Category Archives: Gene therapy

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

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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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:

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

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.

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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

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

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

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

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

Gene therapy is making a world where cancer and AIDS can be cured, and a person can alter their genetic makeup, changing the direction of their own and their offsprings evolution in the process. However, the effects of gene therapy are long-lasting, and may impact both your and the health of your future children.

What Is Gene Therapy?

Utilizing a gene or genes to treat or cure a disease or medical condition is known as gene therapy. Gene therapy frequently involves replacing a damaged gene in a patients cells with a healthy copy or inserting new copies of a damaged gene.

Instead of drugs or surgery, gene therapy procedures allow doctors to treat a problem by changing a persons genetic composition. The first gene therapy technique, also known as gene transfer or gene addition, was created to:

Add a new gene to cells to aid in treating a disease

Introduce a healthy gene copy to replace the disease-causing altered copy

Later research helped to advance gene therapy methods. A more recent method, genome editing, takes a different tack in addressing genetic discrepancies. Genome editing uses molecular tools to alter the DNA already present in cells rather than introducing new genetic material. Research on genome editing aims to:

Restore a genes normal function

Correct the genetic change that underlies an illness

Activate a gene that isnt working correctly

Eliminate a section of DNA that interferes with gene activity and causes illness

However, gene therapy is still a field that primarily exists in research labs, and its application is still being tested. The majority of trials take place in Europe, US, and Australia. The treatment is extensive and is being tested to be used to treat acquired genetic diseases like cancer and some viral infections like AIDS, as well as diseases brought on by recessive gene defects, including cystic fibrosis, hemophilia, muscular dystrophy, and sickle cell anemia.

Types of Gene Therapy

Gene therapy comes in various forms, including:

Gene editing: Gene editing aims to remove undesirable genes or fix mutated genes

Cellular gene therapy: The patients cells are taken out, genetically altered (typically via a viral vector), and then put back in

Plasmid DNA: It is possible to genetically modify circular DNA molecules to deliver healing genes into human cells

Viral vectors: Certain gene therapy items are made from viruses because they naturally possess the potential to introduce genetic material into cells. These altered viruses can be employed as vectors to transport therapeutic genes into cells once viruses have been altered to reduce their capacity to spread infectious diseases

Ethical Issues in Gene Therapy

The idea of genetically altering genes has long been the subject of contentious debate in the scientific community. When new techniques are developed, bioethics is always present to evaluate the procedures hazards and moral ramifications. Genetic therapy in somatic cells is widely accepted in the scientific community, particularly in severe diseases like cystic fibrosis and Duchenne muscular dystrophy.

For instance, the first experiment for modifying healthy human embryos was allowed in the United Kingdom. On the other hand, American research organizations remained conservative, restating their stance that they do not support this kind of trial and saying that they must wait for advancements in both the methods and the definitions of ethical issues.

View original post here:
What to Know About Gene Therapy - Biotech - HIT Consultant

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Systemic gene therapy with thymosin 4 alleviates glomerular injury in mice | Scientific Reports - Nature.com

New York, July 15, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Gene Therapy Market by Type of Therapy, Type of Gene Delivery Method Used, Type of Vector Used, Target Therapeutic Areas, Route of Administration, and Key Geographical Regions : Industry Trends and Global Forecasts, 2022-2035" - https://www.reportlinker.com/p06292885/?utm_source=GNW To provide more context, the treatment regimen of such therapies, encompassing gene replacement and gene-editing modalities, is aimed at correction of the mutated gene in patients using molecular carriers (viral and non-viral vectors). Further, post the onset of the COVID-19 pandemic, there has been a steady increase in the investigational new drug (IND) applications filed for cell and gene therapies. In fact, in 2021, more than 200 gene therapies were being evaluated in phase II and III studies. Moreover, in 2022, six gene therapies are expected to receive the USFDA market approval. Promising results from ongoing clinical research initiatives have encouraged government and private firms to make investments to support therapy product development initiatives in this domain. In 2021 alone, gene therapy developers raised around USD 9.5 billion in capital investments. Taking into consideration the continuous progress in this domain, gene therapies are anticipated to be used for the treatment of 1.1 million patients suffering from a myriad of disease indications, by 2035.

Presently, more than 250 companies are engaged in the development of various early and late-stage gene therapies, worldwide. In recent years, there has been a significant increase in the integration of novel technologies, such as gene modification, gene-editing, genome sequencing and manipulation technologies (molecular switches), in conjugation with gene delivery methods. For instance, the CRISPR-Cas9 based gene-editing tool is one of the remarkable technological advancements, which enables the precise alteration of the transgene. It is worth mentioning that the new generation delivery platforms, including nanoparticles and hybrid vector systems, have been demonstrated to be capable of enabling effective and safe delivery of gene based therapeutics. Further, a variety of consolidation efforts are currently ongoing in this industry. Such initiatives are primarily focused on expanding and strengthening the existing development efforts; this can be validated from the fact that 56% of the total acquisitions reported in the domain were focused on drug class consolidation. Driven by the collective and consistent efforts of developers and the growing demand for a single dose of effective therapeutic, the gene therapy market is anticipated to witness significant growth in the foreseen future.

SCOPE OF THE REPORTThe Gene Therapy Market (5th Edition) by Type of Therapy (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Delivery Method Used (Ex vivo and In vivo), Type of Vector Used (Adeno-associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Non-Viral Vectors, Retrovirus and Others), Target Therapeutic Areas (Cardiovascular Diseases, Dermatological Diseases, Genetic Diseases, Hematological Diseases, Infectious Diseases, Metabolic Diseases, Muscle-related Diseases, Oncological Diseases, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebral, Intracoronary, Intradermal, Intralesional, Intramuscular, Intrapleural, Intrathecal, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal, Topical and Others), and Key Geographical Regions (US, Europe, Asia-Pacific and rest of the world): Industry Trends and Global Forecasts, 2022-2035 report features an extensive study of the current market landscape and the likely future potential associated with the gene therapy market, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies.

Amongst other elements, the report features:A detailed overview of the overall market landscape of gene therapies, including information on their phase of development (marketed, clinical, preclinical and discovery), key therapeutic areas (autoimmune diseases, cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, hepatic diseases, immunological diseases, infectious diseases, inflammatory diseases, metabolic diseases, muscle-related diseases, neurological diseases, oncological diseases, ophthalmic diseases and others), target disease indication(s), type of vector used, type of gene / molecule targeted, type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), type of gene delivery method used (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any).A detailed overview of the current market landscape of players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain.An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors.A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies.An elaborate discussion on the various commercialization strategies that have been adopted by drug developers engaged in this domain across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing stage.Detailed profiles of marketed and late stage (phase II / III and above) gene therapies, along with information on the development timeline of the therapy, current development status, mechanism of action, affiliated technology, patent portfolio strength, dosage and manufacturing details, as well as details related to the developer company.A review of the various emerging technologies and therapy development platforms that are being used to manufacture gene therapies, featuring detailed profiles of technologies that were / are being used for the development of four or more products / product candidates.An in-depth analysis of various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2017, based on several relevant parameters, such as type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed / granted), and patent valuation.A detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2022, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product, target therapeutic area and deal multiples.An analysis of the investments made at various stages, such as seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings, by companies that are engaged in this domain.An analysis of completed, ongoing and planned clinical studies, based on several relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.An analysis of the various factors that are likely to influence the pricing of gene therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies.An analysis of the startup companies engaged in this domain (established between 2017-2022) based on year of experience.A detailed review of the various gene therapy-based initiatives undertaken by big pharma players, highlighting trend across parameters, such as number of gene therapies under development, funding information, partnership activity and patent portfolio strength.An informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.A case study on the prevalent and emerging trends related to vector manufacturing, along with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors.A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, over the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2022-2035. Our year-wise projections of the current and future opportunity have further been segmented on the basis of [A] type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), [B] type of gene delivery method used (ex vivo and in vivo), [C] type of vector used (adeno-associated virus, adenovirus, herpes simplex virus, lentivirus, non-viral vectors, retrovirus and others), [D] target therapeutic areas (cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, infectious diseases, metabolic diseases, muscle-related diseases, oncological diseases, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebral, intracoronary, intradermal, intralesional, intramuscular, intrapleural, intrathecal, intratumoral, intravenous, intravesical, intravitreal, subretinal, topical and others), and [F] key geographical regions (US, Europe, Asia-Pacific and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industrys growth.

The opinions and insights presented in this study were Influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals:Buel Dan Rodgers (Founder and CEO, AAVogen)Sue Washer (President and CEO, AGTC)Patricia Zilliox (President and CEO, Eyevensys)Christopher Reinhard (CEO and Chairman, Gene Biotherapeutics (previously known as Cardium Therapeutics))Adam Rogers (CEO, Hemera Biosciences)Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela))Al Hawkins (CEO, Milo Biotechnology)Jean-Phillipe Combal (CEO, Vivet Therapeutics)Robert Jan Lamers (former CEO, Arthrogen)Tom Wilton (former CBO, LogicBio Therapeutics)Michael Triplett (former CEO, Myonexus Therapeutics)Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics)Cedric Szpirer (Executive and Scientific Director, Delphi Genetics)Marco Schmeer (Project Manager) and Tatjana Buchholz (former Marketing Manager, PlasmidFactory)Jeffrey Hung (CCO, Vigene Biosciences)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

RESEARCH METHODOLOGYThe data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

Th secondary sources of information include:Annual reportsInvestor presentationsSEC filingsIndustry databasesNews releases from company websitesGovernment policy documentsIndustry analysts views

While the focus has been on forecasting the market till 2035, the report also provides our independent view on various emerging trends in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market, gathered from various secondary and primary sources of information.

KEY QUESTIONS ANSWEREDWho are the key industry players engaged in the development of gene therapies?How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products?Which types of vectors are most commonly used for effective delivery of gene therapies?What are the key regulatory requirements for gene therapy approval, across various geographies?Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development?What are the different pricing models and reimbursement strategies currently being adopted for gene therapies?What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies?Who are the key CMOs / CDMOs engaged in supplying viral / plasmid vectors for gene therapy development?What are the key value drivers of the merger and acquisition activity in the gene therapy industry?Who are the key stakeholders that have actively made investments in the gene therapy domain?Which are the most active trial sites (in terms of number of clinical studies being conducted) in this domain?How is the current and future market opportunity likely to be distributed across key market segments?

CHAPTER OUTLINES

Chapter 2 provides an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.

Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It further highlights the different types of gene therapies (namely somatic and germline therapies, and ex vivo and in vivo therapies), potential application areas of such products and route of administration of these therapeutic interventions. In addition, it provides information on the concept of gene editing, highlighting key historical milestones, applications and various techniques used for gene editing. The also chapter includes a discussion on the various advantages and disadvantages associated with gene therapies. Further, it features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.

Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages and limitations of currently available vectors.

Chapter 5 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, Australia, China, Hong Kong, Japan and South Korea. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.

Chapter 6 includes information on over 1150 gene therapies that are currently approved or are in different stages of development. It features a detailed analysis of the therapies, based on several relevant parameters, such as key therapeutic areas (autoimmune diseases, cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, hepatic diseases, immunological diseases, infectious diseases, inflammatory diseases, metabolic diseases, muscle-related diseases, neurological diseases, oncological diseases, ophthalmic diseases and others), target disease indication(s), phase of development (marketed, clinical, preclinical and discovery), type of vector used, type of gene / molecule targeted, type of gene delivery method used (ex vivo and in vivo), type of therapy (gene augmentation, oncolytic viral therapy, immunotherapy and others), route of administration and special drug designation (if any). Further, we have presented a grid analysis of gene therapies based on phase of development, therapeutic area and type of therapy.

Chapter 7 provides a detailed overview of the current market landscape of players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. Further, we have presented a logo landscape of product developers in North America, Europe, Asia-Pacific, and Middle East and North Africa region on the basis of company size.

Chapter 8 provides detailed profiles of marketed gene therapies. Each profile includes information about the innovator company, its product pipeline (focused on gene therapy only), development timeline of the therapy, its mechanism of action, target indication, current status of development, details related to manufacturing, dosage and sales, the companys patent portfolio and collaborations focused on its gene therapy product / technology.

Chapter 9 features an elaborate discussion on the various strategies that have been adopted by therapy developers engaged in this domain across key commercialization stages, including prior to drug launch, during drug launch and post-launch stage. In addition, it presents an in-depth analysis of the key commercialization strategies that have been adopted by developers of gene therapies approved during the period 2015-2022.

Chapter 10 provides detailed profiles of drugs that are in advanced stages of clinical development (phase II / III and above). Each drug profile provides information on the development timeline of the therapy, current developmental status, route of administration, developers, primary target indication, special drug designation received, target gene, dosage, mechanism of action, affiliated technology, patent portfolio strength, clinical trials and collaborations (if any).

Chapter 11 provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. In addition, it features brief profiles of some of the key technologies. Each profile features details on the various pipeline candidates that have been / are being developed using the technology, its advantages and the partnerships that have been established related to the technology platform. Further, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.

Chapter 12 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include genetic diseases, metabolic diseases, neurological diseases, oncological diseases and ophthalmic diseases.

Chapter 13 provides an overview of the various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2017, based on several relevant parameters, such as type of patent, publication year, regional applicability, CPC symbols, emerging areas and leading industry players (in terms of number of patents filed / granted). In addition, it features a competitive benchmarking analysis of the patent portfolios of leading industry players and patent valuation.

Chapter 14 features a detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2022, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product, target therapeutic area and deal multiples.

Chapter 15 presents details on various funding instances, investments and grants reported within the gene therapy domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and secondary offerings) received by the companies between 2015 and 2022, highlighting the growing interest of the venture capital community and other strategic investors in this market.

Chapter 16 presents an analysis of completed, ongoing and planned clinical studies, based on several relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.

Chapter 17 highlights our views on the various factors that may be taken into consideration while deciding the price of a gene therapy. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt in order to decide the price of its proprietary products.

Chapter 18 presents a detailed analysis of the start-up companies engaged in the field of gene therapy, established between 2017-2022, based on year of experience.

Chapter 19 provides a detailed review of the various gene therapy-based initiatives undertaken by big pharma players, highlighting trend across parameters, such as number of gene therapies under development, funding information, partnership activity and patent portfolio strength. In addition, it also a detailed analysis of the big pharma players based on several parameters, such as therapeutic area, type of vector used, type of therapy and type of gene delivery method used.

Chapter 20 features an informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.

Chapter 21 presents an elaborate market forecast analysis, highlighting the future potential of the market till the year 2035. It also includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II / III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of [A] type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), [B] type of gene delivery method used (ex vivo and in vivo), [C] type of vector used (adeno-associated virus, adenovirus, herpes simplex virus, lentivirus, non-viral vectors, retrovirus and others), [D] target therapeutic area (cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, infectious diseases, metabolic diseases, muscle-related diseases, oncological diseases, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebral, intracoronary, intradermal, intralesional, intramuscular, intrapleural, intrathecal, intratumoral, intravenous, intravesical, intravitreal, subretinal, topical and others), and [F] key geographical regions (US, Europe, Asia-Pacific and rest of the world).

Chapter 22 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. In addition, it features the challenges that exist in this domain. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.

Chapter 23 provides a glimpse of the gene therapy supply chain. It discusses the steps for implementing a robust model and provides information related to the global regulations for supply chain. Moreover, the chapter discusses the challenges associated with supply chain of gene therapies. In addition, it features the technological solutions that can be adopted for the management of gene therapy supply chain.

Chapter 24 summarizes the overall report, wherein we have mentioned all the key facts and figures described in the previous chapters. The chapter also highlights important evolutionary trends that were identified during the course of the study and are expected to influence the future of the gene therapy market.

Chapter 25 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interviews held with Buel Dan Rodgers (Founder and CEO, AAVogen), Sue Washer (President and CEO, AGTC), Patricia Zilliox (President and CEO, Eyevensys), Christopher Reinhard (CEO and Chairman, Gene Biotherapeutics (previously known as Cardium Therapeutics)), Adam Rogers (CEO, Hemera Biosciences), Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela)), Al Hawkins (CEO, Milo Biotechnology), Jean-Phillipe Combal (CEO, Vivet Therapeutics), Robert Jan Lamers (former CEO, Arthrogen), Tom Wilton (former CBO, LogicBio Therapeutics), Michael Triplett (former CEO, Myonexus Therapeutics), Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics), Cedric Szpirer (Executive and Scientific Director, Delphi Genetics), Marco Schmeer (Project Manager) and Tatjana Buchholz (former Marketing Manager, PlasmidFactory), and Jeffrey Hung (CCO, Vigene Biosciences). In addition, a brief profile of each company has been provided.

Chapter 26 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 27 is an appendix, which contains a list of companies and organizations mentioned in this report.Read the full report: https://www.reportlinker.com/p06292885/?utm_source=GNW

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Original post:
Gene Therapy Market by Type of Therapy, Type of Gene Delivery Method Used, Type of Vector Used, Target Therapeutic Areas, Route of Administration, and...