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

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

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

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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Bluebird CFO resigns ahead of gene therapy launch - BioPharma Dive

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

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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SOURCE Research and Markets

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

French biotech company WhiteLab Genomics has raised $10 million in funding for an AI platform designed to aid the discovery and development of genomic therapies.

Founded out of Paris in 2019, recent Y Combinator (YC) graduate WhiteLab Genomics provides gene and cell therapy companies with predictive software simulations to expedite the design of gene and cell therapies. Gene therapy, for the uninitiated, is an emerging treatment that involves replacing missing or defective genes in cells to correct genetic disorders, while cell therapy is about altering a cell or sets of cells to trigger an effect throughout the body.

Thousands of diseases, including cystic fibrosis, Parkinsons and Alzheimers stem from flaws in an individuals DNA, and emerging research in gene and cell therapies may eventually treat such conditions at their source, supplanting the need for drugs or surgery.

However, such therapies are typically costly to develop with no guarantee that theyll work. Current methodologies in developing new gene and cell therapies typically involves a trial-and-error approach, according to WhiteLab Genomics CEO and co-founder David Del Bourgo, whereby scientists make a scientific hypothesis and test it in a lab: if successful, it progresses to the next stage, but if its unsuccessful, they go back to square one with a different hypothesis. And this is where WhiteLab Genomics enters the fray, with a computational approach that meshes machine learning and deep learning techniques to process multiple scientific hypotheses at once, looking at different genetic variants to predict the best molecular design for the therapy based on the objectives.

Gene and cell therapies still suffer from poor efficacy, immunological secondary effects and very high development cost, Del Bourgo explained to TechCrunch. We provide customers with exhaustive predictive models combining genetics and computational biology in order to design and select the top candidates to be tested in the lab.

WhiteLab Genomics founders: CEO David Del Bourgo and Chief Science Officer Julien Cottineau. Image Credits: WhiteLab Genomics

As with just about any AI models, WhiteLab Genomics uses myriad datasets to train its algorithms spanning genomic, RNA, oncologic and protein repositories, in addition to an exhaustive set of documentation such as public scientific and clinical research data.

We also partner with specialized institutions to enrich the models with additional non-public data, Del Bourgo added. We train and validate our algorithms on well-characterized datasets that are interconnected within our Knowledge Graph Database. These included public databases, proprietary data and datasets from our partners. It predicts biological features from new genetic sequences to build new therapeutic vectors and generates new therapeutic constructs.

In terms of the kinds of treatments WhiteLab Genomics is helping to develop, Del Bourgo said that the company is currently working on projects including DNA-based therapies for metabolic conditions such as lysosomal diseases, as well as cell stem therapies for blood diseases such as sickle cell disease and immuno-gene therapies to treat cancers.

Elsewhere, anumber of companies are using AI to aid drug discovery, such as BenevolentAI, which has raised sizeable investments from the VC world, while theres TechCrunchs Startup Battlefield winner Cellino, which recently raised $80 million to develop cell therapies using AI. WhiteLab Genomics is similar, but its working with both gene and cell therapy companies.

Its also worth noting that WhiteLab Genomics represents a growing roster of French startups to successfully infiltrate the YC ranks. Of the 40 European companies in WhiteLab Genomics Winter 22 cohort, five were French, a figure that rose to eight of 34 for the Summer 22 tranche the second highest number from the whole of Europe.

With $10 million in the bank from French investor Omnes Capital and biopharmaceutical heavyweight Debiopharm, WhiteLab Genomics is now well-financed to build out its platform and double-down on its partnerships with the scientific realm, which so far has included notable collaborations with the French National Institute of Health and Medical Research (Inserm) and Genethon Laboratories, among other pharmaceutical and biotech companies.

Originally posted here:
How WhiteLab Genomics is using AI to aid gene and cell therapy development - TechCrunch

TumorGen and PhenoVista Biosciences partnered toexplore metastatic cancer cell clustersto validate technology targeting these clusters and prevent cancer metastasis, which causes 90% of cancer-related deaths.

Using PhenoVistas analytical imaging technology, the two companies, whopartnered last year, reported TumorGens micro-fluidic technology platform was able to detect multiple metastatic clusters from several lung cancer patients. The platform showed tremendous sensitivity and specificity, which proved to be more effective than previous efforts to collect and characterize the clusters.

The clusters contained cancerous and non-cancerous cells, James Evans, CEO of PhenoVista, noted in the announcement. The companies announced the advancements could lead to improved therapeutic options for these patients.

The study, which the National Cancer Institute supported, notes that these metastasized clusters can be captured. This will allow for multiple analyses that could identify weaknesses and potential therapeutic approaches, which TumorGen dubbed cluster busters. This therapeutic could then be able to target the cells and prevent metastasis.

Jeffrey K. Allen, founder and president of TumorGen, noted in the companys announcement that there is a tremendous unmet medical need for anti-metastatic therapies that can prevent tumors from forming across the body. He said the companys platform is able to identify where these metastatic cancer cell clusters are vulnerable, enabling the development of those needed therapeutics.

He told BioSpace the company is energized to pursue therapeutic targets that can lead to a whole new class of anti-metastatic drugs that will save lives.

Our next steps will be to expand our efforts beyond lung cancer to other solid tumor types, all of which have been shown to release circulating metastatic cancer cell clusters, he said. We have also begun discussions with a biopharmaceutical company about beginning to use our technology to characterize MCCCs, confirm biomarkers of interest, and evaluate therapeutic targets within their pipeline.

When it comes to looking for a partner, Allen said TumorGen hopes to pair up with a company that is looking to differentiatethemselves in the oncology space by focusing on stopping metastatic disease before it spreads.

Allen said that the majority of cancer therapies remain focused on the primary tumor, while the different mechanismscontrolling metastasis remain unaddressed. Since MCCCs are often heterogeneousand may contain immune cells that protect them from immune surveillance, Allen said they hope to find a partner interested in investigating targeting more than just the cancer cells within the cluster.

Gene Therapy Developed for Rare Blindness Disease

Researchers from theNational Institutes of Health havedeveloped a potential new gene therapy approachto Leber congenital amaurosis (LCA), a disease that causes blindness in children.

A research team from the National Eye Institute, a division of the NIH, revealed that mutations cause a type of LCA in the NPHP5 gene, which leads to defects in the primary cilium found in cells throughout the body,Technology Networksreported. A deficiency in NPHP5 can cause blindness in children. However, the team explained that deficiency could lead to kidney disease in severe cases.

Defects in 25 different genes can cause LCA. The FDA has approved Spark Therapeutics gene therapy Luxturna for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, a form of LCA. That leaves other forms of this disease without treatment.

Using stem cells collected from two patients with NPHP5 deficiency, the NIH team generated retinal organoids, which possess functional features of the natural retina. A deficiency of NPHP5 was found in the retinal organoids, along with lower levels of the protein CEP-290, which works alongside NPHP5.

Using an adeno-associated viral vector that contained a functional version of NPHP5, the NIH researchers were able to target these extracted stem cells and demonstrate a significant restoration of opsin protein concentrated in the proper location in outer segments. The NIH research warrants further study and suggests a functional protein could prevent blindness in these children.

Further research is warranted, particularly in more severe forms of the disease.

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Cluster-busters against Cancer and a Potential New Gene Therapy for LCA - BioSpace

Wilmington, Delaware, United States, Transparency Market Research Inc. CAR T cell therapy is considered as an extremely effective strategy for the treatment of patients experiencing blood malignant growth and has effectively experienced other methods of disease treatment like chemotherapy. The effectiveness of the cycle in treating disease is probably going to fuel growth of the global CAR-T cell therapy market in the years to come.

Cancer is one of the main sources of fatalities around the world. Illusory antigen receptor or CAR-T cell therapy is used for fighting cancer cells, and includes re-designing of the white platelets of the cancer-affected patient. While CAR-T cell therapy, The T-cells of an individual are inferred, changed, and the put inside the body of the person where the restructured cells multiply themselves in number and afterward attack the cancer cells.

The development of the CAR-T cell therapy market worldwide is predicted to gather momentum from the increasing number of investors, designers, and analysts in the guide therapy. Rising frequencies of cancer across the globe together with the relentless technological advancement for dependable and effective cancer treatment is expected to support growth of the worldwide CAR-T cell therapy market. Furthermore, heavy investments on clinical research and development in cell therapy are estimated to boost the market for CAR-T cell therapy in the years to come.

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Global CAR-T cell therapy Market: Overview

Cancer is one of the leading causes of fatalities worldwide. Chimeric antigen receptor or CAR-T cell therapy comprises re-engineering of WBC or the white blood cells of the cancer-affected patient. This is fight off cancer cells. During the time of CAR-T cell therapy, the T-cells of a person are derived, reformed, and the put inside the body of a human being wherein the restructured cells multiply in number and then attack the cancer cells.

Currently, CAR T cell therapy is considered as an extremely effective procedure for the treatment of patients suffering from blood cancer and has already been through other methods of cancer treatment such as chemotherapy. The effectiveness of the process in treating cancer is likely to fuel growth of the global CAR-T cell therapy market in the years to come.

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Rising incidences of cancer across the globe together with the relentless technological advancement for reliable and effective cancer treatment is expected to favor growth of the global CAR-T cell therapy market over the period of projection, from 2019 to 2027. Furthermore, growing number of clinical studies in cell therapy is estimated boost the market over the period of forecast.

This report on the global CAR-T cell therapy market offers a closer look at the significant changes that took place in the industry of medical science. The developments are likely to shape the contours of the global CAR-T cell therapy market over the assessment tenure, from 2019 to 2027. The insights offered in the report are estimated to help the industry players and investors to make a correct assessment of the completive ecosystem and make strategies accordingly.

Global CAR-T cell therapy Market: Trends and Opportunities

Rising Geriatric Population with Weakening Immune System Boost Market Growth

The global CAR-T cell therapy market is primarily driven by increasing number of acquisitions, thanks to the augmented importance of cellular therapies. In addition to that, some of the acquisitions are done to facilitate more innovative research and development activities, which further boosts the global CAR-T cell therapy market in near future. A case in point is the leading role played by both Novartis AG and Gilead Sciences, Inc. following the approval of first therapies in pediatric Acute Lymphoblastic Leukemia and adult Diffuse Large B-cell Lymphoma or DLBCL.

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Scientists, investors, and developers agree unequivocally that solution to two major problems can offer long-term success in this field. One of them is the recognition of antigens except CD19 that could be efficaciously targeted with CAR-T cell therapy. The other problem lies in the indications of tumor, which lies beyond liquid cancers. The development of the global CAR-T cell therapy market is predicted to garner momentum from the rising interest of investors, developers, and researchers in the aid therapy.

Global CAR-T cell therapy Market: Regional Outlook

The five important regions in the global CAR-T cell therapy market include North America, Europe, the Middle East and Africa, Latin America, and Asia Pacific.

North America is estimated to take lead of the global CAR-T cell therapy market over the projection period. The dominance of the region is ascribed to the favorable policies of reimbursement, growing incidences of severe lymphoblastic leukemia, early availability of products of CAR-T cell therapy, and high per capita expenditure on healthcare. In addition, presence of a large number of clinical pipeline drugs that are expected to get approval for commercialization in near future is likely to fuel expansion of the global CAR-T cell therapy market.

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Global CAR-T cell therapy Market: Companies Mentioned in Report

Right now, only two companies namely Novartis AG and Gilead Sciences, Inc. have already initiated the process of commercializing their products of CAR-T cell therapy. These two companies jointly hold most of the global CAR-T cell therapy market. However, there are products of other companies that are lying in the pipeline for approval. Other market players comprise Amgen, Inc., Juno Therapeutics, Eli Lilly and Company, Fate Therapeutics, Inc., Sorrento Therapeutics, and Johnson & Johnson.

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CAR-T Cell Therapy Market Segmentation and Forecast Analysis up to 2027 - BioSpace

Ori is focused on bringing automated manufacturing to the cell and gene therapy (CGT) industry, while the CTMCis a joint venture between National Resilience Inc and MD Anderson Cancer Center.

The alliance is aimed at speeding up process development, clinical implementation, and commercialization of novel cell therapies.

It will leverage Oris technology platform with CTMCs manufacturing capabilities and, in the first instance, will focus on a cell therapy invented by Dr Sattva Neelapu, professor of lymphoma/myeloma at MD Anderson.

Ori and CTMC have specifically partnered with the goal of increasing patient access to potentially life-saving cell therapies. This partnership will have a considerable impact on the cell therapy field by implementing innovative, digitally enabled process discovery and automated manufacturing solutions, said Jason C Foster, CEO, Ori Biotech.

Last week we reported on how the CGT manufacturing tech innovator had expanded its global footprint and team, with it executing its strategic plan following the US$100m it raised in its Series B funding round earlier this year.

Ori has invested in high-level expertise, in personnel, having recruited across digital infrastructure, data analytics, software, manufacturing, and quality, and in facilities globally, with the goal of rapid transition into the launch of its CGT manufacturing platform.

The developer has boosted its senior leadership team with several new appointments, including three directors, to support further growth and push the commercialization of its platform, along with further expanding its Science Advisory Board (SAB), appointing CGT experts, Isabelle Rivire and Jason Bock.

It has also opened a new facility in Princeton, New Jersey, US and will be opening a new technology center, in Cambridge, UK.

Commenting on those new recruits and SAB members, Foster told BioPharma-Reporter: As recent data demonstrates, there is an urgent need for increased access to cell and gene therapies, with less than 2% of eligible patients globally gaining access to marketed cell therapies and 20% of those patients who are selected for treatment still die while waiting for their treatment to be manufactured. It is imperative that we address these access issues quickly and there is no time to waste.

"Our new team members add to our strengths across all domains including biology, engineering, data science, manufacturing, supply chain and commercial. Typically, it has taken 10+ years for manufacturing platforms like Oris to get to market, we hope to bring the first generation of our platform to market in early 2024, less than five years post our first round of funding in 2019.

The CEO said the companys Lightspeed Early Access Program (LEAP) is an important step toward commercial launch. It is a very exciting time as we have several partners directly engaging with our beta platform and testing it with their protocols.

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Ori Biotech and CTMC team up on cell therapy delivery - BioPharma-Reporter.com