In this study, researchers will observe and study the data from participants with hemophilia A who receive damoctocog alfa pegol as prescribed by their doctors. Participants will not receive any advice or changes to their healthcare during the study.
Hemophilia A is a genetic bleeding disorder. It is caused by the lack of a protein called clotting factor 8 (FVIII) that helps blood to clot properly. Lack of FVIII can result in excessive blood loss or bleeding inside the body after being injured or having surgery.
The study drug, damoctocog alfa pegol, can be used to prevent or treat bleeding episodes by replacing missing FVIII in the body of people with hemophilia A. It is already approved for people with hemophilia A who are at least 12 years old and have previously used other hemophilia A treatments.
Through this study, researchers want to learn more about its safety in a real-world setting.
The participants will receive damoctocog alfa pegol as prescribed by their doctors during routine practice according to the approved product information.
The main purpose of this study is to learn more about how safe damoctocog alfa pegol is in Korean participants with hemophilia A who previously used other hemophilia A treatments. To do this, researchers will collect information about any medical problems participants have during their treatment.
Data will be collected from December 2023 to March 2026 and cover a period of about 8 months for each participant. Data will come from participants' health records and information collected during their routine clinic visits.
In this study, only available data from routine care will be collected. No visits or tests are required as part of this study.

Start Date24 Jan 2024

Sponsor / Collaborator

Bayer AG

NCT05643560

/ RecruitingNot Applicable

Long-term Treatment Outcome of Jivi® Prophylaxis on Joint Health in Adult Patients With Hemophilia A

This is an observational study in which data from people with hemophilia A who decide on their own or by recommendation of their doctors to take Jivi are collected and studied. In observational studies, only observations are made without specified advice or interventions.
Hemophilia A is a genetic bleeding disorder that is caused by the lack of a protein in the blood called "clotting factor 8" (FVIII). FVIII is naturally found in the blood where it causes the blood to clump together to help prevent and stop bleeding. People with lower levels of FVIII or with FVIII that does not work properly may bleed for a long time from minor wounds, have painful bleeding into joints, or have internal bleeding.
The study treatment, Jivi (also called damoctocog alfa pegol), is already available for doctors to prescribe to people with hemophilia A to treat and prevent bleeding. It works by replacing the missing FVIII, or the FVIII that does not work properly.
People with hemophilia A need frequent injections of FVIII products into the vein. So called standard half-life (SHL) products need to be given 2 to 4 times a week for the prevention of bleeding. In recent years, new products like Jivi called extended half-life (EHL) products have available. These products last longer in the body so that they require to be given less often with injections up to every 7 days. Thus, these treatments may be easier and more comfortable to stick to in daily life. There is no general plan concerning the best amount of treatment and the frequency of injections for the prevention of bleeding, since the severity may be different and individual risk factors have to be considered. Doctors often decide on a treatment plan based on patient's disease and response.
Clinical studies have already shown that people with hemophilia A benefit from the treatment with Jivi. However, there are no data available coming from the real-world about how well Jivi works to support joint health, measured by ultrasound (US) examination and HEAD-US score.
In this study, researchers want to learn more about how well Jivi works if used for prolonged periods of treatment under real-world settings to prevent problems with joints in people with hemophilia A. How well it works means to find out if participants' joints status can be improved by treatment with Jivi.
To do this, researchers will collect data about participants' joints status by
* making images of participants' joints by using sound waves (ultrasound), and
* using HEAD-US score after 24 months of treatment with Jivi. The researchers will then compare these data to the participants' joints status before treatment start with Jivi.
Besides this data collection, no further tests or examinations are planned in this study.
Some participants in this study will already be receiving treatment with Jivi as part of their regular care no more than 12 months. And some participants will start to take Jivi in this study as prescribed by their doctors during routine practice according to the approved product information.
The researchers will collect data from each patient for a period of 26 months after initiation of the Jivi treatment.
There are no required visits or tests in this study

Start Date29 Dec 2022

Sponsor / Collaborator

Bayer AG

NCT05036278

/ CompletedPhase 4

A Multicenter, Prospective, Open-label, Clinical Study to Assess the Effect of Using a New Risk Score Approach to Select the Most Appropriate Prophylaxis Regimen for Reaching a Favorable Outcome, When Hemophilia A Patients Switch From Standard Half-life Products to Damoctocog Alfa Pegol (Jivi)

Researchers are looking for a better way to treat people who have hemophilia A. Hemophilia A is a genetic bleeding disorder that is caused by the lack of a protein in the blood called "clotting factor 8" (FVIII). FVIII is naturally found in the blood where it causes the blood to clump together to help prevent and stop bleeding. People with lower levels of FVIII or with FVIII that does not work properly may bleed for a long time from minor wounds, have painful bleeding into joints, or have internal bleeding.
The study treatment, Jivi (also called damoctocog alfa pegol), is already available for doctors to prescribe to people with hemophilia A to treat and prevent bleeding. It works by replacing the missing FVIII, or the FVIII that does not work properly.
People with hemophilia A need frequent injections of FVIII products into the vein. So called standard half-life (SHL) products need to be given 2 to 4 times a week for the prevention of bleeding. In recent years, new products like Jivi called extended half-life (EHL) products have become available. These products last longer in the body so that they require to be given less often with injections every 3-5 days. Thus, these treatments may be easier and more comfortable to stick to in daily life. There is no general plan concerning the best amount of treatment and the frequency of injections for the prevention of bleeding, since the severity may be different and individual risk factors have to be considered. Doctors often decide on a treatment plan based on their experience.
The main purpose of this study is to learn how well a new scoring approach works to select a treatment plan for the prevention of bleeding in people with hemophilia A who switch their treatment from SHL products to Jivi. Different types of information are used to calculate the risk score like bleeding history, certain biological factors, and physical activity of the participant.
All participants will receive Jivi for 6 months.
In the first four weeks, all participants will receive Jivi 2 times a week at a dose level of 40 IU per kilogram body weight (also known as 40 IU/kg/dose, recommended maximum dose is 6,000 IU). Then, based on their risk score, each participant will be assigned to one of three treatment plans:
* participants with a high risk remain on Jivi administration 2 times a week at 40 IU/kg/dose
* participants with a medium risk will switch to Jivi administration every 5 days at 50 IU/kg/dose
* participants with a low risk will switch to Jivi administration every 5 days at 50 IU/kg/dose and after 4 weeks to a less frequent administration (e.g., every 7 days) at 60 IU/kg/dose To check how well the new scoring approach works for choosing the right treatment plan, researchers will look at how many participants have a favourable outcome.
This means that the participant has either fewer bleeding events vs. the pre-study treatment and takes Jivi less often or as often as the previous SHL treatment but with fewer bleeding events, or that the participant has a comparable number of bleeding events but needs to take Jivi less often than the previous treatment. Each participant will be in the study for approximately 7.5 months. During this time, 4 visits to the study site and 3 phone calls are planned. During the study, the doctors and their study team will: • do physical examinations • take blood samples • ask the participants questions about how they are feeling and what adverse events they are having. In addition, participants or their guardians are required to write down the dates of Jivi treatments and bleeding events in an electronic diary and to fill in different questionnaires on their quality of life, health status, work/ school productivity, pain, and treatment satisfaction. In addition, participants are expected to keep appointments for visits and to adhere to the assigned treatment regimen.
An adverse event is any medical problem that a participant has during a study. Doctors keep track of all adverse events that happen in studies, even if they do not think the adverse events might be related to the study treatments.

Start Date28 Jul 2022

Sponsor / Collaborator

Bayer AG

100 Clinical Results associated with Damoctocog Alfa Pegol

100 Translational Medicine associated with Damoctocog Alfa Pegol

100 Patents (Medical) associated with Damoctocog Alfa Pegol

Literatures (Medical) associated with Damoctocog Alfa Pegol

01 Dec 2024·Blood Research

Real-world insights into the management of hemophilia A in Italy: treatment patterns and healthcare resource utilization

Article

Author: Perrone, Valentina ; Cappuccilli, Maria ; Leogrande, Melania ; Esposti, Luca Degli

AbstractPurposeThis real-world analysis described the Hemophilia A (HA) population in Italy, evaluating drug utilization and consumption of factor VIII (FVIII) products of patients under prophylaxis and on-demand therapy.MethodsFrom Jan-2017 to Jun-2022, male patients with HA were identified through prescriptions of FVIII products [extended half-life FVIII, standard half-life recombinant FVIII, and plasma-derived FVIII (EHL FVIII, SHL rFVIII, and pdFVIII, respectively)], or emicizumab or FVIII plus von Willebrand factor or HA-related hospitalization using administrative flows of Italian healthcare entities. Patients on treatment with FVIII products during 2021–2022 were stratified by treatment regimen (prophylaxis/on-demand). The mean annual consumption expressed in International Units (IU) of EHL FVIII and SHL FVIII in patients treated during 2021–2022 having at least 12-month follow-up were assessed.ResultsAmong included HA patients, 145 (39.5%) received EHL FVIII and 222 (60.5%) SHL FVIII. Of 165 patients on prophylaxis, 105 (64%) received an EHL FVIII and 60 (36%) an SHL FVIII. The mean annual consumption of FVIII was 336,700 IU (median 319,000 IU) for EHL FVIII and 440,267 IU (median 360,500 IU) for SHL FVIII. Specifically, for patients on EHL FVIII, the most common drugs were efmoroctocog alfa (N = 51) and damoctocog alfa pegol (N = 50), followed by turoctocog alfa pegol (N = 25) and rurioctocog alfa pegol (N = 19). Of 702 HA patients initially treated with FVIII products, 74 (10.5%) switched to emicizumab during follow-up.ConclusionThese findings revealed an extensive use of EHL FVIII products, suggesting growing efforts from clinicians to optimize prophylactic strategies and achieve better bleeding protection.

01 Sep 2024·European Journal of Haematology

Plain language summary on “Interim analyses of the multinational real‐world prospective cohort HEM‐POWR study evaluating the effectiveness and safety of damoctocog alfa pegol in patients with hemophilia A”

Article

Author: Meijer, Karina ; Castaman, Giancarlo ; Reding, Mark T. ; Schmidt, Kathrin ; Matsushita, Tadashi ; Janbain, Maissaa ; Alvarez-Roman, Maria Teresa ; Oldenburg, Johannes

To assess effectiveness and safety of damoctocog alfa pegol in interim analyses of the ongoing real-world hemophilia A HEM-POWR study.HEM-POWR (NCT03932201) is a multinational Phase 4 prospective observational study.The primary objective was annualized bleeding rate (ABR) in previously treated patients (PTPs) with hemophilia A.Secondary objectives included adverse events and number of affected joints.At data cut-off (August 17, 2022), the safety anal. set included 268 patients and the full anal. set (FAS) included 161 patients.The most common dosing regimen during observation period was prophylaxis (FAS = 158/161, 98.1%) every 3-4 days (twice weekly; FAS = 78/158, 49.4%) and a median (min, max) infusion dose of 37.5 (10, 72) IU/kg.PTPs receiving prophylactic damoctocog alfa pegol have fewer infusions compared with prior treatment.Median total ABR (Q1, Q3) was 0.0 (0.0, 1.8) and mean total ABR (SD) was 2.4 (8.2).The proportion of patients with no affected joints increased between initial visit and follow-up.No FVIII inhibitors, treatment-related adverse events, or deaths were reported.Damoctocog alfa pegol shows effectiveness and acceptable safety, as well as consistent utilization, in real-world PTPs with hemophilia A, including in patients with non-severe hemophilia and those with a history of inhibitors.

01 Sep 2024·European Journal of Haematology

A post hoc analysis of PROTECT VIII Kids assessing long‐term efficacy and safety of damoctocog alfa pegol in adolescents with severe haemophilia A

Article

Author: Biss, Tina ; Kenet, Gili ; Steele, MacGregor ; Mancuso, Maria Elisa ; Ahuja, Sanjay ; Maas Enriquez, Monika

The safety and efficacy of the extended half-life factor VIII (FVIII) product damoctocog alfa pegol (BAY 94-9027, Jivi) has been demonstrated in the PROTECT VIII Kids study (NCT01775618), where male previously-treated patients (PTPs) aged <12 years old with severe haemophilia A and ≥ 50 exposure days (EDs) were treated prophylactically.The PROTECT VIII Kids extension study assessed the long-term safety and efficacy of damoctocog alfa pegol in the same population.To evaluate the long-term impact of damoctocog alfa pegol in a post hoc subgroup anal. of adolescent patients in the PROTECT VIII Kids study and its extension from 12th birthday onwards.The current anal. included PTPs aged ≥12 years old, who remained in the extension for ≥6 mo following their 12th birthday.The observation period was defined as the time from 12th birthday to the end of the extension period; all data from this birthday were included whether in the main study or extension phase.The main efficacy variable was annualised bleeding rate (ABR) and the main safety variable was the frequency of inhibitor development.This subgroup anal. comprised 25 patients.Median observation time after 12th birthday was 3.2 years.Median total/joint/spontaneous ABRs in the observation period were 1.7/0.7/0.3, resp.Safety findings were consistent with those reported for the overall study population; no confirmed FVIII inhibitors or anti-drug antibodies were reported.Damoctocog alfa pegol is efficacious with a favorable safety profile in adolescents with haemophilia A, supporting its long-term use in children and adolescents.

News (Medical) associated with Damoctocog Alfa Pegol

29 Feb 2024

·www.biospace.com

Baisong Mei’s Immigrant Journey Through American Biopharma

Pictured: Baisong Mei/Nicole Bean for BioSpace Baisong Mei is no stranger to leadership roles. At just 15 years old, he was already in medical school leading a group of students 10 years his senior. (In China, medical students skip undergraduate degrees and instead go straight to a doctoral program.) That was just one of many instances of leadership peppered throughout the career of Editas’s current chief medical officer. Mei was born in a small town in the southern part of Anhui province in China. He skipped grades a couple of times, which allowed him to start college at Bengbu Medical College (now Bengbu Medical University) at just 15 years old, the first time in his life he went to a bigger city, Bengbu. Because he had done well in high school, scored reasonably well on the college entrance examination, held leadership positions and participated in competitions, he was made the head of a group in the class. This entailed leading older students in group activities, an experience he called “challenging.” But that didn’t discourage him. After graduating from Bengbu Medical College at the age of 20, he went on to earn a master’s degree at Wuhan University School of Medicine, where he then worked as a faculty member for five years. At 28, he left China to move to Ohio with only two suitcases. There, Mei earned a Ph.D. in molecular biology and biochemistry from the University of Toledo. He then completed fellowships at the University of California, San Francisco and UC Berkeley; held leadership positions at Bayer, Biogen and Sanofi; and co-invented an approved drug, Jivi, an injectable medicine from Bayer for people with hemophilia A. Mei’s laundry list of accomplishments goes on. But he said that one of the most rewarding successes of his career is seeing patients in clinical trials that he runs sign on for an extension. He explained that this usually means patients feel like the drug is working for them without too many complications, which is a huge success in the R&D world. It’s happened many times throughout his career, he said, the first time with a hemophilia B drug at Biogen that was later approved by the FDA and marketed as Alprolix. Learning to Adapt Mei’s position at early-stage biotech Editas is more entrepreneurial than his earlier career, allowing him to remain close to the science and to clinical trials. But after coming from academia and Big Pharma, learning how to operate in a genome editing biotech with a few hundred employees involves a learning curve, he said. Fortunately for him, his formula for success is all about continuously learning and adapting. The most valuable thing he’s learned since starting at Editas in 2022 is how to communicate in a way that inspires confidence in the company, he said, so that stakeholders know that it has a solid strategy and can deliver helpful products. He’s been part of the executive leadership team working to transform Editas from a technology platform company to a commercial therapeutics company and leader in in vivo programmable gene editing. The team made changes to sharpen the company’s strategic focus and emphasize execution of clinical programs. “When you become a CMO in a private company, in addition to driving all these clinical trials, you also [have to think about] how the company can grow and what is the strategy of the company so that [it] can help more patients and help the stakeholders of the company,” he said. With these additional responsibilities, “I feel that I’m able to make more impact, not only to the company, [but] to the patients.” Mei categorizes the challenges he’s encountered in his career into two types: cultural and career-related. Most of his cultural challenges happened when he first came to the U.S. and had to adapt to food, language, TV, interpersonal interactions and many other aspects of daily life that were completely different from China. And his cultural environment has varied greatly even throughout his time in the U.S., he said, since he’s now lived in the Midwest and on both coasts. “The education system in China really . . . set a foundation for me to be able to adapt here in United States,” he said, by emphasizing math, physics and chemistry, and putting a lot of value on hard work and discipline. For example, in high school, he often studied past midnight and in medical school he had over 30 hours of classes each week. “This allowed me to really focus on learning and be humble, and that really helped me a lot.” When he first came to the U.S. for his Ph.D., it was uncertain whether he would be able to remain in the country to work after his student visa period. So he viewed it as an experiment, completing his Ph.D. without knowing how the rest of his career would turn out. If he couldn’t stay in the U.S., his backup plan was to bring his learning back to China. “And now, of course, after more than 30 years, I feel I’m American,” he said. “I feel the United States has given immigrants like myself so many opportunities. I feel so grateful for that . . . this is actually an amazing place [that] can provide people opportunity. And I understand why many people want to come.” Mollie Barnes is a freelance science writer based in Los Angeles. Reach her at mollie@100yearsco.com. Follow her on Threads and Instagram @shejustlikedtogo and see more of her work at molliebarnes.contently.com.

Drug ApprovalExecutive Change

31 Oct 2023

·www.prnewswire.com

Octapharma AG: Indirect comparison data indicate that personalised prophylaxis with Nuwiq® may lead to fewer patients with haemophilia A having bleeds than with other recombinant factor VIII products

The well-established matching-adjusted indirect comparison (MAIC) methodology was used to compare the efficacy of personalised prophylaxis with Nuwiq® against other recombinant FVIII products Personalised prophylaxis with Nuwiq® can lead to higher rates of patients with zero bleeds compared with other recombinant FVIII products, albeit with higher weekly doses The data were published recently in the European Journal of Haematology LACHEN, Switzerland, Oct. 31, 2023 /PRNewswire/ -- Data from a matching-adjusted indirect comparison (MAIC) of Nuwiq® (simoctocog alfa) versus extended half-life recombinant factor VIII (rFVIII) concentrates in haemophilia A patients undergoing personalised prophylaxis were published in the European Journal of Haematology1. Preliminary data from the MAIC were presented by Dr Craig Kessler, Lombardi Comprehensive Cancer Center and Georgetown University Medical Center, at the 65th ASH Annual Meeting & Exposition in December 20222. Continue Reading Octapharma AG: Indirect comparison data indicate that personalised prophylaxis with Nuwiq® may lead to fewer patients with haemophilia A having bleeds than with other recombinant factor VIII products The analysis used MAIC – a well-established statistical approach – to compare the efficacy of pharmacokinetic (PK)-guided, personalised prophylaxis with Nuwiq® (simoctocog alfa) versus Eloctate® (efmoroctocog alfa), Jivi® (damoctocog alfa pegol), and Adynovate® (rurioctocog alfa pegol). The analysis used data from prospective clinical trials of personalised prophylaxis in adults with severe haemophilia A. Dr Kessler commented, "This MAIC exercise provides important comparative data on the efficacy and consumption of various recombinant FVIII products in adults with haemophilia A on personalised prophylaxis. The results can be considered by clinicians and patients when treatment decisions are being made." MAICs are a way of comparing the efficacy of different medical interventions or products in the absence of head-to-head randomised trials3. By matching patient populations based on clinically relevant baseline characteristics, it reduces inherent differences between the studies and can provide comparative evidence of relative treatment effects. Individual patient data from patients treated with Nuwiq® in the NuPreviq study were compared with aggregated data from 117 patients treated with Eloctate® in the personalised prophylaxis arm of the A-LONG study, 110 patients treated with Jivi® in the PROTECT VIII study, and patients treated with Adynovate® in the PROPEL study (which had two arms targeting different FVIII trough levels: 1–3% (57 patients) and 8–12% (58 patients)). A significantly higher percentage of patients with zero bleeds was reported with Nuwiq® compared with Eloctate® (75% vs 45%), Jivi® (77% vs 38%) or Adynovate® (target trough level 1–3%; 78% vs 42%). The efficacy of Nuwiq® was similar to that of Adynovate® in the 8–12% arm (77% vs 62%). The mean annualised bleeding rate was significantly lower with Nuwiq® than with Jivi® (1.5 vs 4.9). Consistent with the approved dosing, the mean FVIII weekly dose was significantly higher for Nuwiq® than Eloctate®, Jivi® or Adynovate® 1–3%, but lower than for Adynovate® 8–12%. Further details of the study can be found in the European Journal of Haematology publication. In addition, a short video summarising the results is available in the online version of the article. About Octapharma Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. Octapharma employs more than 11,000 people worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Haematology and Critical Care. Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 190 plasma donation centres across Europe and the US. Octapharma has 40 years of experience in patient care. About Nuwiq® Nuwiq® (simoctocog alfa) is a 4th generation recombinant factor VIII (rFVIII) protein, produced in a human cell line without chemical modification or fusion with any other protein4. It is cultured without additives of human or animal origin, is devoid of antigenic non-human protein epitopes and has a high affinity for von Willebrand factor4. Nuwiq® treatment has been assessed in nine4-6 completed clinical trials which included 201 previously treated patients (190 individuals)4 and 108 previously untreated patients5 with severe haemophilia A. Nuwiq® is available in 250 IU, 500 IU, 1,000 IU, 1,500 IU, 2,000 IU, 2,500 IU, 3,000 IU and 4,000 IU presentations7. Nuwiq® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups7. References Kessler CM et al. Eur J Haematol 2023; 111:757–67. Kessler CM and Tiede A. Blood 2022; 140(Suppl. 1): 2755–6. Signorovitch JE et al. Value Health 2012; 15:940–7. Lissitchkov T et al. Ther Adv Hematol 2019; 10:2040620719858471. Liesner RJ et al. Thromb Haemost 2021; 121:1400–8. Octapharma AG, data on file. Nuwiq® Summary of Product Characteristics. Logo - Video - SOURCE Octapharma AG

Clinical ResultDrug ApprovalClinical Study

10 Apr 2023

·www.biospace.com

HuidaGene Appoints New Members to the Scientific Advisory Board

SHANGHAI and CLINTON, N.J., Apr. 10, 2023 /PRNewswire/ -- HuidaGene Therapeutics (辉大基因; HuidaGene), a global clinical-stage biotechnology company focused on developing genomic medicine, today announced the selection of Drs. Haiyan Jiang and Alvin Luk as the new members of the Scientific Advisory Board (SAB). The SAB, which includes internationally renowned experts in gene and cell therapies, from discovery to translational research, and clinical development across different therapeutic areas, will provide strategic guidance for the global development of HuidaGene's expansive pipeline products. HuidaGene Appoints New Members to the Scientific Advisory Board "To meaningfully alter the course of genetic diseases, we must advance better-validated therapeutic candidates," said Dr. Haiyan Jiang. "My longstanding interest and commitment in gene and cell therapies have led me to HuidaGene's technology platforms that offer a potential cure to diseases with high unmet medical needs, and I am eager to work with the HuidaGene team to move their pipeline forward." Dr. Alvin Luk stated, "In looking to the future of the field, I have been impressed with HuidaGene's innovative genome-editing platforms. Dr. Hui Yang has been a long-time leader in CRISPR-based DNA/RNA editing, and I expect that our mutual interests and complementary areas of expertise within gene and cell therapy will be a powerful engine for advancing, and even accelerating, the progess at HuidaGene." "We are honored to have an accomplished and diverse group of scientific and clinical experts join us as members of our SAB, who represent extensive experience and knowledge in gene and cell therapies, global drug development, and data-driven clinical trial design," said Hui Yang, PhD, Co-Founder, Chief Scientific Advisor, and Chairman of the SAB at HuidaGene. "Haiyan and Alvin are industry veterans who have achieved a number of 'firsts' in gene therapy clinical programs and who will bring deep and relevant development expertise to HuidaGene. With our scientific advisors' leadership, HuidaGene is poised to translate decades of scientific discovery in programmable genome medicines to people living with devastating diseases worldwide," said Xuan Yao, PhD, Co-Founder and Chief Executive Officer of HuidaGene. About HuidaGene - 辉大基因 HuidaGene Therapeutics (辉大基因) is a global clinical-stage biotechnology company focusing on discovering, engineering, and developing CRISPR-based genetic medicine to rewrite the future of genomic medicine. Based in Shanghai and New Jersey, HuidaGene is committed to addressing patients' needs globally with various preclinical therapeutic programs covering ophthalmology, otology, myology, and neurology. We are currently advancing clinical programs in RPE65 mutation-associated inherited retinal diseases and our preclinical pipeline, including programs in neovascular age-related macular degeneration, retinitis pigmentosa, hereditary hearing loss, Duchenne muscular dystrophy, and MECP2 duplication syndrome. Company's CRISPR-based therapeutics offer the potential to cure patients with life-threatening conditions by repairing the cause of their disease. HuidaGene is committed to transforming the future of genome-editing medicine. For more information, please visit or follow us on LinkedIn at Details of the additional SAB members' biographies: Haiyan Jiang, Ph.D. has over 20 years of research and development expertise in biologics, AAV-mediated gene therapy, and in-vivo and ex-vivo CRISPR/Cas9- mediated genome editing products from research to BLA. She has held leadership roles with progressive responsibilities at various companies including Editas Medicine, Biogen, Bayer HealthCare, and Avigen. She led research, preclinical development or clinical pharmacology in the development of ALTUVIIIO, ELOCTATE, ALPROLIX, and JIVI, as well as a number of programs in Phase 1/2a studies. She has over 40 original publications in highly-regarded peer-reviewed journals, including New England Journal of Medicine, Nature Medicine, and Blood; as well as 21 issued US patents. Dr. Jiang received her B.S. in Biochemistry from Fudan University in Shanghai, China, Ph.D. in Oncology from University of Wisconsin-Madison, and postdoctoral training at the University of California-San Francisco. Alvin Luk, Ph.D., M.B.A., C.C.R.A. brings over 30 years' experience in global drug development, from the bench to the beside through successful commercial launch in small, large, public, and private biotech/biopharma firms, including Shanghai Henlius, Spark Therapeutics (acquired by Roche), Biogen, Bayer HealthCare, Avigen, and Tularik (acquired by Amgen). He is a transformational leader with reputation for navigating through political landscape which leads to a proven track record of more than 250 cumulative total of IND, BLA, and MAA submissions in Americas, Europe, Australia, Japan, and China. Spark Therapeutics' LUXTURNA was approved by US FDA as the first gene therapy in 2017 when he severed as the Head of Clinical Research and Operations. Today, he has participated in the development of 21 approved products. He is the co-author of more than 100 book chapters and scientific papers in highly-regarded peer-reviewed journals, including New England Journal of Medicine, Nature Medicine, Nature, Cell, and Science. Dr. Luk holds an MBA from Harvard Business School and received his Ph.D. in Neuroscience as well as being certified in clinical research from the University of California-San Francisco.

Drug ApprovalExecutive ChangeGene TherapyAcquisition

100 Deals associated with Damoctocog Alfa Pegol

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KEGG	Wiki	ATC	Drug Bank
D10759	Damoctocog Alfa Pegol	B02BD02	DB14700

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hemophilia A	US	Bayer Healthcare LLC	29 Aug 2018

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hemorrhage	Phase 3	NO	Bayer AG	29 May 2013
Hemorrhage	Phase 3	IT	Bayer AG	29 May 2013
Hemorrhage	Discovery	LT	Bayer AG	29 May 2013
Hemorrhage	Discovery	NZ	Bayer AG	29 May 2013
Hemorrhage	Discovery	NL	Bayer AG	29 May 2013
Hemorrhage	Discovery	IL	Bayer AG	29 May 2013
Hemorrhage	Discovery	PL	Bayer AG	29 May 2013
Hemorrhage	Discovery	BG	Bayer AG	29 May 2013
Hemorrhage	Discovery	GR	Bayer AG	29 May 2013
Hemophilia A	Discovery	KR	Bayer AG	23 Apr 2012

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04085458 (CTgov)	Phase 4	Hemophilia A	32	BAY94-9027	zahzcmazil(hwqhgqnakc) = dwvuuraqpa lcfvpalbql (pbrijrvihh, xgbolwuiqs - woxrtydeyy) View more	-	28 Jun 2023
ISTH2023	Not Applicable	-	10	Damoctocog alfa pegol	(cconwctvdq) = mtzasxejxg nxxpoydxdv (ycujoapbzc ) View more	-	24 Jun 2023
ISTH2023	Not Applicable	Hemophilia A	-	BAY 94-9027	kxpzgdylbv(pmzchtyyfr) = ekbytbnfsg jssqytagtg (fotlsfwxtg )	Positive	24 Jun 2023
				BAY 81-8973	kxpzgdylbv(pmzchtyyfr) = kourksieuz jssqytagtg (fotlsfwxtg )
HEM-POWR Study (ISTH2022)	Not Applicable	Hemophilia A	162	Damoctocog alfa pegol	lsqwesduow(ieqsjuvmoi) = wggngkvbfd rduyaztzkz (vaazdxuhaz )	Positive	09 Jul 2022
ISTH2021	Not Applicable	Hemophilia A	134	BAY 94-9027	jfgczbjugw(watelnihjk) = hdebccnfgq jgoifwtbxv (scdjpbgorp )	Positive	17 Jul 2021
NCT04015492 (Pubmed \| Ann Hematol)	Phase 1	Hemophilia A	18	damoctocog alfa pegol	lrzunhyriy(jlyusuboev) = vlvhivwyjw espzfckyua (etbwqlhyok )	-	01 Nov 2020
				rurioctocog alfa pegol	lrzunhyriy(jlyusuboev) = lruiamlfsi espzfckyua (etbwqlhyok )
NCT01580293 (PROTECT VIII, ISTH2020)	Phase 2/3	Hemophilia A	121	BAY 94-9027	dqtkopkmea(jnmtdnthgn) = dnwzabeypk wuueyzdkhs (cfoncvkpbv ) View more	-	12 Jul 2020
NCT01580293 (PROTECT VIII, ISTH2020)	Phase 2/3	Hemophilia A	114	BAY 94-9027 prophylaxis every 5 days	pzuhxecmmw(gdikonestd) = 5 (13.9%) patients experienced non-serious treatment-emergent drug-related adverse events izmfuexevx (ehhcbtjrkd )	Positive	12 Jul 2020
				BAY 94-9027 prophylaxis every 7 days
NCT01580293 (PROTECT-VIII, Pubmed \| J Thromb Haemost) Manual	Phase 2/3	Hemophilia A	132	BAY 94-9027 (every-5-days (45-60 IU kg-1 ))	(pjxifuzfmt) = hqasxpcpcf ghaqavmlib (uqmmeteaph )	Positive	01 Mar 2017
				BAY 94-9027 (every-7-days (60 IU kg-1 ))	(pjxifuzfmt) = uohgpcdafz ghaqavmlib (uqmmeteaph )
NCT01184820 (Pubmed \| J Thromb Haemost)	Phase 1	Hemophilia A	14	BAY 94-9027	odrwfbcxjg(usizkfykyp) = kvjcrxeder ospfftnvxb (voefxrxeab )	-	01 Apr 2014
				sucrose-formulated rFVIII (rFVIII-FS)	odrwfbcxjg(usizkfykyp) = nlhfhydovs ospfftnvxb (voefxrxeab )

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