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Category Archives: Gene therapy

KSQ Therapeutics Announces Leadership and Board Additions – Business Wire

Posted: September 25, 2022 at 2:12 am

LEXINGTON, Mass.--(BUSINESS WIRE)--KSQ Therapeutics, a clinical-stage biotechnology company developing therapies to treat cancer and autoimmune diseases using its proprietary, integrated discovery CRISPRomics platform, today named Micah Benson, PhD as the new Chief Scientific Officer (CSO) and announced the addition of a new Chief Technology Officer (CTO), Tom Leitch, to the KSQ leadership team. Dr. Benson previously served as Senior Vice President, Immunobiology for KSQ and has been a catalyst for the companys adaptive cell therapy programs. He will succeed Frank Stegmeier, PhD, who served as KSQ's founding CSO, who is leaving to pursue a new opportunity. Dr. Stegmeier will join KSQ's Board of Directors. Board member Pearl Huang, PhD, is stepping down from the board.

"Micah's work leading our Immunobiology Team has been invaluable in advancing our engineered TIL (eTIL) programs, and his appointment as CSO comes at a critical time, as we move those programs towards the clinic. We're also thrilled to have Tom on board as our new CTO, as he brings deep experience in cell & gene therapy, manufacturing sciences, CMC strategy development, manufacturing operations, quality, and supply chain," said Qasim Rizvi, Chief Executive Officer of KSQ Therapeutics. "I would also like to thank Frank for his contributions to KSQ since the companys founding. Frank's scientific leadership has shaped and guided KSQ into the company it is today, and we're thrilled he will remain an advisor and member of our Board of Directors. Similarly, I would like to thank Pearl for her guidance over the last three years. I wish her the best in future endeavors."

"KSQ's CRISPRomics platform has the potential to change the foundation of how we treat a wide variety of cancers and autoimmune diseases and has already generated multiple high impact programs that are either in or nearing clinical trials. I look forward to continuing to advance our science and leading our fantastic team," said Dr. Benson.

KSQ has made great strides advancing its CRISPRomics platform, identifying important disease targets and pioneering therapies to target them. Im impressed by the tenacity and incredible hard work of this team, and Im energized to work closely with them to realize our goal of developing transformative therapies for the patients that we all serve, said Leitch.

Micah Benson, PhD, Chief Scientific Officer

Micah has over 15 years of academic and industry experience as an immunologist and drug hunter. Prior to joining KSQ, Micah served as Head of Tolerance Therapeutics in the Immunology and Inflammation Research Unit at Pfizer, Inc. He earned his PhD in Immunology from Dartmouth Medical School and was a postdoctoral fellow at Harvard Medical School. He has authored several patents and published extensively in top-tier scientific journals.

Tom Leitch, Chief Technology Officer

Tom brings more than 20 years of leadership experience in cell and gene therapy, biologics, and vaccines. His experience spans manufacturing sciences, CMC strategy development, internal and external manufacturing operations, quality, engineering, tech transfer, and supply chain across a broad range of leading biopharmaceutical companies.

Leitch came to KSQ from bluebird bio, where, as head of Manufacturing, he led the development and execution of the company's manufacturing strategy during a period of rapid growth that expanded the network to include more than ten internal and external manufacturing sites around the world. Before bluebird, Tom held roles at Alexion and Merck. He holds M.S. and B.S. degrees in engineering from Virginia Tech.

About KSQ Therapeutics

KSQ Therapeutics is advancing a pipeline of tumor- and immune-focused drug candidates to treat cancer and autoimmune disease across multiple drug modalities, including targeted therapies, adoptive cell therapies, and immunotherapies. KSQ's proprietary CRISPRomics discovery engine enables genome-scale, in vivo validated, unbiased drug discovery across broad therapeutic areas. For more information, please visit the company's website at http://www.ksqtx.com and follow @ksq_tx on Twitter.

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KSQ Therapeutics Announces Leadership and Board Additions - Business Wire

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Hemophilia gene therapies from BioMarin, CSL pick up an early ICER endorsement – FiercePharma

Posted: September 16, 2022 at 2:08 am

  1. Hemophilia gene therapies from BioMarin, CSL pick up an early ICER endorsement  FiercePharma
  2. BioMarin reports cancer case in hemophilia gene therapy trial  BioPharma Dive
  3. ICER Deems Hemophilia Gene Therapies Worth the $2.5M Price Tag  BioSpace
  4. BioMarin touts report on cost effectiveness of gene therapy Roctavian for hemophilia A  Seeking Alpha
  5. BioMarin says PhIII gene therapy study participant's cancer is unrelated to therapy  Endpoints News
  6. View Full Coverage on Google News

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Challenges In Gene Therapy – University of Utah

Posted: September 16, 2022 at 2:08 am

A good gene therapy is one that will last. Ideally, an introduced gene will continue working for the rest of the patient's life. For this to happen, the introduced gene must become a permanent part of the target cell's genome, usually by integrating, or "stitching" itself, into the cell's own DNA. But what happens if the gene stitches itself into an inappropriate location, disrupting another gene?

This happened in two gene therapy trials aimed at treating children with X-linked Severe Combined Immune Deficiency (SCID). People with this disorder have virtually no immune protection against bacteria and viruses. To escape infections and illness, they must live in a completely germ-free environment.

Between 1999 and 2006, researchers tested a gene therapy treatment that would restore the function of a crucial gene, gamma c, in cells of the immune system. The treatment appeared very successful, restoring immune function to most of the children who received it.

But later, 5 of the children developed leukemia, a blood cancer. Researchers found that the newly transferred gamma c gene had stitched itself into a gene that normally helps regulate the rate at which cells divide. As a result, the cells began to divide out of control, causing leukemia. Doctors treated 4 of the patients successfully with chemotherapy, but the fifth died.

This unfortunate incident raised important safety concerns, and researchers have since developed safer ways to introduce genes. Some newer vectors have features that target DNA integration to specific "safe" places in the genome where it won't cause problems. And genes introduced to cells outside of the patient can be tested to see where they integrated, before they are returned to the patient.

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Gene Therapy Restores Some Vision in Colorblind Children – Pharmacy Times

Posted: September 16, 2022 at 2:08 am

Study suggests gene therapy may effectively treat young children who were born colorblind via pathways connecting the brain and the retina.

Gene therapy was able to partially restore cone function in colorblind children, according to research conducted by the University of London published in Brain. Achromatopsia affects rods and cone cells, both photoreceptors in the eyes. The disease, which is a result of gene variants, prevents cone cells from signaling to the brain and causes colorblindness, poor overall vision, and sensitivity to bright light.

"In our trials, we are testing whether providing gene therapy early in life may be most effective while the neural circuits are still developing. Our findings demonstrate unprecedented neural plasticity, offering hope that treatments could enable visual functions using signaling pathways that have been dormant for years, said co-lead author Michel Michaelides of the UCL Institute of Ophthalmology and Moorfields Eye Hospital, in a press release

Investigators have been researching ways to activate the cone cells and recover color vision. The 2 trials are examining therapies targeting a different gene and to test its safety and efficacy in improving vision.

In the study, the investigators examined 4 individuals aged 10 to 15 years who have achromatopsia. After receiving gene therapy in only 1 eye, investigators evaluated the efficacy of the treatment. Using a functional magnetic resonance imaging (fMRI) brain scan, the study authors pinpointed changes in patient post-treatment visual function by separating their post-treatment cone signals from existent rod-driven signals.

Following the brain scan, the team used a silent substitution technique to separately stimulate rods or cones to test the effects of the treatment. They compared the results of the study with data from 9 untreated patients and 28 patients with normal vision.

Prior to the test, no child had normal cone function on any test. After treatment, the results were similar to those of patients with normal vision. Between 6- and 14-months post-treatment, 2 of the 4 participants showed signs of cone-mediated signals in the visual cortex of the brain from the treated eye.

Those same 2 children also showed a difference in cone-supported vision. After taking a psychophysical test, their results showed that the treated eye could see multiple levels of contrast.

"Our study is the first to directly confirm widespread speculation that gene therapy offered to children and adolescents can successfully activate the dormant cone photoreceptor pathways and evoke visual signals never previously experienced by these patients, said lead author Tessa Dekker, of the UCL Institute of Ophthalmology, in a press release.

At the time of this study, researchers were conducting additional phase 1 and 2 trials to test the neural pathways of the cones.

"We are still analyzing the results from our two clinical trials, to see whether this gene therapy can effectively improve everyday vision for people with achromatopsia. We hope that with positive results, and with further clinical trials, we could greatly improve the sight of people with inherited retinal diseases," Michaelides said in the press release.

Reference

University College London. Gene therapy partly restores cone function in two completely colorblind children. Science Daily. August 24, 2022. Accessed on September 2, 2022.

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Gene Therapy Restores Some Vision in Colorblind Children - Pharmacy Times

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Hopkinton bioscience company forms advisory board for cell and gene therapy – Worcester Business Journal

Posted: September 16, 2022 at 2:08 am

Two bioscience companies, including one based in Hopkinton, have formed a scientific advisory board to provide insight in development and manufacturing of advanced therapies.

Lykan Bioscience, a cell-based therapies contract development and manufacturing services provider based in Hopkinton, has partnered with RoslinCT of the United Kingdom to develop a scientific advisory board comprised of pioneers in the cell and gene therapy spaces.

The board will work closely with members of the management teams of both companies to provide insight into a wide range of topics across the development and manufacturing of advanced therapies, according to a Tuesday press release from the companies.

Members include Michael Chambers, cofounder of North Dakota-based Aldevron; Heidi Hagen, chief technology officer at Sonoma Biotherapeutics in California and Washington; Margit Jeschke, an independent consultant and expert on the analytical development strategy for cell and gene therapy; and Michael Kalos, founder and managing director at Next Pillar Consulting, LLC in Pennsylvania.

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Coave Therapeutics partners with IMN on gene therapy – Labiotech.eu

Posted: September 16, 2022 at 2:08 am

Coave Therapeutics is collaborating with the Institute of Neurodegenerative Diseases (IMN) of Bordeaux to develop gene therapy proteins targeting protein degradation in neurodegenerative disorders.

The IMN is a joint research unit associating the University of Bordeaux and the French National Centre of Scientific Research (CNRS) developing gene therapy programs targeting protein degradation in neurodegenerative disorders.

The collaboration will explore the development of gene therapy products targeting the transcription factor EB (TFEB) for the treatment of alpha-synucleinopathies, such as Multiple System Atrophy (MSA) and idiopathic Parkinsons disease (PD).

TFEB is a master regulator of the autophagy lysosomal pathway, a central cellular pathway controlling the degradation of toxic protein aggregates. Overexpression of TFEB via gene therapy demonstrates potential to reduce and prevent the accumulation of toxic protein aggregates1and to consequently prevent neurodegeneration.

Erwan Bzard an IMN research director, a member pf Coave Therapeutics Scientific Advisory Board (SAB) and Andrea Ballabio, scientific director at the Telethon Institute of Genetics and Medicine (TIGEM) also a member of Coave Therapeutics SAB, have authored a paper. It demonstrates a reduction of a-synuclein aggregates, prevention of dopaminergic neuron destruction and recovery of clinical phenotype.

Bzard said: Targeting the autophagy lysosomal pathway by using coAAV based gene therapy is a unique approach to address neurodegenerative diseases, such as Parkinsons disease or Multiple System Atrophy.

Thanks to the partnership with Coave Therapeutics, IMN scientists have an important opportunity to confirm their scientific findings towards the clinic. We look forward to leveraging our collective strengths to best develop gene therapy programs for neurodegenerative diseases with the potential to improve patient outcomes.

Dr Ballabio, who discovered the role of TFEB as a master regulator of lysosomal biogenesis and autophagy will be an advisor to this collaboration.

Rodolphe Clerval, CEO, Coave Therapeutics said: We are delighted to be collaborating with IMN to develop coAAVs carrying TFEB and explore these gene therapy constructs for the treatment of neurodegenerative diseases.

TFEB is an exciting target and we look forward to working with IMN and Andrea Ballabio to evaluate the effect of our novel gene therapies from our ALIGATER platform for the treatment of MSA and PD, with the potential to develop further programs.

Under the collaboration, Coave Therapeutics will use its AAV-Ligand Conjugate (ALIGATER) platform to design, develop and manufacture coAAV viral vectors carrying the TFEB gene for targeted delivery to deep brain structures.

IMN will be responsible for carrying out jointly designed in vivo studies to evaluate the effect of the gene therapy products in animal models of MSA and PD. The collaboration aims to generate further in vivo proof of concept data and enable the selection of therapeutic candidates to enter IND enabling studies.

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MeiraGTx’s Industry-Leading Gene Therapy Manufacturing Facility in Shannon, Ireland Formally Introduced by Head of Irish Government -…

Posted: September 16, 2022 at 2:08 am

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--Manufacturing facilities built to accelerate development and delivery of advanced medicines to patients using state-of-the-art technology at scale, with quality appropriate for commercialization

--Site reflects MeiraGTxs unique, end-to-end approach to gene therapy manufacturing to expedite clinical development

LONDON and NEW YORK, Sept. 16, 2022 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (NASDAQ: MGTX), a vertically integrated, clinical stage gene therapy company, will today formally unveil its GMP manufacturing facility in Shannon, Ireland, with the Head of Irish Government, Michel Martin, in attendance.

The facility, online since earlier this year and stretching over 150,000 square feet, is the first commercial-scale gene therapy manufacturing site in Ireland. The facility is unique in its scale and integrated capabilities. The site contains three facilities, one built to be flexible and scalable for viral vector production for clinical and commercial supply, in addition, a facility to manufacture plasmid DNA the critical starting material for producing gene therapy products and thirdly, a Quality Control (QC) hub performing advanced biochemical quality control testing for MeiraGTx clinical and commercial programs.

The formal unveiling marks a critical step in MeiraGTxs mission to develop and deliver potentially curative treatments for patients living with serious diseases. The Shannon site allows MeiraGTx to accelerate the development and delivery of gene therapy treatments to patients facing a wide range of both genetic and non-hereditary disorders ranging from inherited vision loss, salivary-gland conditions, and neurological diseases such as Parkinson's, to potentially diabetes, obesity, and some cancers.

By building end-to-end gene therapy development, testing and manufacturing capabilities in-house, MeiraGTx has put in place the infrastructure and technology required to avoid bottlenecks in clinical development, reduce regulatory risk, and ensure the highest quality products for patients all while lowering costs. The facility will also allow MeiraGTx the ability to provide manufacturing services to potential collaborators, helping to lessen the impact of industry-wide shortages of vital elements such as plasmid DNA and quality control services.

The facility, which is set to employ 100 people in its current phase, with the potential for that to increase to over 300, has been sited in Shannon due to its proximity to a number of world-class bioscience institutions, as well as partner companies in the healthcare sector. MeiraGTx is proud to have collaborated with The University of Limerick and the National Institute for Bioprocessing Research and Training (NIBRT) on skills and capability development and looks forward to building ever-closer relationships with other leading institutions across Ireland.

During the unveiling today, the Head of the Irish Government will meet senior leaders from MeiraGTx, undertake a walking tour of the facility and make a short address to invited guests.

Head of Irish Government, Michel Martin, said:

Today is an important day for MeiraGTx in Ireland, as we mark the formal unveiling of their state-of-the-art manufacturing facility. Great strides are being made in the area of gene therapy and Ive no doubt that these new facilities will allow MeiraGTx to remain at the forefront of that development. Todays unveiling is testament to the companys continued commitment to Shannon and the Mid-West and speaks to the considerable skills and ability of the Irish workforce.

Alexandria Forbes, Ph.D., President, and Chief Executive Officer of MeiraGTx, said:

MeiraGTxs Shannon facility is unique, not only in Ireland but globally, as it streamlines gene therapy development, testing and manufacturing capabilities together in-house. This significantly reduces the time to patients for advanced therapeutic products, with months or potentially years saved. Along with our other facilities in New York, London and Amsterdam, the Shannon site will scale up and manufacture a broad range of gene therapies for people living with a variety of serious conditions.

Alastair Leighton, Ph.D., Senior Vice President of Manufacturing and Supply Chain at MeiraGTx, said:

The COVID-19 pandemic has placed a strain on the global gene therapy industry to manufacture critical components, as well as exposed the shortcomings in the supply chain. While vaccines are not gene therapies, they share many of the same manufacturing processes and resources. The Shannon facility has been designed to address these challenges in order to provide access to transformative potential medicines to patients as well as be ready for significant future expansion.

CEO of IDA Ireland Martin Shanahan said:

In 2020, MeiraGTx announced its intention to locate this globally unique GMP manufacturing facility in Shannon. So, its terrific that two years later we can celebrate this official unveiling. IDA Ireland remains committed to winning jobs and investments across the country and MeiraGTxs decision to locate in the Mid-West demonstrates the regions reputation as a key location for the next generation of biopharmaceutical manufacturers."

About MeiraGTx

MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage gene therapy company with six programs in clinical development and a broad pipeline of preclinical and research programs. MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, and a transformative gene regulation platform technology which allows precise, dose responsive control of gene expression by oral small molecules with dynamic range that can exceed 5000-fold. Led by an experienced management team, MeiraGTx has taken a portfolio approach by licensing, acquiring, and developing technologies that give depth across both product candidates and indications. MeiraGTxs initial focus is on three distinct areas of unmet medical need: ocular diseases, including both inherited retinal diseases as well as large degenerative ocular diseases, neurodegenerative diseases and severe forms of xerostomia. Though initially focusing on the eye, central nervous system, and salivary gland, MeiraGTx plans to expand its focus to develop additional gene therapy treatments for patients suffering from a range of serious diseases.

For more information, please visitwww.meiragtx.com

Forward Looking Statement

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding our product candidate development and the use of our manufacturing facilities in Shannon, Ireland, including in light of the COVID-19 pandemic, as well as statements that include the words expect, will, intend, plan, believe, project, forecast, estimate, may, could, should, would, continue, anticipate and similar statements of a future or forward-looking nature. These forward-looking statements are based on managements current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, our incurrence of significant losses; any inability to achieve or maintain profitability, raise additional capital, repay our debt obligations, identify additional and develop existing product candidates, successfully execute strategic priorities, bring product candidates to market, expansion of our manufacturing facilities and processes, successfully enroll patients in and complete clinical trials, accurately predict growth assumptions, recognize benefits of any orphan drug designations, retain key personnel or attract qualified employees, or incur expected levels of operating expenses; the impact of the COVID-19 pandemic on the status, enrollment, timing and results of our clinical trials and on our business, results of operations and financial condition; failure of early data to predict eventual outcomes; failure to obtain FDA or other regulatory approval for product candidates within expected time frames or at all; the novel nature and impact of negative public opinion of gene therapy; failure to comply with ongoing regulatory obligations; contamination or shortage of raw materials or other manufacturing issues; changes in healthcare laws; risks associated with our international operations; significant competition in the pharmaceutical and biotechnology industries; dependence on third parties; risks related to intellectual property; changes in tax policy or treatment; our ability to utilize our loss and tax credit carryforwards; litigation risks; and the other important factors discussed under the caption Risk Factors in our most recent quarterly report on Form 10-Q or annual report on Form 10-K or subsequent 8-K reports, as filed with the Securities and Exchange Commission. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, unless required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Thus, one should not assume that our silence over time means that actual events are bearing out as expressed or implied in such forward-looking statements. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

Contacts

Investors: MeiraGTx [emailprotected]

Media: Jason Braco, Ph.D. LifeSci Communications [emailprotected]

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Bluebird CFO resigns ahead of gene therapy launch – BioPharma Dive

Posted: September 16, 2022 at 2:08 am

Bluebird bio on Monday said its chief strategy and financial officer, Jason Cole, will leave the company this fall, marking the second time in less than a year the cash-strapped gene therapy developer will replace its top financial executive.

In a statement, Bluebird said that Cole is resigning to pursue other opportunities. Cole has been with the company for more than eight years, most recently as its chief business officer, and took on the CFO role in March, after Gina Consylman resigned.

Hell be replaced by Katherine Breedis, an executive from Danforth Advisors, on an interim basis while Bluebird searches for a full-time replacement.

Coles departure comes just as Bluebird is preparing to launch its gene therapy Zynteglo for the inherited blood disease beta thalassemia in the U.S. The drug, which the Food and Drug Administration approved last month, is priced at $2.8 million, making it one of the worlds most expensive pharmaceutical products on a single-use basis and a test of the commercial potential of gene therapies.

Bluebird has said it will refund up to 80% of the treatments price for two years if the treatment doesnt work as expected. It estimates that the cost of lifetime treatments for beta thalassemia, which include chronic blood transfusions, can exceed $6 million.

The sales trajectory of Zynteglo, along with a second product called eli-cel that the FDA could approve this week, are critical to Bluebirds future. The company entered 2022 in financial peril following a string of clinical, regulatory and commercial setbacks. Its been looking to the launches of Zynteglo and eli-cel, and the special, sellable vouchers the FDA could award upon their approvals, to bolster its dwindling cash reserves.

But Bluebird took some additional steps to shore up its balance sheet under Coles tenure as well. In April, the company announced plans to lay off 30% of its workforce. The following month, it began moving into a new headquarters.

We are extremely grateful to Jason for stepping in as CFO during a difficult time for the company. His experience and thoughtful approach have been a stabilizing force through a period of significant change, said CEO Andrew Obenshain, adding the company has solidified [its] financial position and financing plans.

Coles last day will be on Oct. 14, though hell consult for the company on corporate strategy through next April.

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Enrollment completed in STARLIGHT Phase 2 clinical trial of MCO-010 optogenetic gene therapy for Stargardt disease – Ophthalmology Times

Posted: September 16, 2022 at 2:08 am

Nanoscope Therapeutics Inc. today announced full enrollment of its Phase 2 clinical trial of MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic monotherapy to restore vision in blind patients, for Stargardt disease. Six-month data from the Phase 2 STARLIGHT trial are expected in H1 2023.

"We are excited by the therapeutic potential of MCO-010, supported by robust data from previous preclinical and clinical studies," Sulagna Bhattacharya, CEO of Nanoscope, said in a statement. "Completing the quick two-month enrollment of this Phase 2 trial, which will evaluate the safety and effects of a single intravitreal injection of MCO-010, brings us another major step forward in developing this novel therapy with broad therapeutic application."

The Phase 2 STARLIGHT open-label trial (NCT05417126) enrolled 6 subjects with advanced vision loss due to a clinical or genetic diagnosis of Stargardt disease. In this study, all subjects received the same single intravitreal dose of 1.2E11gc/eye of MCO-010 as used in the Company's Phase 2b retinitis pigmentosa (RP) study.

"Stargardt disease is a blinding condition affecting adults and children alike. It is the most common macular dystrophy and has no established treatment," said Byron Lam, MD, Robert Z. & Nancy J. Greene Chair and a professor of Ophthalmology at the University of Miami and principal investigator in the Phase 2 STARLIGHT trial. "MCO-010 has the potential to improve visual function in RP and Stargardt patients, and we look forward to seeing the data from this study."

Nanoscope's optogenetic therapy uses a proprietary AAV2 vector to deliver MCO genes into retinal cells to enable vision in different color environments. The therapy is administered as a single intravitreal injection for in-office delivery without the need for any other devices or interventions. MCO-010 has received orphan drug designations for RP and Stargardt disease from the FDA and is concurrently being evaluated in Nanoscope's Phase 2b RESTORE trial in patients with RP, with trial results also expected in H1 2023.

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Enrollment completed in STARLIGHT Phase 2 clinical trial of MCO-010 optogenetic gene therapy for Stargardt disease - Ophthalmology Times

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Global Cancer Gene Therapy Market to Reach $11.35 Billion by 2030 at a CAGR of 23.3% – PR Newswire

Posted: September 16, 2022 at 2:08 am

DUBLIN, Sept. 14, 2022 /PRNewswire/ --The "Cancer Gene Therapy Market By Therapy, By End User: Global Opportunity Analysis and Industry Forecast, 2020-2030" report has been added to ResearchAndMarkets.com's offering.

Cancer Gene Therapy Market was valued at $1,389.42million in 2020 and is estimated to reach $11,359.35 million by 2030, registering a CAGR of 23.3% from 2021 to 2030.

Cancer gene therapy is a technique used for the treatment of cancer where therapeutic DNA is being introduced into the gene of the patient with cancer. Owing to the high success rate during the preclinical and clinical trials, cancer gene therapy has gained popularity.

Many techniques are used for cancer gene therapy, for example, a procedure where the mutated gene is being replaced with a healthy gene or inactivation of the gene whose function is abnormal. Recently, a new technique has been developed, where new genes are introduced into the body to help fight against cancer cells.

The rise in the prevalence of cancer, the benefits of cancer gene therapy over conventional cancer therapies, and the advancement in this field are the major factors that drive the market growth.

In addition, the surge in government support, ethical acceptance of gene therapy for cancer treatment, and rise in biotechnological funding encouraging the R&D activities for cancer gene therapy and thus fuel the growth of the cancer gene therapy market.

In addition rise in awareness regarding cancer gene therapy is a major factor that drives the global cancer gene therapy market growth.

In addition, an increase in government support for research in gene therapy, ethical acceptance of gene therapy for cancer treatment, and a rise in the prevalence of cancer boost the growth of the cancer gene therapy market. However, the high cost associated with the treatment and unwanted immune responses is expected to restrain the market growth.

Key Benefits For Stakeholders

Key Market SegmentsBy Therapy

By End User

By Region

Key Market Players

Key findings of the Study

Key Topics Covered:

CHAPTER 1: INTRODUCTION

CHAPTER 2: EXECUTIVE SUMMARY

CHAPTER 3: MARKET OVERVIEW

CHAPTER 4: CANCER GENE THERAPY MARKET, BY THERAPY

CHAPTER 5: CANCER GENE THERAPY MARKET, BY END USER

CHAPTER 6: CANCER GENE THERAPY MARKET, BY REGION

CHAPTER 7: COMPANY LANDSCAPE

CHAPTER 8: COMPANY PROFILES

For more information about this report visit https://www.researchandmarkets.com/r/kb14ky

Media Contact:

Research and MarketsLaura Wood, Senior Manager[emailprotected]

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

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Global Cancer Gene Therapy Market to Reach $11.35 Billion by 2030 at a CAGR of 23.3% - PR Newswire

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