Category Archives: Genetic Therapy

Albany NY, United States: The rising prevalence of chronic conditions such as cancer and other cell-based diseases may bring considerable growth prospects for the cell analysis instruments market during the forecast period of 2021-2031. In addition, the rising awareness among the government bodies of numerous countries regarding the importance of cell-based research activities are estimated to serve as growth boosters for the cell analysis instruments market between 2021 and 2021.

Cell analysis can be defined as the study of cells isolated from tissues in unicellular and multicellular organisms. It is a popular process to understand the phenotypical and genetic characteristics of organisms. Thus, the advantages associated with cell analysis instruments ay trigger considerable growth opportunities for the global market.

Request a report brochure to gain comprehensive insights at: https://www.transparencymarketresearch.com/sample/sample.php?flag=B&rep_id=2296

The report on the cell analysis instruments market has been prepared with a view to offer exclusive insights to the stakeholders and CXOs. The report has various factors that are beneficial for strengthening the revenues of the players in the cell analysis instruments market. Furthermore, the report also takes into account the various factors that may prove to be a restraint in the growth of the cell analysis instruments market. In addition, the COVID-19 impact is included in the report. The pandemic has changed the growth projections of many sectors and industries. The cell analysis instruments market is no exception. The effect of the COVID-19 pandemic has been described in the report.

Cell Analysis Instruments Market: Competitive Insights

The cell analysis instruments market is highly fragmented. The cell analysis instruments market is a mixture of local and international players. The local players in various regions contribute a lions share to the growth of the cell analysis instruments market. The players frame extensive marketing strategies to generate awareness about their instruments to the end-users. This factor helps in the overall growth of the cell analysis instruments market. Furthermore, the players are also involved in research and development activities. These activities help in discovering formulations and upgrades that add value to the growth trajectory of the cell analysis instruments market.

Request for Covid-19 Impact Analysis: https://www.transparencymarketresearch.com/sample/sample.php?flag=covid19&rep_id=2296

Strategic collaborations are also crucial for the growth of the cell analysis instruments market. The players are involved in mergers, acquisitions, joint ventures, and partnerships for increasing their influence across untapped regions. These factors eventually help increase the growth rate of the cell analysis instruments market.

Some key players into the cell analysis instruments market are Danaher Corporation, Sigma Aldrich, Dickinson and Company, Thermo Fisher Scientific, and Merck KGaA.

Cell Analysis Instruments Market: Key Trends

The extensively developing healthcare sector and the expanding cases of cancer are prognosticated to bring considerable growth opportunities for the cell analysis instruments market during the forecast period of 2021-2031. Furthermore, the advent of technologically advanced products and growing research activities in cell analysis are extrapolated to offer considerable growth opportunities across the cell analysis instruments market.

The expanding use of cell analysis instruments in hospitals, diagnostic laboratories, pharmaceuticals, research institutes, biotechnology companies, and others may bring considerable growth for the global market.

For any Queries Related with the Report, Ask an Analyst: https://www.transparencymarketresearch.com/sample/sample.php?flag=EB&rep_id=2296

Cell Analysis Instruments Market: Regional Perspective

North America is estimated to have a dominant streak in terms of regional growth for the cell analysis instruments market during the forecast period of 2021-2031. The growing incidences of cancer in the region and increased funding in cell-based research may prove to be growth accelerators for the cell analysis instruments market.

Asia Pacific is also estimated to emerge as a prominent region growth contributor for the cell analysis instruments market, The growing awareness about cell analysis instruments across various regions in the region may serve as a prime growth factor for the cell analysis instruments market in Asia Pacific.

More Trending Reports by Transparency Market Research:

Single-Cell Analysis Market: The growing importance of cellular biology and microbiology has created fresh opportunities for growth within the global single-cell analysis market. The dearth of effective technologies to study the microscopic parts of singular cells has led to increased use of existing research apparatus and equipment.

GMP Cell Banking Services Market: A burgeoning pharmaceutical industry hard-pressed to find treatments and cures for various chronic and other ailments has given rise to the concept of cell banking. With limited capacity to store cells required for research in the field of regenerative medicines, somatic cell therapy, gene therapy, and tissue-engineered products they are increasingly outsourcing it to other entities specializing in it.

About Us

Transparency Market Research is a next-generation market intelligence provider, offering fact-based solutions to business leaders, consultants, and strategy professionals.

Our reports are single-point solutions for businesses to grow, evolve, and mature. Our real-time data collection methods along with ability to track more than one million high growth niche products are aligned with your aims. The detailed and proprietary statistical models used by our analysts offer insights for making right decision in the shortest span of time. For organizations that require specific but comprehensive information we offer customized solutions through ad hoc reports. These requests are delivered with the perfect combination of right sense of fact-oriented problem solving methodologies and leveraging existing data repositories.

TMR believes that unison of solutions for clients-specific problems with right methodology of research is the key to help enterprises reach right decision.

Contact

Rohit BhiseyTransparency Market Research

State Tower,

90 State Street,

Suite 700,

Albany NY - 12207

United States

USA - Canada Toll Free: 866-552-3453

Email: sales@transparencymarketresearch.com

Website: https://www.transparencymarketresearch.com/

View original post here:
Cell Analysis Instruments Market: Rising prevalence of chronic conditions to drive the market - BioSpace

BEAM-101 Patient Enrollment, BEAM-102 and BEAM-201 IND Submissions and BEAM-301 IND-Enabling Studies All On-track for Second Half of 2022

Natural History Study Initiated in People with Sickle Trait to Provide Insights into Key Characteristics of Sickle Cell Trait and Sickle Cell Disease

Manmohan Singh, Ph.D., Appointed to Executive Leadership Team, and Anne Marie Woodland Appointed as Head of Regulatory

Ended First Quarter 2022 with $1.2 Billion in Cash, Cash Equivalents and Marketable Securities to Support Advancement of Precision Genetic Medicines Portfolio

CAMBRIDGE, Mass., May 09, 2022 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced pipeline and business highlights and financial results for the first quarter ended March 31, 2022. In addition, as part of a long-term effort to better understand sickle cell disease (SCD), a genetic disease in which individuals carry two copies of the sickle cell mutation, and sickle cell trait, in which individuals carry only one copy of the mutation, Beam will fund and collaborate with the National Alliance of Sickle Cell Centers (NASCC) to initiate the AUNT (Achieving Understanding of the Natural History of Sickle Trait) Study, a natural history study of sickle cell trait.

Base editing has the potential to offer life-changing medicines for a broad range of diseases, and we are committed to better understanding the pathophysiology of the diseases in our pipeline, and their impacts on the lives of patients and their families, said John Evans, chief executive officer of Beam. We are excited to be collaborating with the Globin Research Network for Data and Discovery (GRNDad) on the AUNT natural history study in people with sickle cell trait. We are on track and expect to commence SCD patient enrollment in our Phase 1/2 BEACON-101 clinical trial to evaluate the safety and efficacy of BEAM-101 in patients with SCD, as well as make our planned IND submission for BEAM-102, which is also in development for the treatment of SCD. Our immunology and liver-directed pipelines are also progressing, with plans for additional regulatory submissions, research studies and program nominations throughout the year, and we continue to build upon our delivery capabilities, including our novel LNP technology platform, potentially allowing us to expand the future reach of our programs. We believe the parallel advancement of these diverse programs creates a broad foundation for our future growth and is evidence of our commitment to developing new and better treatments for multiple patient populations and to unlocking the full potential of precision genetic medicine.

Pipeline Highlights & Anticipated MilestonesEx Vivo HSC Programs

Ex Vivo T Cell Programs

In Vivo LNP Liver-targeting Programs

Initiation of AUNT Natural History StudySCD is a severe, inherited blood disorder that alters the structure and function of oxygen-carrying hemoglobin in red blood cells and is caused by a single point mutation in the beta globin gene. Carriers of the disease, or individuals with sickle cell trait, have only one copy of the sickle mutation and produce variable amounts of the abnormal sickle hemoglobin (25-45% of total hemoglobin). Despite the high prevalence of sickle cell trait (estimated to affect 300 million individuals worldwide), research to understand its full biology and clinical features has been limited.

Beam is developing two programs for SCD, BEAM-101 and BEAM-102. BEAM-101 is designed to raise fetal hemoglobin while lowering abnormal sickle hemoglobin to <40% of total, which is similar to levels seen in individuals with sickle cell trait. BEAM-102 is designed to replace the sickle mutation with a normal human variant of hemoglobin, HbG-Makassar, potentially reducing even further the abnormal sickle hemoglobin in patient cells. A better understanding of sickle cell trait, along with an in-depth understanding of SCD, will help better establish the relationship between levels of the abnormal sickle hemoglobin and long-term clinical outcomes.

The AUNT Study will create a first of its kind multi-center, prospective, longitudinal cohort of individuals with sickle trait, targeting a large enrollment of approximately 1,000 participants. This research is designed to establish an understanding of the hematologic and clinical phenotype of people with sickle cell trait, including blood rheology, potential complications, and genetic modifiers, in an effort to better understand the hematologic phenotype that is associated with good health and lack of organ dysfunction, as well as provide increased counseling to people with sickle cell trait.

Business Updates

Upcoming ASGCT Presentation DetailsTitle: Single, systemic administration of BEAM-301 mitigates fasting hypoglycemia and restores metabolic function in a transgenic mouse model of glycogen storage disease type IaDate & Time: Monday, May 16, 2022, from 4:15-4:30 p.m. ET

Title: Optimized base editing reagents yield more potent genetic correction in a mouse model of alpha-1 antitrypsin deficiency (poster M-123)Date & Time: Monday May 16, 2022, from 5:30-6:30 p.m. ET

Title: Efficient LNP delivery of mRNA in vivo and in vitro to T and NK cells (poster Tu-107)Date & Time: Tuesday May 17, 2022, from 5:30-6:30 p.m. ET

First Quarter 2022 Financial Results

About Beam Therapeutics

Beam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beams suite of gene editing technologies is anchored by base editing, a proprietary technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This has the potential to enable a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: the intended design and planned initiation of the AUNT Study; our presentations at ASGCT, our plans, and anticipated timing, to nominate additional development candidates, initiate IND-enabling studies, and submit IND applications; the therapeutic applications and potential of our technology, including with respect to SCD, T-ALL/T-LL, GSDIa, Alpha-1, CAR-T cells and LNPs, including our ability to deliver base editors to target organs in and beyond the liver; the planned initiation and design of our BEACON-101 clinical trial, including the timing of enrolling the first subject in the trial; the sufficiency of our capital resources to fund operating expenses and capital expenditure requirements; and our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the potential impact of the COVID-19 pandemic, including its impact on the global supply chain; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that enrollment of our clinical trials may take longer than expected; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; and the other risks and uncertainties identified under the headings Risk Factors Summary and Risk Factors in our Annual Report on Form 10-K for the year ended December 31, 2021, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.

Contacts:

Investors:Chelcie ListerTHRUST Strategic Communicationschelcie@thrustsc.com

Media:Dan Budwick1ABdan@1abmedia.com

Read more here:
Beam Therapeutics Announces Pipeline and Business Highlights and Reports First Quarter 2022 Financial Results - GlobeNewswire

Five percent of the human population is carrier of some form of disorder of Hemoglobin (Hb) (Hemoglobinopathy) affecting its oxygen carrying capacity in blood, as per to WHO. Prevalence rate for Thalassemia (a type of hemoglobinopathy) mutations have been reported to be as high as 17% by studies from Indian subcontinent. Thalassemia and Sickle cell disease are the most common of Hemoglobinopathies and are inherited in an Autosomal recessive manner, meaning that if both partners are carrier of a mutation in the Hb gene, then the risk of having an child affected with Thalassemia major is about 25%. This risk becomes even more significant in view of the high prevalence of Hb gene mutation carrier status in general population (tribal belts, Sindhis, Parsis, Gujratis and specific pockets in south India), marriages within closed communities and consanguinity (marriages within relation)

Treatment of Thalassemia major is lifelong and includes regular blood transfusions, chelation medicines to tackle the issue of increasing iron content in body, managing any complications such as infections, endocrine resulting hormonal imbalances. While Hematopoietic stem cell transplantation (HSCT) is the only curative therapy available currently for patients with -thalassemia major, it is limited by feasibility, cost and availability of suitable matched donor. HSCT is also associated with potential risk of immune-mediated rejection and graft-versus-host disease (GVHD) in few cases. Gene therapy trails have provided a new impetus in this field.

Routine screening for Hemoglobinopathies is done by Hb electrophoresis of HPLC (high performance liquid chromatography). This helps identify the particular disorder and institute prompt treatment and follow up. However such HPLC has major have limitations as all Hb variants may not be detected by HPLC and when screening the neonates or doing prenatal testing (specific testing done during pregnancy to know if the fetus is affected) as the pattern of functioning Hb gradually shifts from fetal type to adult type by around one year of age. Also blood transfusions may influence the HPLC results. Herein comes the importance of Molecular genetic testing. Hundreds of Hemoglobinopathies causing alterations in the HBB gene have been reported, curated and catalogued in various databases. It must be noted that about 5 common mutations in the HBB gene account for over 90% cases of Thalassemia. Making them the first line of mutations to be tested if suspecting Thalassemia. If these are negative then we proceed with the HBB gene sequencing.

Dr. AnupKumar Rawool, Associate Director, Clinical Genomics, SRL Diagnosticssaid,"Living with Thalessemia is not an easy lifestyle and if not diagnosed correctly or at the right time, can lead to other health problems. We know that Thalessemia and Beta Thalessemia is prevalent in India and there is research underway for better cure, treatment and therapy of the illness. While there are other forms of treatment available, the newest treatment now making waves all over the world is Gene Therapy. We now have studies to prove that Gene Therapy is a viable cure for beta thalessemia. It has therapeutic potential and we are excited to have with us this indispenasable expertise that is is key to good health for countless patients in our country.

"Timely diagnosis of genetic disorders with appropriate Molecular genetic tests provides an optimum window for offering prenatal diagnosis and decision making for the family. It is recommended that the person with thalassemia or any Hemoglobinopathy undergo HBB sequencing test to identify the disease causing alterations in the HBB gene. Once these are identified and confirmed then diagnostic testing during pregnancy can be offered by either Chorionic villi sampling between 11-13 weeks or amniocentesis after 16 completed weeks of gestation, leaving ample time for the molecular genetic lab to perform and report the test and for the clinician, medical geneticist and the family for appropriate pre and post test genetic consultation and decision making accordingly. It is advisable that Planning for prenatal testing to be done prior to pregnancy. Advances in molecular testing techniques also provide option for testing embryos on day 5 by pre-implantation genetic diagnosis (PGD) if availing an option of IVF/ART. So prior screening of embryo can be done for any known genetic disorder in the family. However PGD services are available at limited centres. A prior Genetic consultation with a Medical Geneticist for the families in such situation is strongly recommended to smoothen the entire process.

Timely diagnosis of genetic disorders with appropriate Molecular genetic tests provides an optimum window for offering prenatal diagnosis and decision making for the family. It is recommended that the person with thalassemia or any Hemoglobinopathy undergo HBB sequencing test to identify the disease causing alterations in the HBB gene. Once these are identified and confirmed then diagnostic testing during pregnancy can be offered by either Chorionic villi sampling between 11-13 weeks or amniocentesis after 16 completed weeks of gestation, leaving ample time for the molecular genetic lab to perform and report the test and for the clinician, medical geneticist and the family for appropriate pre and post test genetic consultation and decision making accordingly. It is advisable that Planning for prenatal testing to be done prior to pregnancy. Advances in molecular testing techniques also provide option for testing embryos on day 5 by pre-implantation genetic diagnosis (PGD) if availing an option of IVF/ART. So prior screening of embryo can be done for any known genetic disorder in the family. However PGD services are available at limited centres. A prior Genetic consultation with a Medical Geneticist for the families in such situation is strongly recommended to smoothen the entire process.

(To receive our E-paper on whatsapp daily, please click here. We permit sharing of the paper's PDF on WhatsApp and other social media platforms.)

See the rest here:
International Thalassaemia Day 2022: The genetic perspective of Thalassemia - Free Press Journal

Apertura Developer Michael Greenberg, Ph.D., Chair of Harvard's Department of Neurobiology/Courtesy of Ulf Andersen/Getty Images

With $67 million in Series A financing, newly-launched Apertura Gene Therapy hit the ground with a goal to develop genetic medicines using platform technologies that address key limitations of gene delivery and expression.

Based in New York, Apertura was founded on full-stack platform technologies developed by the Broad Instituteof MIT and Harvardand researchers from Harvard Medical School. The platform is designed to engineer novel capsids, gene regulatory elements and payloads that are able to address key limitations of gene delivery and expression. Developed in the labs of Ben Deverman from the Broad Institute and Harvards Michael Greenberg, the platform harnesses machine learning capabilities to develop next-generation gene therapies that are expected to offer greater translational potential.

Greenberg said one of the key challenges with current gene therapies is ensuring the therapeutic payload is expressed at the correct level in target cells. The technology that is the basis of Aperturas approach overcomes that hurdle by targeting transgene expression to specific cell types, fine-tuning expression levels in these cells, and, at the same time, avoiding expression of the transgene in non-target cell types, Greenberg said in a statement.

When developing a gene therapy, it has been common to use naturally occurring serotype AAV capsids. The technology we have developed uses proprietary assays and machine learning to design custom AAV capsids that have the chosen characteristics for treating specific diseases, and we believe this approach will result in new and effective gene therapies, Deverman said of the Broad Institute,which is the scientific founder of Apertura.

Dave Greenwald, the acting CEO of Apertura and vice president of business development at Deerfield, said the gene therapy platform provides Apertura with the potential to innovate next-generation therapies across delivery vectors, as well as in expression and payload capabilities. The technology platform provides Apertura with the opportunity to address technical challenges that have prevented gene therapy from reaching its full potential.

In an interview with BioSpace, Greenwald said Deerfield became familiar with Devermans work following a 2017 collaboration with the Broad Institute. Deerfield and Broad forged the research partnership to solve complex, early-stage therapeutic challenges related to serious unmet medical needs.

By harnessing the platform capabilities, Greenwald said its all about making better, safer drugs for serious diseases. The company is not disclosing potential targets at this time but he shared that Apertura's technology provides it with a broad opportunity to target rare diseases.

There are more than 7,000 rare diseases and most of those are caused by a genetic mutation of some kind. There are lots of opportunities out there, he said.

Greenwald was also quick to point out that a small company like Apertura cannot target every rare disease on its own. He said the company will be open to partnerships with biopharma companies large and small, as well as potential academic institutions.

Kristina Wang, director of corporate development and a board member at Apertura, noted that a significant number of the known rare diseases are out of the reach of existing gene therapy technologies. Aperturas next-generation technologies should create new target opportunities, she said.

Although no specific targets have yet to be announced, Greenwald said in the coming weeks Apertura will begin to make some announcements regarding preclinical data. He said that the company will announce some findings as it prepares to participate in upcoming scientific conferences.

The Apertura platforms are capable of simultaneously engineering AAV capsids to exhibit enhanced cellular tropism and the evasion of pre-existing immunity. In addition to the technology platforms that form the basis of its gene therapy program, Apertura also secured methods of identifying cell-type-specific genetic regulatory elements (GREs) from Harvard and also gained access to the Harvard-developed Paralleled Enhancer Single-Cell Assay (PESCA) platform.

The GRE platform is designed to focus on genetic regulatory elements and enhancers that drive cell type-specific expression, disease state-specific expression, and tunable expression levels, the company said. These capabilities are expected to enable Apertura to develop best-in-class gene therapies.

Our platform has the potential to unlock many new indications for gene therapy, Wang said in a statement. We aim to maximize our impact through dedicated internal programs and meaningful partnerships with other biopharma companies and academic groups. Committed to advancing the field of gene therapy, Apertura seeks to collaborate broadly to accelerate impact to patients.

The Series A was backed by Deerfield Management Company, which also committed additional operational support to the startup in order to bolster the companys ability to advance gene therapy discoveries.

See the original post:
Apertura Launches Leveraging Harvard, MIT Gene Therapy Platforms: Updated - BioSpace

Astellas Gene Therapies has terminated research and development of its gene therapy programs AT702, AT751, and AT753 for Duchenne muscular dystrophy (DMD).

The move was based on recent preclinical data, the company announced in a press release.

AT702, AT751, and AT753 are exon skipping medicines designed to treat people with DMD caused by certain genetic mutations in the DMD gene, which encodes the dystrophin protein, essential for muscle stability. Due to mutations in this gene, muscle cells cannot produce enough dystrophin, leading to progressive muscle weakness.

Exons are the specific segments in a gene that provide the instructions to make proteins.

The technology uses a modified, harmless adeno-associated virus (AAV) to deliver small molecules antisense oligonucleotides complementary to the gene of interest, allowing cells to skip over specific exons while generating proteins. The goal is to replace or restore dystrophin production in muscle cells.

Each of these three therapies target different DMD gene exons to treat distinct groups of patients. AT702 was designed to skip exon 2 and is meant for DMD patients who either have duplications in exon 2 or mutations in exons 1-5. AT751 is for those with mutations amenable to exon 51 skipping, and AT753 is for those with defects amenable to exon 53 skipping.

A mouse model with a DMD exon 2 duplication showed AT702 effectively promoted skipping of exon 2, resulting in a dose-dependent increase in dystrophin levels and improvements in muscle function. No clinically significant toxicity was seen in mice or nonhuman primates.

The data supported the launch of a Phase 1/2 clinical trial (NCT04240314) in three boys, ages 6 months to 13 years, with an exon 2 duplication in their DMD gene.

Conducted at the Nationwide Childrens Hospital (NCH) in Ohio, the one-time gene therapy was infused directly into their bloodstream at a dose of 31013 vector genomes per kilogram of body weight. Assessment of toxicity was set at two years of treatment.

Interim results after six months showed AT702 safely and effectively increased dystrophin levels in muscle biopsy samples and stabilized muscle function in two of the boys. The treatment was well tolerated, with temporary side effects including nausea, vomiting, and abdominal pain, but there were no serious side effects reported.

AT702 was originally developed by researchers at the NCH, then licensed in 2019 to Audentes Therapeutics, which was acquired by Astellas soon after.

Original post:
Three DMD Gene Therapy Programs Terminated by Astellas, Citing Data - Muscular Dystrophy News

LEIDEN, Netherlands & CAMBRIDGE, Mass., April 26, 2022 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the Company), a company dedicated to changing lives through the creation of transformative RNA therapies, today announced several presentations at the 7th Annual Retinal Cell and Gene Therapy Innovation Summit being held Friday, April 29, 2022 and the Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) being held May 1-4, 2022, both in Denver, CO, U.S.

Presentations at the Retinal Cell and Gene Therapy Innovation Summit

Presentation title: AON treatment for CEP290-LCA: Efficacy, safety, and durabilityPresenter: Dr. Artur Cideciyan, University of PennsylvaniaPresentation type: Pre-recorded talkSession: Session 4: Antisense Oligonucleotide TherapyDate: April 29, 2022 at 4:00pm MDT

Presentation title: Efficacy and safety of sepofarsen, an intravitreal RNA antisense oligonucleotide, for the treatment of CEP290-associated inherited retinal disease called Leber congenital amaurosis (LCA10): A randomized, double-masked, sham-controlled, phase III study (ILLUMINATE)Presenter: Dr. Bart Leroy, Ghent University HospitalPresentation type: Oral presentationSession: Session 4: Antisense Oligonucleotide TherapyDate: April 29, 2022 at 4:15pm MDT

Presentations at ARVO

Presentation title: Long-term safety and efficacy of sepofarsen in a Ph1b/2 INSIGHT extension trial in CEP290-associated Leber congenital amaurosis (LCA10)Presenter: Dr. Stephen R. Russell, Iowa UniversityPresentation type: Oral presentationDate: May 1, 2022 at 3:36pm MDT

Presentation title: QR-1011 corrects splicing in the Stargardt disease type 1-causing variant ABCA4 c.5461-10T>CPresenter: Melita Kaltak, ProQR TherapeuticsPresentation type: Oral presentationDate: May 4, 2022 at 10:34am MDT

Presentation title: Efficacy and safety of sepofarsen, an intravitreal RNA antisense oligonucleotide, for the treatment of CEP290-associated Leber congenital amaurosis (LCA10): a randomized, double-masked, sham-controlled, Phase 3 study (ILLUMINATE)Presenter: Dr. Bart Leroy, Ghent University HospitalPresentation type: Poster presentationDate: May 4, 2022 at 3:00pm MDT

CEP290-mediated Leber congenital amaurosis 10, or LCA10, is a difficult to treat ultra-rare inherited retinal disease for which there is currently no treatment, said Dr. Bart Leroy, Head of the Ophthalmology Department and Professor of Ophthalmology and Ophthalmic Genetics at Ghent University in Belgium and Attending Physician at The Children's Hospital of Philadelphia. After missing the primary endpoint in the Illuminate Phase 2/3 clinical trial, sepofarsen showed an encouraging efficacy signal across multiple endpoints in post-hoc analyses comparing sepofarsen treated and sham treated eyes to their corresponding untreated contralateral eyes. I look forward to continuing to work with ProQR on this investigational treatment.

About Leber congenital amaurosis 10 (LCA10)

Leber congenital amaurosis (LCA) is the most common cause of blindness due to genetic disease in children. It consists of a group of diseases of which LCA10 is the most frequent and one of the most severe forms. LCA10 is caused by mutations in the CEP290 gene, of which the c.2991+1655A>G (p.Cys998X) mutation has the highest prevalence. LCA10 leads to early loss of vision causing most people to lose their sight in the first few years of life. To date, there are no treatments approved that treat the underlying cause of the disease. Approximately 2,000 people in the Western world have LCA10 because of this mutation.

About sepofarsen

Sepofarsen (QR-110) is an investigational RNA therapy designed to restore vision in Leber congenital amaurosis 10 due to the c.2991+1655A>G mutation (p.Cys998X) in the CEP290 gene. The mutation leads to aberrant splicing of the mRNA and non-functional CEP290 protein. Sepofarsen is designed to enable normal splicing, resulting in restoration of normal (wild type) CEP290 mRNA and subsequent production of functional CEP290 protein. Sepofarsen is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and received fast-track designation and rare pediatric disease designation from the FDA as well as access to the PRIME scheme by the EMA.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer, which uses a cells own editing machinery called ADAR (adenosine deaminase acting on RNA) to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for genetic diseases. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.Learn more about ProQR at http://www.proqr.com.

FORWARD-LOOKING STATEMENTS

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding participation in these conferences as well as sepofarsen (QR-110) and the clinical development and the therapeutic potential thereof, and our planned interactions with regulatory authorities relating to our programs. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted by the COVID-19 pandemic; the likelihood of our clinical programs being executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for later data to alter initial and preliminary results of early-stage clinical trials, including as a result of differences in the trial designs and protocols across different trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the outcomes of our planned interactions with regulatory authorities; the ability to secure, maintain and realize the intended benefits of collaborations with partners; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; our ability to maintain and service our loan facility with Pontifax and Kreos; general business, operational, financial and accounting risks; and risks related to litigation and disputes with third parties. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.

ProQR Therapeutics N.V.

Investor Contact:Sarah KielyProQR Therapeutics N.V.T: +1 617 599 6228skiely@proqr.comorHans VitzthumLifeSci AdvisorsT: +1 617 430 7578hans@lifesciadvisors.com

Media Contact:Robert StanislaroFTI ConsultingT: +1 212 850 5657robert.stanislaro@fticonsulting.com

See the article here:
ProQR to Present at the Retinal Cell and Gene Therapy Innovation Summit and the Association for Research in Vision and Ophthalmology (ARVO) 2022 -...