urgent need to keep the rare disease community abreast of developments in the rapidly changing fields of gene therapy and genome editing, said P.J. Brooks, program director at the Office of Rare Diseases Research at the NCATS. In this collaboration, NIH will help Global Genes identify the ideas, technologies and advances that have broad implications for many patients and families affected by rare diseases and provide information that could positively impact their lives and care in the future.

This collaboration will bring visibility into cutting-edge science at the frontier of genetic medicines and provide the rare disease community with insights into emerging technologies and therapies in development for rare diseases, said Christian Rubio, vice president, strategic advancement at Global Genes. Its critically important to educate the rare disease community on these rapidly evolving events.

For more information, visit http://www.globalgenes.org/media-hub.

About Global Genes

Global Genes is a 501(c)(3) nonprofit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. In pursuit of our mission, we connect, empower, and inspire the rare disease community to stand up, stand out, and become more effective on their own behalf -- helping to spur innovation, meet essential needs, build capacity and knowledge, and drive progress within and across rare diseases. We serve the more than 400 million people around the globe and nearly one in 10 Americans affected by rare diseases. If you or someone you love has a rare disease, or are searching for a diagnosis, contact Global Genes at 949-248-RARE, or visit our resource hub.

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Global Genes Announces New Multimedia Series Focused on Advances in Gene Therapy and Editing, in Collaboration with the National Institutes of Health...

PARIS--(BUSINESS WIRE)--Regulatory News:

Lysogene (FR0013233475 LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announces that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for LYS-GM101, the companys gene therapy candidate for the treatment of GM1 gangliosidosis, a serious, pediatric, life threatening disease. LYS-GM101 builds on Lysogenes extensive experience in direct to CNS adeno-associated viral vector (AAV)-based gene therapy clinical development.

The IND clearance follows the recent clinical trial authorization granted by the MHRA in the United Kingdom. Lysogene intends to initiate its global, multi-center, single-arm, two-stage, adaptive-design clinical trial of LYS-GM101 in patients with a diagnosis of early or late infantile GM1 gangliosidosis. The clinical trial will include a safety phase and a confirmatory efficacy phase. The company intends to dose a total of 16 patients, with dosage of the first patient expected in the first half of 2021.

We are very pleased to receive this IND clearance for LYS-GM101 which completes the MHRA approval received a few weeks ago. It represents a major milestone that marks our second CNS gene-therapy program to enter into a global clinical trial said Karen Aiach, Founder Chairman and Chief Executive Officer of Lysogene. This IND clearance once again demonstrates our quality and timely execution, and our strong determination to bring new therapeutic solutions for diseases that currently have no treatment.

Christine Waggoner, President and Co-Founder of Cure GM1 Foundation added: Children with GM1 gangliosidosis represent a clear unmet medical need and we are thrilled to see a new therapeutic option entering the clinic, as it brings tremendous hope to families and the entire GM1 gangliosidosis community.

LYS-GM101 (adeno-associated viral vector serotype rh.10 expressing beta-galactosidase) received orphan drug designation for the treatment of GM1 gangliosidosis in the European Union and in the US in 2017, as well as Rare Pediatric Disease designation in the US in 2016.

Leading international gene therapy and Lysosomal Storage Disease centers plan to participate in the clinical trial (NCT04273269).

Lysogene is also funding a GM1 gangliosidosis natural history study being conducted by Casimir Trials to collect prospective and/or retrospective videos of children doing certain everyday tasks and behaviors in infantile and juvenile GM1 gangliosidosis (NCT04310163).

About LysogeneLysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS). The Company has built a unique capability to enable delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing. An adaptative clinical trial in GM1 gangliosidosis is in preparation. In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. will hold exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene will maintain commercial exclusivity of LYS-SAF302 in Europe. Lysogene is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism. http://www.lysogene.com.

Forward Looking StatementThis press release may contain certain forward-looking statements, especially on the Companys progress of its clinical trials and cash runway. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice, (ii) factors beyond the Companys control, (iii) clinical trial results, (iv) increased manufacturing costs and (v) potential claims on its products. These statements may include, without limitation, any statements preceded by, followed by or including words such as target, believe, expect, aim, intend, may, anticipate, estimate, plan, objective, project, will, can have, likely, should, would, could and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Companys control that could cause the Companys actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. A further list and description of these risks, uncertainties and other risks can be found in the Companys regulatory filings with the French Autorit des Marchs Financiers, including in the 2019 universal registration document, registered with the French Markets Authorities on April 30, 2020, under number D.20-0427, and future filings and reports by the Company. Furthermore, these forward-looking statements are only as of the date of this press release. Readers are cautioned not to place undue reliance on these forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. If the Company updates one or more forward-looking statements, no inference should be drawn that it will or will not make additional updates with respect to those or other forward-looking statements.

This press release has been prepared in both French and English. In the event of any differences between the two texts, the French language version shall supersede.

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Lysogene Receives FDA Clearance of Investigational New Drug Application to Initiate the Gene Therapy Clinical Trial in the US with LYS-GM101 for the...

In addition to preservation of neurocognitive development with ABO-102 in MPS IIIA, new clinical results of ABO-102 in MPS IIIA and ABO-101 in MPS IIIB continue to show dose-dependent and sustained reductions in disease-specific biomarkers, denoting clear biologic effects

In addition, ABO-102 and ABO-101 continue to show favorable safety profile in ongoing studies

Abeona to host investor webinar on Tuesday, February 16, 2021 at 1:00 p.m. EST

NEW YORK and CLEVELAND, Feb. 12, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced new positive data from two ongoing Phase 1/2 clinical trials of the companys investigational AAV-based gene therapies ABO-102 and ABO-101 in MPS IIIA and MPS IIIB, respectively. The interim data was presented in late-breaking platform oral presentations at the 17th Annual WORLDSymposium. The presentation slides are available on the companys website at http://www.abeonatherapeutics.com.

Michael Amoroso, Principal Executive and Chief Operating Officer of Abeona, stated, We are excited to share updated positive efficacy and safety results that continue to suggest ABO-102 has the potential to be a life-altering treatment option for children with MPS IIIA, a rare, debilitating condition with no approved treatment that leads to progressive neurodevelopmental and physical decline, and often results in death early in life. We have requested a meeting with the FDA later this quarter to discuss the ABO-102 data and the potential path towards a Biologics License Application filing for ABO-102. In addition, the new results from the Transpher B study continue to support ABO-101s biologic activity in patients with MPS IIIB.

The updated results from the Transpher A study evaluating ABO-102 in Sanfilippo syndrome type A (MPS IIIA) demonstrated that neurocognitive development was preserved within normal range of a non-afflicted child for 2.5 years to 3 years (the latest time point measured) after treatment with ABO-102 (3x1013 vg/kg) in three young patients in the high-dose cohort 3. The three young patients were treated with ABO-102 at ages 27 months, 19 months, and 12 months and are now at ages ranging from 3.5 years to 5+ years, the timepoint at which patients with MPS IIIA have already started to experience neurocognitive decline based on the natural history of disease progression. Dose-dependent and statistically significant reductions in cerebrospinal fluid heparan sulfate, denoting enzyme activity in the central nervous system (CNS), and liver volume were sustained for two years after treatment. ABO-102 has been well-tolerated with long-term safety remaining favorable 24-55 months following treatment. There have been no treatment-related severe adverse events and no clinically significant adverse events reported.

Kevin Flanigan, M.D., Director, Center for Gene Therapy at AWRI at Nationwide Children's and Transpher A study principal investigator, said, The results presented today show a single intravenous dose of ABO-102 can help preserve neurocognitive development for up to 3 years following treatment in young MPS IIIA patients during early stages of their disease. The data shows ABO-102s ability to deliver a functional copy of the disease-causing SGSH gene to cells of the CNS and peripheral organs, as evidenced by the clinical benefits in neurocognition and biophysical measures and improvements in disease-specific biomarkers.

Results from the Transpher B study evaluating ABO-101 in Sanfilippo syndrome type B (MPS IIIB) showed that treatment with ABO-101 is associated with a dose-dependent and sustained improvement in central nervous system and systemic biomarkers, indicating the potent biologic effect of ABO-101 in patients with MPS IIIB. ABO-101 has been well-tolerated with no infusion related or early acute reactions and no clinically significant adverse events or laboratory abnormalities.

Maria Jose de Castro, M.D., Hospital Clnico Universitario Santiago de Compostela and Transpher B study investigator, said, The results from the Transpher B study provide evidence of ABO-101s impact on disease biomarkers and potential to break down the accumulation of glycosaminoglycans that underlie MPS IIIB pathology. We look forward to continued follow-up to assess ABO-101s potential to preserve neurocognitive development in patients with MPS IIIB.

Investor Webinar on MPS III Gene Therapy Programs

Abeona management along with Dr. Flanigan and Dr. de Castro will host an investor webinar on February 16, 2021 at 1:00 p.m. EST. The live webinar, including audio and presentation slides, will be accessible at https://investors.abeonatherapeutics.com/events at the time of the meeting. To register in advance for the live webinar, click here.

An archived replay of the webinar will be available after the conclusion of the live event at https://investors.abeonatherapeutics.com/events.

About the Annual WORLDSymposium The WORLDSymposium is designed for basic, translational and clinical researchers, patient advocacy groups, clinicians, and all others who are interested in learning more about the latest discoveries related to lysosomal diseases and the clinical investigation of these advances. For additional information on the 17th Annual WORLDSymposium, please visit https://worldsymposia.org/.

About the Transpher A Study The Transpher A Study (NCT02716246) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). The study, also known as ABT-001, is intended for patients 6 months to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-102 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

About the Transpher B Study The Transpher B Study (NCT03315182) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-101 for the treatment of patients with Sanfilippo syndrome type B (MPS IIIB). The study, also known as ABT-002, is intended for patients 6 months to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-101 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

About ABO-102 ABO-102 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). ABO-102 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations for the ABO-102 clinical program. In the EU, the Company holds PRIME and Orphan medicinal product designations.

About ABO-101 ABO-101 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type B (MPS IIIB), a rare lysosomal storage disease with no approved therapy that primarily affects the central nervous system (CNS). ABO-101 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the NAGLU gene to cells of the CNS and peripheral tissues. The therapy is designed to address the underlying NAGLU enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Fast Track and Rare Pediatric Disease designations for ABO-101 and Orphan Drug designation in both the U.S. and EU.

About Sanfilippo Syndrome Type A (MPS IIIA) Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the CNS and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIA present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive facial features with thick eyebrows or a unibrow, full lips and excessive body hair for ones age, and liver/spleen enlargement are also present in early childhood. MPS IIIA is caused by genetic mutations that lead to a deficiency in the SGSH enzyme responsible for breaking down glycosaminoglycans, which accumulate in cells throughout the body resulting in rapid health decline associated with the disorder.

About Sanfilippo syndrome type B (MPS IIIB) Sanfilippo syndrome type B (MPS IIIB) is a rare and fatal lysosomal storage disease with no approved therapy that primarily affects the central nervous system and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIB present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive signs such as facial features with thick eyebrows or a unibrow, full lips and excessive body hair for ones age and liver/spleen enlargement are also present. The underlying cause of MPS IIIB is a deficiency in the NAGLU enzyme responsible for breaking down glycosaminoglycans, which accumulate throughout the body resulting in rapid decline associated with the disorder.

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeonas clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Companys portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical needs. Abeonas novel, next-generation AIM capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeonas fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit http://www.abeonatherapeutics.com.

Forward-Looking StatementsThis press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company exploring all strategic options, including the sale of some or all of its assets or sale of the Company. We have attempted to identify forward-looking statements by such terminology as may, will, believe, estimate, expect, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, the outcome of the strategic review, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Companys most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

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New Positive Phase 1/2 Interim Data Presented at WORLDSymposium Shows Neurocognitive Development of Young MPS IIIA Patients Preserved up to Three...

- IND-Enabling Studies Demonstrated Potential of a Single I.V. Administration of HMI-203 to Address Peripheral and CNS Components of Disease -

- Data at WORLDSymposium Support Plans to Initiate a HMI-203 Phase 1/2 Clinical Trial This Year -

BEDFORD, Mass., Feb. 08, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a clinical-stage genetic medicines company, announced today the first scientific presentation of data from IND-enabling studies with its HMI-203 gene therapy development candidate for Hunter syndrome (MPS II). The results from Homologys in vivo approach demonstrated the potential of HMI-203 to address peripheral manifestations in the murine disease model and showed that HMI-203 crossed the blood-brain-barrier following a single I.V. administration. These data will be presented during a poster presentation at the 17th Annual WORLDSymposium Meeting, where Homology will also present long-term data from its HMI-202 in vivo gene therapy program for metachromatic leukodystrophy (MLD).

By leveraging our family of AAVHSC vectors to target both peripheral organs and the central and peripheral nervous systems in preclinical models of Hunter syndrome and MLD, debilitating diseases that have high unmet medical need, we also demonstrated the continued expansion of our CNS platform, stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. This is our first presentation of preclinical data from our Hunter syndrome gene therapy program, and these IND-enabling studies showed that a single I.V. administration produced high levels of enzymatic expression across disease-relevant tissues and achieved phenotypic correction of skeletal deformities. These results support our plans to move this program forward into a Phase 1/2 clinical trial this year.

In the poster titled, HMI-203: Investigational Gene Therapy for Mucopolysaccharidosis Type II (MPS II), or Hunter Syndrome, a single I.V. administration of HMI-203 in the adult murine model:

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Led to robust biodistribution and sustained human I2S (hI2S) enzyme expression, which resulted in:

Significant reductions in key Hunter syndrome biomarkers of heparin sulfate glycosaminoglycans (GAGs) and lysosomal-associated membrane protein 1 (LAMP-1) in the brain, liver, heart, spleen, lung and kidneys compared with vehicle.

Significant reductions in heparan sulfate GAGs in the cerebrospinal fluid (CSF) compared with vehicle.

Ameliorated paw deformities, as shown by significant changes in measurements of ankle depth, paw width, paw depth and ankle width compared with vehicle.

Led to uptake of hI2S from the serum of the HMI-203-treated model in human cell lines, demonstrating potential for cell cross-correction.

In an additional poster titled, HMI-202: Gene Therapy Development Candidate for Metachromatic Leukodystrophy (MLD), a single I.V. administration of HMI-202:

Crossed the blood-brain-barrier and blood-nerve-barrier in the murine MLD disease model and in non-human primates (NHPs), with human ARSA (hARSA) detected in neuronal and glial cells.

Showed durable hARSA activity in the central nervous system of the disease model, with distribution levels resembling those of Arsa in normal age-matched controls.

Demonstrated significant changes in key MLD biomarkers of LAMP-1, glial fibrillary acidic protein (GFAP), MAL transcript and neuronal sulfatides in the disease model compared with vehicle, similar to age-matched wild type controls.

Homologys e-poster presentations will take place on February 11, 2021 at 2:30 p.m. ET. For more information, visit http://www.homologymedicines.com/publications.

About Mucopolysaccharidosis Type II (MPS II), Hunter SyndromeMPS II, or Hunter syndrome, is a rare, X-linked lysosomal storage disorder caused by mutations in the iduronate-2-sulfatase (IDS) gene, which is responsible for producing the I2S enzyme that breaks down large sugar molecules, or cellular waste, called glycosaminoglycans (GAGs). Severe Hunter syndrome results in toxic lysosomal accumulation of GAGs that causes progressive debilitation and decline in intellectual function. Hunter syndrome occurs in approximately 1 in 100,000 to 1 in 170,000 males, and the severe form leads to life expectancy of 10 to 20 years.

About Metachromatic Leukodystrophy (MLD)MLD is a rare lysosomal storage disorder caused by mutations in the ARSA gene. ARSA is responsible for the creation of the arylsulfatase A (ARSA) protein, which is required for the breakdown of cellular components. In MLD, these cellular components accumulate and destroy myelin-producing cells in the peripheral and central nervous system leading to progressive and serious neurological deterioration. The late infantile form of the disorder is estimated to affect 1 in 40,000 people, and it is fatal within five to ten years after onset.

About Homology Medicines, Inc.Homology Medicines, Inc. is a clinical-stage genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homologys proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visit http://www.homologymedicines.com.

Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; our plans to move forward into a Phase 1/2 clinical trial for HMI-203 this year; our position as a leader in the development of genetic medicines; and our participation in upcoming presentations and conferences. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities of our manufacturing facility; risks relating to the regulatory approval process; interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2020 and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

Company Contacts:Theresa McNeelyChief Communications Officerand Patient Advocatetmcneely@homologymedicines.com 781-301-7277

Media Contact:Cara MayfieldSenior Director, Patient Advocacyand Corporate Communicationscmayfield@homologymedicines.com 781-691-3510

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Homology Medicines Announces First Presentation of Data with HMI-203 In Vivo Gene Therapy Development Candidate for Hunter Syndrome - Yahoo Finance