The new report titled Hemophilia Gene Therapy Market offer by Key Players, Types, Applications, Countries, Market Size, Forecast to 2030offered by Market Research, Inc. includes a comprehensive analysis of the market size, geographical landscape along with the revenue estimation of the industry. In addition, the report also highlights the challenges impeding market growth and expansion strategies employed by leading companies in the Hemophilia Gene Therapy Market.

Hemophilia is a genetic bleeding disorder in which an individual lacks or has low levels of proteins called clotting factors. There are around 13 types of clotting factors that work with blood platelets, which are necessary for clotting process to initiate. There are three forms of hemophilia A, B, and C. Hemophilia A is the most common form and is caused due to deficiency in clotting factor VIII. Hemophilia B occurs due to deficiency of clotting factor IX and Hemophilia C is caused due to clotting factor XI deficiency. Hemophilia is incurable with current therapeutic options, which only reduces symptoms such as spontaneous bleeding in muscles and joints as well as increased risk for intracranial hemorrhage.

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This market study covers and analyzes the potential of the global Hemophilia Gene Therapy industry, providing geometric information about market dynamics, growth factors, major challenges, PEST analysis and market entry strategy analysis, opportunities and forecasts. One of the major highpoints of the report is to provide companies in the industry with a strategic analysis of the impact of COVID-19 on Hemophilia Gene Therapy market.

Hemophilia Gene Therapy Market: Competition Landscape

The Hemophilia Gene Therapy market report includes information on the product presentations, sustainability and prospects of leading player including: BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC Sangamo Therapeutics, Inc., and Freeline Therapeutics

Hemophilia Gene Therapy Market: Segmentation

By Type:

By Application:

Hemophilia Gene Therapy Market: Regional Analysis

All the regional segmentation has been studied based on recent and future trends and the market is forecasted throughout the prediction period. The countries covered in the regional analysis of the Global Hemophilia Gene Therapy market report are North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and Latin America.

Years Considered for the Hemophilia Gene Therapy Market Size:

Key Benefits of the report:

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Major Points Covered in TOC:

Market Summary: It incorporates six sections, research scope, major players covered, market segments by type, Hemophilia Gene Therapy market segments by application, study goals and years considered.

Market Landscape: Here, the global Hemophilia Gene Therapy Market is dissected, by value, income, volume, market rate, and most recent patterns. The development and consolidation of the overall industry and top organizations is provided through graphs and piece of the pie for organizations.

Profiles of Companies: Here, driving players of the worldwide Hemophilia Gene Therapy market are considered depending on sales across regions, key innovations, net income, cost, and other factors.

Market Status and Outlook by Region: In this segment, the report examines the net deals, income, creation and portion of the overall industry, CAGR and market size by locale. The global Hemophilia Gene Therapy Market is profoundly examined based on areas and nations like North America, Europe, Asia Pacific, Latin America and Middle East & Africa.

Segment Analysis: Accurate and reliable foretell about the market share of the essential sections of the Hemophilia Gene Therapy market is provided

Market Forecasts: In this section, accurate and validated values of the total market size in terms of value and volume are provided by the research analysts. Also, the report includes production, consumption, sales, and other forecasts for the global Hemophilia Gene Therapy Market.

Market Trends: Deep dive analysis of the markets recent and future trends are provided in this section.

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Hemophilia Gene Therapy Market by Growth Analysis and Precise Outlook 2030 | Key Players BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer,...

The Food and Drug Administration has halted testing of a preclinical cancer medicine from Beam Therapeutics, the biotechnology company announced Monday.

Beam, a high-profile developer of a gene editing technique known as base editing, said in a short statement that the FDA put its request to start human trials of the experimental treatment on clinical hold.

Beam didnt say why the FDA paused its application. The biotech was informed of the agencys decision via an email on Friday, and expects to provide an update pending discussion with the FDA. The regulator will provide Beam with a formal letter within 30 days.

Company shares fell by more than 10% in pre-market trading Monday.

Beam is the leading developer of base editing, an approach borne out of research from the labs of Harvard University gene editing specialist David Liu. Unlike the first generation of CRISPR editing, which cuts both strands of DNA, base editing is designed to change single DNA letters without causing a double-stranded break, a method thats thought to carry fewer risks.

Beam was formed five years ago to turn the approach into human medicines and has since received significant financial support. The company raised $180 million in an initial public offering in February 2020 and in January got $300 million upfront from Pfizer in a wide-ranging research deal. The biotech had $1.2 billion in cash on its balance sheet at the end of the first quarter.

The company has already been cleared by U.S. regulators to start a study of BEAM-101, a drug for sickle cell disease, and expects to start enrolling patients in that trial later this year. Verve Therapeutics also recently began clinical testing of a heart disease drug that uses Beams base editing technology.

BEAM-201, an experimental treatment for leukemia and lymphoma, was expected to follow this year along with a second sickle cell drug called BEAM-102.

Verves treatment is an infusion of a drug that performs base editing inside the body. Beams two most advanced programs, including the cancer drug now on hold, genetically modify cells outside the body.

BEAM-201 is meant to overcome some of the limitations of personalized cancer cell therapies from Bristol Myers Squibb, Novartis and Gilead, which are approved to treat certain leukemias and lymphomas. The treatment uses cells from donors, rather than patients themselves, and silences multiple genes simultaneously an approach Beam claims could make those cells more durable. Several developers of so-called off-the-shelf cell therapies have struggled to prove their drugs are as long-lasting as personalized treatments, however.

The drug is the latest gene-based medicine, meanwhile, to be slowed by regulators. The FDA has recently paused testing of a number of gene replacement or gene editing therapies, wary of potential safety concerns.

This is obviously negative for the stock and reiterates a high level of scrutiny from the regulators on novel technologies like gene/base editing, wrote RBC Capital Markets analyst Luca Issi in a research note. However, we also note that the [application] was submitted at the end of June, so we assume no patient has been dosed, and it is possible that the hold is simply procedural in nature.

Beam is seeking to treat patients with either relapsed or refractory T cell acute lymphoblastic leukemia or T cell lymphoblastic lymphoma.

More:
FDA halts testing of Beam's base editing cancer therapy - BioPharma Dive

BTIG Biotechnology Conference on August 8, 2022 at 12:00 pm ET

2022 Wedbush PacGrow Healthcare Virtual Conference on August 10, 2022 at 9:45 am ET

DALLAS, Aug. 01, 2022 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in panel discussions at the BTIG Biotechnology Conference on August 8th and the 2022 Wedbush PacGrow Healthcare Virtual Conference on August 10th.

A webcast of the panels will be available in the Events & Media section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations. Archived versions of the webcast will be available on the website for 90 days.

About Taysha Gene Therapies

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

Company Contact:Kimberly Lee, D.O. Chief Corporate Affairs OfficerTaysha Gene Therapiesklee@tayshagtx.com

Media Contact:Carolyn HawleyEvoke Canalecarolyn.hawley@evokegroup.com

Read this article:
Taysha Gene Therapies to Participate in Upcoming August Investor Conferences - GlobeNewswire

Voyager Therapeutics, Inc.

Pipeline prioritizes tau antibody for Alzheimer's disease and gene therapies for GBA1 Parkinson's disease and SOD1 ALS, each program employing efficient paths to human proof of biology

Targeting development candidate selection for priority programs in 2022 and H1 2023

Cross species transduction for multiple capsids and characterization of the receptor for a leading capsid support human translation potential of TRACERTM capsids

Catherine J. Mackey, Ph.D., appointed to Board of Directors

Strong balance sheet and disciplined financial approach expected to maintain cash runway into 2024

Conference call at 4:30 p.m. ET today

CAMBRIDGE, Mass., Aug. 04, 2022 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a gene therapy and neuroscience company developing life-changing treatments and next-generation adeno-associated virus (AAV) capsids, today unveiled its prioritized therapeutic pipeline and reported second quarter 2022 financial and operating results.

Im excited to announce our updated portfolio strategy that prioritizes programs targeting lead indications in Alzheimers disease, GBA1 Parkinsons disease, and SOD1 ALS, said Alfred W. Sandrock, Jr., M.D., Ph.D., chief executive officer of Voyager. We believe our highly differentiated approaches against well-validated CNS targets leveraging Voyagers breakthrough TRACER capsids can lead to the development of new therapeutic options for these devastating diseases. In addition, we believe each of these targets has a clear path to human proof of biology that should allow us to further progress these programs in an efficient manner.

Dr. Sandrock continued, The recent characterization of a receptor for one of our most promising TRACER capsids, along with the preclinical cross-species transduction data we presented at ASGCT for several or our capsids, increase our confidence that our capsids may cross the blood-brain barrier in humans. On the partnership front, we believe that our Pfizer and Novartis TRACER capsid collaborations are going well with option exercise decisions upcoming in Q4 2022 and Q1 2023, respectively, and we are optimistic about the potential for similar transactions in the future.

Story continues

Prioritized Pipeline Focused on Programs with Efficient Path to Human Proof of Biology

Following an in-depth internal review process, Voyager has prioritized pipeline programs for its development. This review identified a compelling opportunity for each prioritized program based on the following criteria: high unmet medical need, target validation, efficient path to human proof of biology, robust preclinical pharmacology, and strong commercial potential. Voyager is evaluating partnering opportunities for its other programs.

Prioritized pipeline programs include:

GBA1 gene replacement to treat Parkinsons disease patients with GBA1 mutations

Mutations in glucocerebrosidase 1 (GBA1), the gene encoding the lysosomal enzyme glucocerebrosidase (GCase), are the most common genetic risk factor for synucleinopathies such as Parkinsons disease and dementia with Lewy bodies. Voyager believes restoring GCase activity may attenuate progression and potentially slow neurodegeneration. Voyager anticipates delivering GBA1 (utilizing a blood-brain barrier (BBB)-penetrant, CNS-tropic AAV capsid discovered by the TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform) via intravenous (IV) delivery to enable widespread distribution to multiple affected brain regions and to avoid the need for more invasive approaches.

Voyager believes the measurement of the GCase substrates such as glucosylsphingosine as cerebrospinal fluid (CSF) biomarkers will facilitate efficient clinical demonstration of proof of biology.

At the American Society of GeneandCell Therapy(ASGCT) 25th Annual Meeting in May, Voyager presented preclinical data demonstrating CNS target engagement and delivery of therapeutically relevant levels of the lysosomal enzyme GCase (encoded by GBA1) in a GBA loss of function mouse model, as well as sustained expression for 3 months following IV administration.

A non-human primate (NHP) capsid evaluation study is currently underway. Voyager intends to select a development candidate in the first half of 2023, to initiate a dose range finding study in NHPs in the second half of 2023, and to initiate GLP toxicology studies in 2024. Voyager anticipates an IND filing in 2025 but is actively reviewing options to accelerate the program.

SOD1 gene silencing to treat amyotrophic lateral sclerosis (ALS) caused by the superoxide dismutase 1 (SOD1) mutation

Voyager believes that a therapeutic combining a highly potent siRNA construct with a CNS-tropic, BBB-penetrant capsid discovered by Voyagers TRACER platform allowing for IV delivery may enable broad CNS knockdown of SOD, potentially slowing the decline of functional ability. Voyager believes a Phase 1 clinical trial to demonstrate reduction in SOD1 in CSF and neurofilament light chain in plasma will provide evidence of target engagement and the attenuation of motor neuron loss, respectively.

At the ASGCT meeting, Voyager presented preclinical data demonstrating robust SOD1 knockdown in all levels of the spinal cord and significant improvements in motor performance, body weight, and survival in an SOD1-ALS mouse model following IV delivery of a vectorized siRNA using a mouse BBB-penetrant capsid.

A NHP capsid evaluation study is currently underway. The Company intends to select a development candidate in 2022, complete an NHP dose range finding study in 2023, initiate GLP toxicology studies in the first half of 2024 and file an IND in 2024.

Receptor Identified for TRACER AAV Capsid; AAV9- and AAV5-Derived TRACER Capsids Demonstrate Enhanced Brain Transduction Across NHPs and Rodents

Voyager announced today that it has identified the receptor for one of its most promising TRACER AAV capsids. The Company has confirmed that those capsids can bind to the human isoform of the receptor, which is expressed in brain endothelial cells and other CNS cell types. Voyager believes that characterization of this receptor-capsid interaction increases the probability that the related capsid will cross the BBB in humans. Voyager intends to share data on this finding at an upcoming scientific conference.

At the ASGCT meeting, Voyager presented new preclinical data on several families of novel capsids from its TRACER capsid discovery platform.

Voyager presented preclinical results for an AAV9-derived capsid, VCAP-102, which demonstrated 50-fold better transduction in mice and 60-fold better transduction in NHPs versus conventional AAV9 capsids following IV administration. Voyager believes demonstrating equivalent cross-species functionality is critical to increasing a capsids potential for translation into humans. The study also demonstrated that VCAP-102 and other TRACER capsids showed preferential tropism for glial cells in mice, which may facilitate addressing CNS indications that would benefit from non-neuronal cell transduction.

Voyager also presented preclinical data for an AAV5-derived capsid with enhanced CNS transduction across species. AAV5 capsids have a reduced prevalence of pre-existing neutralizing antibodies, but conventional AAV5 capsids do not allow sufficient BBB-penetration to be used for gene therapies targeting the CNS. Voyagers AAV5-derived capsid showed 20-fold higher brain transduction and five-fold higher spinal cord transduction compared to conventional AAV9 in NHPs. Modified capsids showed improved transduction in multiple CNS regions and cell types in NHPs, together with partial de-targeting from the dorsal root ganglia.

Voyager continues to perform screening campaigns with its TRACER platform to identify additional proprietary AAV9- and AAV5-derived capsids and to refine already identified capsids to target or de-target multiple tissue and cell types. These capsids offer the potential to broaden the therapeutic window substantially and to enable gene therapies in a wide range of diseases based on enhanced tissue/cell tropisms that allow for lower doses and with lower off-target effects or toxicities.

Initial option exercise decision of the license agreements with Pfizer and Novartis for TRACER AAV capsids are expected by October 2022 and March 2023, respectively.

Catherine J. Mackey, Ph.D., Appointed to Board of Directors

In July, Voyager announced the appointment of Catherine J. Mackey, Ph.D. to its Board of Directors. Dr. Mackey is a seasoned life science executive with more than thirty years of operational experience highlighted by her tenure as Senior Vice President, Global Research & Development, for Pfizer. Dr. Mackey, who will join Voyagers Board on August 15, has been designated as a Class I director with a term ending as of the 2025 annual meeting of Voyager shareholders. Dr. Mackey has also been appointed to the Boards Audit Committee and Science and Technology Committee.

Second Quarter 2022 Financial Results

Collaboration Revenues: Voyager had collaboration revenue of $0.7 million for the second quarter of 2022, compared to $1.4 million for the same period in 2021. The decrease in collaboration revenue was due to a reduction of Voyagers research activities within the collaboration with Neurocrine.

Net Loss: Net loss was $19.1 million for the second quarter of 2022, compared to a net loss of $30.1 million for the same period of 2021.The decrease in net loss resulted primarily from a decrease in R&D expenses and G&A expenses.

R&D Expenses: Research and development expenses were $12.5 million for the second quarter of 2022, compared to $19.5 million for the same period in 2021. The decrease in R&D expenses was primarily a result of lower employee-related costs and lower clinical spend related to the Huntingtons disease program, as well as lower facilities costs.

G&A Expenses: General and administrative expenses were $7.6 million for the second quarter of 2022, compared to $10.4 million for the same period in 2021. The decrease in G&A expenses was primarily a result of lower employee-related costs, as well as lower facilities costs.

Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2022, were $148.1 million.

Financial Guidance

Voyager expects that its cash, cash equivalents, and marketable securities, together with amounts expected to be received as reimbursement for development costs under the Neurocrine collaboration, will be sufficient to meet Voyagers planned operating expenses and capital expenditure requirements into 2024.

Participation in Upcoming Investor Conferences

BTIG Healthcare Conference, New York City, NY, August 8, 2022

Wedbush Healthcare Conference, Virtual, August 9, 2022

Canaccord Genuity Healthcare Conference, Boston, MA, August 10, 2022

Wells Fargo Healthcare Conference, Boston, MA, September 7, 2022

RW Baird Healthcare Conference, New York City, NY, September 14, 2022

Conference CallThe Voyager Therapeutics leadership team will host a conference call and webcast today at 4:30 p.m. ET to discuss Voyagers prioritized therapeutic pipeline and to provide second quarter 2022 financial and operating results. To access the call, please dial 1-833-634-2276 (domestic) or 1-412-902-4144 (international) and ask for the Voyager Therapeutics earnings call. A live webcast of the call will also be available on the Investors section of the Voyager website at ir.voyagertherapeutics.com, and a replay will be available at the same link approximately two hours after its completion. The replay will be available for at least 30 days following the conclusion of the call.

About the TRACERTM AAV Capsid Discovery PlatformVoyagers TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of AAV capsids with robust penetration of the blood brain barrier and enhanced CNS tropism in multiple species, including non-human primates (NHPs). TRACER generated capsids have demonstrated superior and widespread gene expression in the CNS compared to conventional AAV capsids as well as cell- and tissue-specific transduction, including to areas of the brain that have been traditionally difficult to reach. Separate results have demonstrated the enhanced ability of certain capsids to target cardiac muscle and to de-target the dorsal root ganglia. Voyager is expanding its library of AAV capsids optimized to deliver diverse therapeutic payloads to address a broad range of CNS and other diseases.

About Voyager TherapeuticsVoyager Therapeutics (Nasdaq: VYGR) is leading the next generation of AAV gene therapy to unlock the potential of the modality to treat devastating diseases. Proprietary capsids born from the Voyagers TRACER capsid discovery platform are powering a rich early-stage pipeline of programs and may elevate the field to overcome the narrow therapeutic window associated with conventional gene therapy vectors across neurologic disorders and other therapeutic areas.

voyagertherapeutics.com LinkedIn Twitter

Voyager Therapeuticsis a registered trademark, and TRACER is a trademark, ofVoyager Therapeutics, Inc.

Forward-Looking StatementsThis press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, might, will, would, should, expect, plan, anticipate, believe, estimate, undoubtedly, project, intend, future, potential, or continue, and other similar expressions are intended to identify forward-looking statements.

For example, all statements Voyager makes regarding Voyagers participation in future scientific conferences; Voyagers ability to continue to identify and develop proprietary capsids from its TRACER capsid discovery platform with increased transgene expression, increased blood-brain barrier penetration and increased biodistribution compared to conventional AAV9 and AAV5 capsids; Voyagers ability to utilize its novel proprietary capsids in its own product development programs; Voyagers ability to attract parties to license its novel proprietary capsids or to participate with Voyager in research and development collaborations utilizing its novel proprietary capsids; Voyagers ability to advance its AAV-based gene therapy programs; the preclinical and clinical development and regulatory status of the Companys product candidates; Voyagers ability to develop its tau antibody program; the size of potential markets for Voyagers product candidates; Voyagers scientific approach, including its ability to demonstrate efficient clinical proof-of-biology and/or proof-of-mechanism for its programs; Voyagers ability to perform its obligations under its respective license option agreements with Novartis and Pfizer; Voyagers entitlement to receive upfront, option exercise, milestone and royalty-based fees from Novartis and Pfizer under the respective license option agreements; Voyagers ability to maintain its current partnerships and collaborations and to enter into new partnerships or collaborations; the timing and suitability of Dr. Mackeys election to Voyagers Board of Directors; Voyagers anticipated financial results, including the receipt by Voyager of revenues or reimbursement payments from collaboration partners; and Voyagers ability to generate sufficient cash resources to enable it to continue to identify and develop proprietary capsids from its TRACER capsid discovery platform are forward-looking.

All forward-looking statements are based on estimates and assumptions by Voyagers management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, the final acceptance by the organizers of upcoming scientific conferences; the ability of Voyager scientists to effectively deliver their presentations at upcoming scientific conferences; the ability ofDr. Mackeyto perform her duties as member of the Board; Voyagers ability to manage the financial and human resources challenges arising from the COVID-19 health crisis; the continued development of various technology platforms, including Voyagers TRACER platform; the development by third parties of capsid identification platforms that may be competitive to Voyagers TRACER capsid discovery platform; Voyagers scientific approach and general development progress; Voyagers ability to attract and retain talented contractors and employees to continue the development of the TRACER capsid discovery platform and the identification of proprietary capsids; Voyagers ability to create and protect intellectual property rights associated with the TRACER capsid discovery platform and the capsids identified by the platform; the response of the FDA and other regulators to Voyagers regulatory submissions and communications; the ability to attract and retain talented contractors and employees, including key scientists and business leaders; the ability to create and protect intellectual property; Voyagers ability to perform its obligations under its license option agreements and its counterparties respective abilities to perform their obligations under such agreements; the sufficiency of cash resources; the initiation, timing, conduct, and outcomes of Voyagers preclinical studies and clinical trials; the possibility or the timing of the exercise of development, commercialization, license and other options under the Pfizer and Novartis license option agreements and other collaborations; the ability of Voyager to negotiate and complete licensing or collaboration agreements on terms acceptable to Voyager and third parties; and the availability or commercial potential of Voyagers product candidates.

These statements are also subject to a number of material risks and uncertainties that are described in Voyagers most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, as updated by its subsequent filings with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Selected Financial Information($ amounts in thousands, except per share data)(Unaudited)

Three Months Ended

June 30,

Statement of Operations Items:

2022

2021

Collaboration revenue

$

712

$

1,357

Operating expenses:

Research and development

12,527

View original post here:
Voyager Therapeutics Prioritizes Pipeline and Reports Second Quarter 2022 Financial and Operating Results - Yahoo Finance

PTC Therapeutics, Inc., announced that the European Commission has granted marketing authorization for Upstaza (eladocagene exuparvovec), the first approved gene therapy treatment developed for aromatic L-amino acid decarboxylase (AADC) deficiency. Upstaza is manufactured in partnership with MassBiologics of UMass Chan Medical School, the only nonprofit, FDA-licensed manufacturer of vaccines, biologics and viral vector gene therapies in the United States. The European Commissions approval clears the regulatory pathway for this first-of-its-kind treatment to be available to patients. PTCs announcement can be read here.

In partnership with PTC Therapeutics, MassBiologics provided contract manufacturing, process development and registration support.

We are proud to contribute to PTC Therapeutics drive to bring this much needed treatment to market, said Mireli Fino, MBA, executive vice chancellor of MassBiologics. Upstaza is one of only a handful of gene therapy treatments to be approved, worldwide, to improve the lives of patients diagnosed with a range of diseases. Our partnership with PTC Therapeutics builds upon our long history and mission of bringing Medicines for Better Lives.

AADC deficiency is a fatal genetic disorder that typically causes severe disability and suffering from the first months of life, affecting every aspect of lifephysical, mental and behavioral. AADC is exceptionally rare and the lives of affected children are severely impacted and shortened.

Stuart W. Peltz, PhD, chief executive officer, PTC Therapeutics, called the approval momentous for patients, for PTC, as well as for the larger gene therapy community. We are proud to bring this innovative therapy to the marketplace so that patients may benefit. Upstaza is the first and only approved disease-modifying treatment for patients living with AADC deficiency. We are ready to deliver this long-awaited treatment to patients as soon as possible.

Read more from the original source:
PTC Therapeutics announces European Commission marketing authorization for Upstaza - UMass Medical School

Additional Libmeldy reimbursement discussions advancing across Europe with product sales totaling $3.1M in Q2 and $8.2M YTD

OTL-200 U.S. BLA filing for MLD on track for late 2022 / early 2023

Seven abstracts accepted at SSIEM highlight potential of HSC gene therapy platform to address difficult-to-treat neurodegenerative disorders

Ended Q2 with $170.9M of cash and investments and runway into 2024; R&D expenses in Q2 declined 22% from the prior quarter

BOSTON and LONDON, Aug. 04, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced recent business highlights along with its financial results for the second quarter and six-months ended June 30, 2022.

Our work in the second quarter has honed our focus to areas where we believe HSC gene therapy is scientifically and clinically positioned to address difficult-to-treat neurodegenerative conditions, such as our marketed therapy for MLD, Libmeldy, said Bobby Gaspar, M.D., Ph.D., chief executive officer. Commercially, we are focused on growing Libmeldy revenues in Europe and neighboring regions via multiple initiatives that will expand patient access and treatment while also preparing for our upcoming regulatory activities in the U.S.

Dr. Gaspar continued, Highlights from our clinical portfolio will be presented at the upcoming SSIEM meeting, including data from our neurometabolic programs that continue to demonstrate the unique ability of our approach to enable broad distribution of gene-corrected cells and localized delivery of therapeutic enzymes and proteins in the brain and in the case of MPSIH, in other organ systems.

Libmeldy Commercial Activities

Chief commercial officer Braden Parker commented, Were very encouraged by the progress on our primary launch initiatives in Europe including a prioritized reimbursement strategy, investments in patient identification and newborn screening and targeted geographic expansion. Our work in these areas is part of a comprehensive growth strategy to optimize Libmeldys potential that we expect to benefit from not only in 2022, but globally over the next three to five years.

Upcoming Data Publications

Key Upcoming Clinical and Research Milestones

Orchard is executing against the following list of upcoming expected milestones:

Second Quarter 2022 Financial Results

Revenue from product sales was $3.8 million for the three months ended June 30, 2022, which included $3.1 million in product sales of Libmeldy and $0.6 million in product sales of Strimvelis. For the six months ended June 30, 2022, revenue from product sales of Libmeldy totaled $8.2 million. The cost of product sales was $1.1 million during the second quarter of 2022. Cost of product sales includes the cost of manufacturing, royalties to third parties and non-cash amortization.

Collaboration revenue was $0.6 million for the three months ended June 30, 2022, resulting from the collaboration of OTL-105 in HAE with Pharming Group N.V. entered into in July 2021. This revenue represents reimbursements for research costs and preclinical studies incurred by the company and a portion of the $17.5 million upfront consideration received by Orchard under the collaboration, which is being amortized into revenue over the expected duration of the agreement.

Research and development expenses were $22.0 million for the three months ended June 30, 2022, compared to $21.8 million in the same period in 2021. R&D expenses remained flat for the period compared to the prior year and declined 22% versus the first quarter of 2022. The company expects our research and development expenses to decline further during the remainder of 2022 as a result of portfolio updates and workforce reduction undertaken during the first half of 2022. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and milestone payments under the companys agreements with third parties, and personnel costs to support these activities.

Selling, general and administrative expenses were $13.7 million for the three months ended June 30, 2022, compared to $14.3 million in the same period in 2021. The decline resulted primarily fromthe realization of savings from the restructuring announced in March 2022, partially offset by increasing commercialization expenses to support a potential U.S. launch of Libmeldy in 2023, and third-party payments related to current sales of Libmeldy during the second quarter.

The company expects to continue to realize the benefits from the March 2022 restructuring and expects any savings to be fully reflected in its operating expenses by year end 2022.

The loss from operations was $32.4 million in the second quarter of 2022 compared to a loss from operations of $36.0 million in the corresponding period of 2021. The reduction in operating loss resulted primarily from revenue from product sales and the collaboration with Pharming in 2022 compared to 2021.

During the quarter, the company had losses on foreign currency transactions of approximately $18.2 million, which were driven primarily by balances denominated in foreign currencies that are subject to exchange rate fluctuations. Since the beginning of 2022, the U.S. dollar has strengthened against the British pound and euro resulting in these unrealized losses.

Net loss was $50.9 million for the three months ended June 30, 2022, compared to $36.6 million in the same period in 2021. The company had approximately 126.4 million ordinary shares outstanding as of June 30, 2022.

Cash, cash equivalents and investments as of June 30, 2022, were $170.9 million, with $33.0 million of debt outstanding, compared to $220.1 million and the same debt figure as of December 31, 2021. During the first half of 2022, the company received approximately $18.5 million in cash from refundable tax credits related to qualifying expenditures in 2020 and 2019, which partially offset the cash used for operating activities during the first six months of 2022. The company expects that its existing cash, cash equivalents and investments will fund its anticipated operating, debt service and capital expenditure requirements into 2024.

About Libmeldy / OTL-200

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

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

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

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

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

About Orchard Therapeutics

At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard is advancing a pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard Therapeutics

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

Forward-looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, and expects, or similar expressions, which are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, the therapeutic potential of Orchards products and product candidates, including the products and product candidates referred to in this release, Orchards ability to secure agreements to gain access and reimbursement for its products in additional countries, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including the product candidates referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, the likelihood of approval of such product candidates by the applicable regulatory authorities, the size of the potential markets for Libmeldy and Orchards product candidates, the expected benefits to Orchards business as a result of the organizational updates referred to in this release, the adequacy of the companys manufacturing capacity and plans for future investment, and the companys financial condition and cash runway into 2024. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that Orchard will not realize the anticipated benefits of its new strategic plan or the expected cash savings; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the risk of delays in Orchards ability to commercialize its product candidates, if approved; the risk that the ongoing and evolving COVID-19 pandemic could affect the company's business; and the risk that the market opportunity for Libmeldy and its product candidates may be lower than estimated or that Orchard may be unable to identify patients for its products on a consistent basis. 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 most recent annual or quarterly report filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

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

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

Contacts

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

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

Excerpt from:
Orchard Therapeutics Reports Second Quarter 2022 Financial Results and Reviews Recent Business Highlights - GlobeNewswire

When coronary arteries are blocked, starving the heart of blood, there are good medications and treatments to deploy, from statins to stents. Not so for heart failure, the leading factor involved in heart disease, the top cause of death worldwide.

Its whats on death certificates, said cardiologist Christine Seidman.

Seidman has long been interested in heart muscle disorders and their genetic drivers. She studies heart failure and other conditions that affect the myocardium the muscular tissue of the heart not the blood vessels where atherosclerosis and heart attacks come from, although their consequences are also felt in the myocardium, including heart failure.

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With her colleagues at Brigham and Womens Hospital and Harvard Medical School, she and a long list of international collaborators have been exploring the genetic underpinnings of heart failure. Based on experiments deploying a new technique called single-nucleus RNA sequencing on samples from heart patients, on Thursday they reported in Science their discovery of how genotypes change the way the heart functions.

Their work raises the possibility that some of the molecular pathways that lead to heart failure could be precisely targeted, in contrast to treating heart failure as a disease with only one final outcome.

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Were not there yet, but we certainly have the capacity to make small molecules to interfere with pathways that we think are deleterious to the heart in this setting, she said. To my mind, thats the way to drive precision therapeutics. We know the cause of heart failure. We intervene in a pathway that we know is activated. And for the first time, we have that information now from human samples, not from an experimental model.

Seidman talked with STAT about the research, including how snRNAseq solves the smoothie problem, and what it might mean for patients. The conversation has been edited for clarity and brevity.

What happens in heart failure?

The heart becomes misshapen in one of two ways. It either becomes hypertrophied, where the walls of heart muscle become thickened and the volume within the heart is diminished, in what we call hypertrophic cardiomyopathy. Or it becomes dilated, when the volume in the heart is expanded and the walls become stretched. I think of it as an overinflated balloon, and that is called dilated cardiomyopathy.

Hypertrophy and dilatation are known to cause the heart over time to have profoundly diminished functional capacity. And clinically, we call that heart failure, much more commonly arising from dilated cardiomyopathy.

What does it feel like to patients?

When we see patients clinically, theyre short of breath, they have fluid retention. When we look at their hearts, we see that the pump function is diminished. That has led to a hypothesis of heart failure as sort of the end stage of many different disorders, but eventually the heart walks down a final common pathway. Then you need a transplant or a left ventricular assist device, or youre going to die prematurely.

What can be done?

Heart failure is a truly devastating condition, and it can arise early in life, in middle age, and in older people. There is no treatment for it, no cure for it, except cardiac transplantation, of course, which provides a whole host of other problems.

How did you approach this problem?

One of the questions we wanted to answer is, are there signals that we can discern that say there are different pathways and there are molecules that are functioning in those pathways that ultimately converge for failure, but through different strategies of your heart?

We treat every patient with heart failure with diuretics. We give them a series of different medications to reduce the pressure against which the heart has to contract. Im clinically a cardiologist, but molecularly Im a geneticist, so it doesnt make sense. If your house is falling down because the bricks are sticking together or if its falling down because the roof leaks and the water is pooling, you do things differently.

Tell me how you used single-cell RNA sequencing to learn more.

Looking at RNA molecules gives us a snapshot of how much a gene is active or inactive at a particular time point. Until recently, we couldnt do that in the heart because the approach had been to take heart tissue, grind it all up, and look at the RNAs that are up or down. But that gives you what we call a smoothie: Its all the different component cells those strawberries, blueberries, bananas mixed together.

But theres a technology now called single-cell RNA sequencing. And that says, what are the RNAs that are up or down in the cardiomyocytes as compared to the smooth muscle cells, as compared to the fibroblasts, all of which are in the cells? You get a much more precise look at whats changing in a different cell type. And thats the approach that we use, because cardiomyocytes [the cells in the heart that make it contract] are very large. Theyre at least three times bigger than other cells. We cant capture the single cell it literally does not fit through the microfluidic device. And so we sequenced the nuclei, which is where the RNA emanates from.

What did you find?

There were some similarities, but what was remarkable was the degree of differences that we saw in cardiomyocytes, in endothelial cells, in fibroblasts. Theres a signature thats telling us I walked down this pathway as compared to a different one that caused the heart to fail, but through activation or lack of activation of different signals along the way.

And that to me is the excitement, because if we can say that, we can then go back and say, OK, what happens if we were to have tweaked the pathway in this genotype and a different pathway in a different genotype? Thats really what precision therapy could be about, and thats where we aim to get to.

Whats the next step?

It may be that several genotypes will have more similarities as compared to other genotypes. But understanding that, I think, will allow us to test in experimental models, largely in mice, but increasingly in cellular models of disease, in iPS [induced pluripotent stem] cells that we can now begin to use molecular technologies to silence a pathway and see what that does to the cardiomyocytes, or silence the fibroblast molecule and see what that does in that particular genotype.

To my mind, thats the way to drive precision therapeutics. We know the cause of heart failure. We intervene in a pathway that we know is activated. And for the first time, we have that information now from human samples, not from an experimental model.

What might this mean for patients?

If we knew that an intervention would make a difference thats where the experiments are we would intervene when we saw manifestations of disease. So the reason I can tell you with confidence that certain genes cause dilated cardiomyopathy is theres a long time between the onset of that expansion of the ventricle until you develop heart failure. So theres years for us to be able to stop it in its tracks or potentially revert the pathology, if we can do that.

What else can you say?

I would be foolish not to mention the genetic cause of dilated cardiomyopathy. Ultimately, if you know the genetic cause of dilated cardiomyopathy, this is where gene therapy may be the ultimate cure. Were not there yet, but we certainly have the capacity to make small molecules to interfere with pathways that we think are deleterious to the heart in this setting.

My colleagues have estimated that approximately 1 in 250 to 1 in 500 people may have an important genetic driver of heart muscle disease, cardiomyopathy. Thats a huge number, but not all of them will progress to heart failure, thank goodness. Around the world, there are 23 million people with heart failure. Its what ends up on most peoples death certificate. It is the most common cause of death.

Its a huge, huge burden. And there really is no cure for it except transplantation. We dont have a reparative capacity, so were going to have to know a cause and be able to intervene precisely for that cause.

Excerpt from:
New research digs into the genetic drivers of heart failure, with an eye to precision treatments - STAT