Category Archives: Genetic Therapy

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.

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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.

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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.

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

Pune, India, April 20, 2022 (GLOBE NEWSWIRE) -- The global gene therapy market size is set to gain momentum from the rising incidence of different types of cancer. The field of this therapy is undergoing several technological advancements that would help in treating cancer in those patients who are at high risks of getting affected by this disease through genetic mutations. The report further mentions that the market size was USD 3.61 billion in 2019 and is projected to reach USD 35.67 billion by 2027, exhibiting a CAGR of 33.6% during the forecast period.

Fortune Business Insights provided this information in a new report, titled, Gene Therapy Market Size, Share & COVID-19 Impact Analysis, By Application (Oncology, Neurology, and Others), By Vector Type (Viral and Non-viral), By Distribution Channel (Hospitals, Clinics, and Others), and Regional Forecast, 2020-2027.

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Industry Developments:

Report Scope & Segmentation

Drivers & Restraints-

Increasing Innovations & Research Activities to Boost Growth

The U.S Food and Drug Administration (FDA) stated that it is expecting to receive more than 200 applications of this therapy by the end of 2020. This showcases that the rising number of research studies and innovations in this field would affect the gene therapy market growth positively in the near future. In North America, almost 208 companies are currently operating in this market. In addition to this, the Alliance for Regenerative Medicine declared that as of 2018, approximately 259 potential drug candidates are under Phase I clinical trials across the globe.

For more information in the analysis of this report, visit: https://www.fortunebusinessinsights.com/industry-reports/gene-therapy-market-100243

However, the outbreak of the COVID-19 pandemic is presently impacting the field of research. According to the director of the Office of Tissues and Advanced Therapy (FDA) named Wilson Brayan, nowadays the officials are prioritizing only those drugs that are associated with coronavirus. Hence, there hasnt been a surge in the application of potential drugs for gene therapy. This factor may hamper the gene therapy market growth in the forthcoming years.

Highlights of This Report:

Segment-

Neurology Segment to Earn High Share Fueled by High Cost of Drugs

Based on product type, the market is divided into neurology, oncology, and others. Out of these, the neurology segment earned 78.2% in terms of gene therapy market share in 2019. This growth is attributable to the increasing usage of this therapy for treating patients living with spinal muscular atrophy.

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The U.S. to Dominate Owing to Presence of Favorable Policies

In 2019, the U.S. generated USD 2.16 billion in terms of revenue. The country is expected to dominate throughout the coming years stoked by the increasing usage of advanced gene therapies for the treatment of rare conditions. Besides, the presence of favorable reimbursement policies and guidelines would also help in propelling the market growth here. As this type of treatment is not legal in several developing nations, industry giants are emphasizing on the U.S. for launching their products.

Europe, on the other hand, is anticipated to grow significantly backed by the adoption of unique treatment options. Asia Pacific is set to hold a comparatively lower share on account of the decreasing usage of gene therapy because of its expensive nature.

Fortune Business Insights lists out the names of all the gene therapy providers present in the global market. They are as follows:

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With 33.6% CAGR, Gene Therapy Market Size worth USD 35.67 Billion in 2027 - GlobeNewswire

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

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

The trends in the hemophilia treatment market are gaining momentum with increasing incidences of hemophilia A and B and staggering need for their treatments, including gene, anti-tissue factor pathway inhibitor, and factor replacement therapies.

With novel developments in hemophilia treatment progressing at an unprecedented pace, the industry is touted to seize an overall remuneration of USD 16 billion by 2028 end- Global Market Insights Inc.

The trends in the hemophilia treatment market are gaining momentum with increasing incidences of hemophilia A and B and staggering need for their treatments (Figure). As per reliable estimates, the global prevalence of hemophilia A is announced to be approximately 1 case per 5000 males, with nearly 1 third of affected individuals not having a family history of the blood disorder. The rare disease frequency also varies with the reporting country, with a range of 5.4 to 14.5 cases per 100,000 males.

Hemophilia is a rare condition for which the approved treatment options have remained practically unchanged for quite a while now. Recently, however, the hemophilia treatment market is observing an explosion of innovation in the treatment options that are either under development or have already been approved, including some medications and injections.

In 2018, the FDA had approved emicizumab-kxwh which was intended to reduce or avert the frequency of bleeding episodes in people with hemophilia A. Some other options might include desmopressin, a manufactured hormone that stimulates the release of stored factor 8, and antifibrinolytic medications, that prevent clots from breaking down.

While oral medication and injections are recognized to treat the condition, its prolonged impact on human health or side effects are not known in detail. In this case, another welcome leap in the business space is the advancement in therapy options.

Given below are some of the latest advancements in the hemophilia treatment that are expected to push different economies toward healthy living and reduce the prevalence of the rare disease:

Gene Therapy

Since hemophilia is coined as a genetic disorder, gene therapy for its treatment could emerge as a possible breakthrough in the health care fraternity. The treatment offers a potential cure for hemophilia patients by establishing constant endogenic expression of factor 8 or 9 following transmission of functional gene to swap the hemophilic patients own flawed gene.

While only about 25% to 30% of the worlds hemophilia population has access to factor replacement owing to its costly alternative, complications, and burden, the ability of gene therapy solution for hemophilia delivers hope for more global admittance to the treatment.

Having said that, results from one of the recent trials of the approach have been known to restore patients anticoagulant factor activity levels to near normal or normal levels and dropped down their annualized bleeding rates by almost 90%.

Anti-TFPI Therapy

Anti-tissue factor pathway inhibitor, or anti-TFPI, therapy is an advanced treatment that seeks to reduce bleeding by decreasing on the system that averts the blood from clotting too much. This therapy restores hemostatic balance by blocking one of the anticoagulants and preventing it from functioning normally.

Considering its success trail, different biopharmaceutical firms are now researching and analyzing ways to incorporate anti-TFPI therapy into their portfolio of hemophilia treatment. One of these companies is Pfizer.

The leading pharmaceutical firm recently announced dosing its first participant in the Phase 3 BASIS study of marstacimab, an anti-TFPI being evaluated for the treatment of people suffering from severe hemophilia A or B. The completed phase 2 study results represented that the treatment with marstacimab demonstrate more than 75% reductions in annual bleeding rates for almost all the participants in the study population.

Factor Replacement Therapy

The factor replacement therapy is deemed as one of the efficient therapies to treat hemophilia. In this, the clotting factors are injected into veins to stop severe blood loss and problems from bleeding such as joints, organs, and muscles. These therapies have been widely used before surgery or operation to avoid excessive blood loss.

According to a recent study by Global Market Insights Inc., it was revealed that the factor replacement therapy surpassed USD 9,412 million in 2021, in terms of valuation, across the overall industry. It is further anticipated to hold a prominent position worldwide owing to the ongoing R&Ds and advancements in the space.

To illustrate, the US FDA announced approval of Factor VII treatment for hemophilia A and B with inhibitors.

In a nutshell, increasing prevalence of hemophilia and surging need for associated treatment therapies globally would help hemophilia treatment market growth curve to ascend significantly through 2028.

Reference

Hemophilia treatment market size by disease (hemophilia a {severe, moderate, mild}, hemophilia b {severe, moderate, mild}), by product (recombinant factor concentrates {factor VIII, factor IX}, plasma-derived factor concentrates {factor VIII, factor IX}, extended half-life products {factor VIII, factor IX}), by patient (pediatric {0 to 4, 5 to 13, 14 to 18}, adult {19 to 44, 45+}), by treatment (prophylaxis, on demand), by therapy (factor replacement therapy, non-factor replacement therapy), by drug class (vasopressin, coagulation factors), by route of administration (injectable, nasal spray), by end-use (hospitals, clinics, hemophilia treatment centers), industry analysis report, regional outlook, application potential, COVID-19 impact analysis, price trends, competitive market share & forecast, 2022 2028. Global Markets Insights website. gminsights.com/industry-analysis/hemophilia-treatment-market. Publication February 2022. Accessed April 2022.

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Contributor: Top 3 Future Therapies to Drive Hemophilia Treatment Market Growth - AJMC.com Managed Markets Network