Latest news with #hemophiliaA
Yahoo
28-06-2025
- Business
- Yahoo
BioMarin's ROCTAVIAN Shows Sustained Efficacy, Safety Over 5 Years for Severe Hemophilia A
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the most undervalued US stocks according to analysts. On June 24, BioMarin Pharmaceutical Inc. presented new 5-year data from its Phase 3 GENEr8-1 trial at the 33rd Congress of the International Society on Thrombosis and Haemostasis/ISTH in Washington, D.C., which took place from June 21 to 25. The data reinforced the long-term efficacy and safety of ROCTAVIAN (valoctocogene roxaparvovec-rvox) as a gene therapy for severe hemophilia A. The GENEr8-1 trial is the longest and largest hemophilia gene therapy study to date, and demonstrated sustained Factor VIII (FVIII) expression and durable bleed control 5 years after a single treatment with ROCTAVIAN. A pharmaceutical plant manufacturing a proprietary synthetic oral form of a C-type natriuretic peptide. FVIII activity remained consistent with previously reported results. Importantly, no new safety signals were observed over the 5-year study period. Across all 134 participants who received ROCTAVIAN in the study, there were no cases of FVIII inhibitors or thromboembolic events, and no treatment-related malignancies were observed. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is a biotechnology company that develops and commercializes therapies for life-threatening rare diseases and medical conditions internationally. While we acknowledge the potential of BMRN as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the . READ NEXT: and . Disclosure: None. This article is originally published at Insider Monkey. Sign in to access your portfolio
Yahoo
22-06-2025
- Health
- Yahoo
Mim8 prophylaxis treatment shown to be well-tolerated when switching from emicizumab in people with hemophilia A in new phase 3 data presented at the ISTH 2025 Congress
New FRONTIER5 results show a direct switch to investigational Mim8 (denecimig) prophylaxis treatment from emicizumab, without the need for a washout period, was well-tolerated in adults and adolescents with hemophilia A, with or without inhibitors1 FRONTIER5 Patient-Reported Outcomes assessment found the Mim8 pen-injector easy to use with strong user preference over their emicizumab injection system2 PLAINSBORO, N.J., June 22, 2025 /PRNewswire/ -- Novo Nordisk today presented results from the phase 3b FRONTIER5 trial showing that a direct switch to investigational Mim8 (denecimig) prophylaxis from emicizumab treatment, without a washout period or Mim8 loading dose, was well tolerated in adults and adolescents living with hemophilia A, with or without inhibitors.1 Additionally, a FRONTIER5 Patient-Reported Outcomes (PROs) assessment found the Mim8 pen-injector easy to use with an overall strong user preference for the pen-injector, in comparison to previous injection systems.2 The results were presented at the International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington, D.C. "Continuous prophylactic coverage is critical to avoiding breakthrough bleeds in people living with hemophilia; with new non-factor therapeutic options, many people could have hesitations about switching treatment options. These data demonstrate that switching to Mim8 from emicizumab can be done without requiring a washout period," said Allison P. Wheeler, MD, Washington Center for Bleeding Disorders, Seattle, WA. "This is critical in ensuring that individuals maintain continuous protection against bleeding events as we seek to help address the ongoing needs of people living with this complex disease." In the open-label phase 3 FRONTIER5 safety study, 61 adults and adolescents aged 12 years and older with hemophilia A were enrolled.3 No thromboembolic events, hypersensitivity reactions, or treatment-emergent adverse events (TEAEs) leading to discontinuation were observed, and there was no evidence of neutralizing anti-Mim8 antibodies.1 Additional safety information from FRONTIER5 demonstrate that between Week 0 and Week 26 of treatment, there were 107 TEAEs observed in 43 patients (70.5%), most of which were mild to moderate (88.6%). There were 24 TEAEs that were possibly/probably related to Mim8 reported in 18 patients (29.5%). No thromboembolic events, hypersensitivity reactions, or TEAEs leading to discontinuation were observed.1 The PROs data from FRONTIER5 indicated overall (97%; n=57/59) patients preferred the Mim8 pen injection, with 97% of those patients reporting a "very strong" or "fairly strong" preference in comparison to their previous emicizumab injection system. Of the participants who completed the Hemophilia Device Handling and Preference Assessment (HDHPA) questionnaire at week 26, 98% (n=58/59) found the Mim8 pen-injector "very easy" or "easy" to use, and 95% (n=56/59) found it "much easier" or "easier" compared with their previous administration method. All participants (100%) were "extremely confident" or "very confident" in using the pen-injector correctly.2 "The FRONTIER5 safety and patient-reported outcomes data support Mim8 as a potential future treatment option for people living with hemophilia A and demonstrate our continued commitment to developing innovative treatment options for the hemophilia community," said Stephanie Seremetis, Chief Medical Officer and CVP for Rare Disease at Novo Nordisk. "These results give valuable insights into hemophilia A management, highlight the feasibility of directly switching to Mim8 from emicizumab, and reveal a strong patient preference for the Mim8 pen-injector device." Novo Nordisk aims to submit Mim8 for U.S. and EU regulatory review during 2025. Data from the ongoing phase 3 FRONTIER program will be disclosed at upcoming congresses and in publications in 2025 and 2026. About hemophiliaHemophilia is a rare inherited bleeding disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding.4 It is estimated to affect approximately 1,125,000 people worldwide.5 There are different types of hemophilia, which are characterized by the type of clotting factor protein that is defective or missing.4 Hemophilia A is caused by a missing or defective clotting Factor VIII (FVIII), and hemophilia B is caused by a missing or defective clotting Factor IX.4 Inhibitors are an immune system response to the clotting factors in replacement therapy. Currently, it is estimated that up to 30% of people living with severe hemophilia A have inhibitors that can cause replacement therapies to stop working.6 About Mim8Mim8 is an investigational FVIIIa mimetic bispecific antibody designed with the aim to deliver once-monthly, once-every-two-weeks, or once-weekly prophylaxis for people living with hemophilia A, with or without inhibitors.7,8 Administered under the skin, Mim8 bridges Factor IXa and Factor X. This action replaces FVIII function, which helps restore the body's thrombin generation capacity, helping blood to clot.7,9 The use of Mim8 in people living with hemophilia A is investigational and not approved by regulatory authorities or available anywhere in the world. About the FRONTIER5 trialFRONTIER5 is a single-arm, open-label, 26-week, phase 3b trial evaluating the safety of switching from previous emicizumab prophylaxis treatment directly to Mim8 prophylaxis treatment using the Mim8 pen-injector in adults and adolescents with hemophilia A, with or without inhibitors.1 The FRONTIER clinical program investigates Mim8 as a prophylaxis treatment for people with hemophilia A, with or without inhibitors. The phase 3 program includes FRONTIER1, FRONTIER2, FRONTIER3, FRONTIER4 and FRONTIER5.1,8,10-12 About Novo Nordisk Novo Nordisk is a leading global healthcare company that's been making innovative medicines to help people with diabetes lead longer, healthier lives for more than 100 years. This heritage has given us experience and capabilities that also enable us to drive change to help people defeat other serious chronic diseases such as obesity, rare blood, and endocrine disorders. We remain steadfast in our conviction that the formula for lasting success is to stay focused, think long-term, and do business in a financially, socially, and environmentally responsible way. With a U.S. presence spanning 40 years, Novo Nordisk U.S. is headquartered in New Jersey and employs over 10,000 people throughout the country across 12 manufacturing, R&D, and corporate locations in eight states plus Washington DC. For more information, visit Facebook, Instagram, and X. Contacts for further information: Media: Liz Skrbkova (US)+1 609 917 0632NNIMediaTeam@ James-Brown (Global)+45 3079 9289Globalmedia@ Investors: Frederik Taylor Pitter (US) +1 609 613 0568fptr@ Martin Wiborg Rode (Global)+45 3075 5956jrde@ Meyer (Global) +45 3079 6656 azey@ Schaap Melvold (Global) +45 3077 5649 idmg@ Ung (Global)+45 3077 6414mxun@ References Oldenberg J, Benson G, Chowdaryet P, et al. FRONTIER5 direct switch study: safety of initiating Mim8 prophylaxis without washout of emicizumab. Oral presentation presented at the Congress of the International Society on Thrombosis and Haemostasis 2025; June 21-25 2025; Walter E. Washington Convention Center, Washington D.C., US. Session code 13686. Mahlangu J, Ahuja S, Cockrell E, et al. FRONTIER5 device handling and patient-reported outcomes. Oral presentation presented at the Congress of the International Society on Thrombosis and Haemostasis 2025; June 21–25 2025; Walter E. Washington Convention Center, Washington D.C., US. Session code 13786. A Research Study Looking at How Safe it is to Switch From Emicizumab to Mim8 in People With Haemophilia A (FRONTIER5). Last accessed May 2025. Available at MedlinePlus. Hemophilia. Last accessed May 2025. Available at Iorio A, Stonebraker JS, Chambost H, et al. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries. Ann Intern Med. 2019;171:540-546. Kim JY, You CW. The prevalence and risk factors of inhibitor development of FVIII in previously treated patients with hemophilia A. Blood Res. 2019;54:204-209. Ostergaard H, Lund J, Greisen PJ, et al. A factor VIIIa-mimetic bispecific antibody, Mim8, ameliorates bleeding upon severe vascular challenge in hemophilia A mice. Blood. 2021;138:1258-1268. A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A (FRONTIER4). Last accessed May 2025. Available at U.S. National Library of Medicine. F8 gene. MedlinePlus Genetics. Last accessed May 2025. Available at A Research Study Investigating Mim8 in People With Haemophilia A. Last accessed June 2025. Available at A Research Study Investigating Mim8 in Adults and Adolescents With Haemophilia A With or Without Inhibitors. Last accessed May 2025. Available at A Research Study Looking at Mim8 in Children With Haemophilia A With or Without Inhibitors. Last accessed May 2025. Available at Novo Nordisk is a registered trademark of Novo Nordisk A/S. © 2025 Novo Nordisk All rights reserved. US25HRBD00174 June 2025 View original content to download multimedia: SOURCE Novo Nordisk Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
22-06-2025
- Health
- Yahoo
Mim8 prophylaxis treatment shown to be well-tolerated when switching from emicizumab in people with hemophilia A in new phase 3 data presented at the ISTH 2025 Congress
New FRONTIER5 results show a direct switch to investigational Mim8 (denecimig) prophylaxis treatment from emicizumab, without the need for a washout period, was well-tolerated in adults and adolescents with hemophilia A, with or without inhibitors1 FRONTIER5 Patient-Reported Outcomes assessment found the Mim8 pen-injector easy to use with strong user preference over their emicizumab injection system2 PLAINSBORO, N.J., June 22, 2025 /PRNewswire/ -- Novo Nordisk today presented results from the phase 3b FRONTIER5 trial showing that a direct switch to investigational Mim8 (denecimig) prophylaxis from emicizumab treatment, without a washout period or Mim8 loading dose, was well tolerated in adults and adolescents living with hemophilia A, with or without inhibitors.1 Additionally, a FRONTIER5 Patient-Reported Outcomes (PROs) assessment found the Mim8 pen-injector easy to use with an overall strong user preference for the pen-injector, in comparison to previous injection systems.2 The results were presented at the International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington, D.C. "Continuous prophylactic coverage is critical to avoiding breakthrough bleeds in people living with hemophilia; with new non-factor therapeutic options, many people could have hesitations about switching treatment options. These data demonstrate that switching to Mim8 from emicizumab can be done without requiring a washout period," said Allison P. Wheeler, MD, Washington Center for Bleeding Disorders, Seattle, WA. "This is critical in ensuring that individuals maintain continuous protection against bleeding events as we seek to help address the ongoing needs of people living with this complex disease." In the open-label phase 3 FRONTIER5 safety study, 61 adults and adolescents aged 12 years and older with hemophilia A were enrolled.3 No thromboembolic events, hypersensitivity reactions, or treatment-emergent adverse events (TEAEs) leading to discontinuation were observed, and there was no evidence of neutralizing anti-Mim8 antibodies.1 Additional safety information from FRONTIER5 demonstrate that between Week 0 and Week 26 of treatment, there were 107 TEAEs observed in 43 patients (70.5%), most of which were mild to moderate (88.6%). There were 24 TEAEs that were possibly/probably related to Mim8 reported in 18 patients (29.5%). No thromboembolic events, hypersensitivity reactions, or TEAEs leading to discontinuation were observed.1 The PROs data from FRONTIER5 indicated overall (97%; n=57/59) patients preferred the Mim8 pen injection, with 97% of those patients reporting a "very strong" or "fairly strong" preference in comparison to their previous emicizumab injection system. Of the participants who completed the Hemophilia Device Handling and Preference Assessment (HDHPA) questionnaire at week 26, 98% (n=58/59) found the Mim8 pen-injector "very easy" or "easy" to use, and 95% (n=56/59) found it "much easier" or "easier" compared with their previous administration method. All participants (100%) were "extremely confident" or "very confident" in using the pen-injector correctly.2 "The FRONTIER5 safety and patient-reported outcomes data support Mim8 as a potential future treatment option for people living with hemophilia A and demonstrate our continued commitment to developing innovative treatment options for the hemophilia community," said Stephanie Seremetis, Chief Medical Officer and CVP for Rare Disease at Novo Nordisk. "These results give valuable insights into hemophilia A management, highlight the feasibility of directly switching to Mim8 from emicizumab, and reveal a strong patient preference for the Mim8 pen-injector device." Novo Nordisk aims to submit Mim8 for U.S. and EU regulatory review during 2025. Data from the ongoing phase 3 FRONTIER program will be disclosed at upcoming congresses and in publications in 2025 and 2026. About hemophiliaHemophilia is a rare inherited bleeding disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding.4 It is estimated to affect approximately 1,125,000 people worldwide.5 There are different types of hemophilia, which are characterized by the type of clotting factor protein that is defective or missing.4 Hemophilia A is caused by a missing or defective clotting Factor VIII (FVIII), and hemophilia B is caused by a missing or defective clotting Factor IX.4 Inhibitors are an immune system response to the clotting factors in replacement therapy. Currently, it is estimated that up to 30% of people living with severe hemophilia A have inhibitors that can cause replacement therapies to stop working.6 About Mim8Mim8 is an investigational FVIIIa mimetic bispecific antibody designed with the aim to deliver once-monthly, once-every-two-weeks, or once-weekly prophylaxis for people living with hemophilia A, with or without inhibitors.7,8 Administered under the skin, Mim8 bridges Factor IXa and Factor X. This action replaces FVIII function, which helps restore the body's thrombin generation capacity, helping blood to clot.7,9 The use of Mim8 in people living with hemophilia A is investigational and not approved by regulatory authorities or available anywhere in the world. About the FRONTIER5 trialFRONTIER5 is a single-arm, open-label, 26-week, phase 3b trial evaluating the safety of switching from previous emicizumab prophylaxis treatment directly to Mim8 prophylaxis treatment using the Mim8 pen-injector in adults and adolescents with hemophilia A, with or without inhibitors.1 The FRONTIER clinical program investigates Mim8 as a prophylaxis treatment for people with hemophilia A, with or without inhibitors. The phase 3 program includes FRONTIER1, FRONTIER2, FRONTIER3, FRONTIER4 and FRONTIER5.1,8,10-12 About Novo Nordisk Novo Nordisk is a leading global healthcare company that's been making innovative medicines to help people with diabetes lead longer, healthier lives for more than 100 years. This heritage has given us experience and capabilities that also enable us to drive change to help people defeat other serious chronic diseases such as obesity, rare blood, and endocrine disorders. We remain steadfast in our conviction that the formula for lasting success is to stay focused, think long-term, and do business in a financially, socially, and environmentally responsible way. With a U.S. presence spanning 40 years, Novo Nordisk U.S. is headquartered in New Jersey and employs over 10,000 people throughout the country across 12 manufacturing, R&D, and corporate locations in eight states plus Washington DC. For more information, visit Facebook, Instagram, and X. Contacts for further information: Media: Liz Skrbkova (US)+1 609 917 0632NNIMediaTeam@ James-Brown (Global)+45 3079 9289Globalmedia@ Investors: Frederik Taylor Pitter (US) +1 609 613 0568fptr@ Martin Wiborg Rode (Global)+45 3075 5956jrde@ Meyer (Global) +45 3079 6656 azey@ Schaap Melvold (Global) +45 3077 5649 idmg@ Ung (Global)+45 3077 6414mxun@ References Oldenberg J, Benson G, Chowdaryet P, et al. FRONTIER5 direct switch study: safety of initiating Mim8 prophylaxis without washout of emicizumab. Oral presentation presented at the Congress of the International Society on Thrombosis and Haemostasis 2025; June 21-25 2025; Walter E. Washington Convention Center, Washington D.C., US. Session code 13686. Mahlangu J, Ahuja S, Cockrell E, et al. FRONTIER5 device handling and patient-reported outcomes. Oral presentation presented at the Congress of the International Society on Thrombosis and Haemostasis 2025; June 21–25 2025; Walter E. Washington Convention Center, Washington D.C., US. Session code 13786. A Research Study Looking at How Safe it is to Switch From Emicizumab to Mim8 in People With Haemophilia A (FRONTIER5). Last accessed May 2025. Available at MedlinePlus. Hemophilia. Last accessed May 2025. Available at Iorio A, Stonebraker JS, Chambost H, et al. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries. Ann Intern Med. 2019;171:540-546. Kim JY, You CW. The prevalence and risk factors of inhibitor development of FVIII in previously treated patients with hemophilia A. Blood Res. 2019;54:204-209. Ostergaard H, Lund J, Greisen PJ, et al. A factor VIIIa-mimetic bispecific antibody, Mim8, ameliorates bleeding upon severe vascular challenge in hemophilia A mice. Blood. 2021;138:1258-1268. A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A (FRONTIER4). Last accessed May 2025. Available at U.S. National Library of Medicine. F8 gene. MedlinePlus Genetics. Last accessed May 2025. Available at A Research Study Investigating Mim8 in People With Haemophilia A. Last accessed June 2025. Available at A Research Study Investigating Mim8 in Adults and Adolescents With Haemophilia A With or Without Inhibitors. Last accessed May 2025. Available at A Research Study Looking at Mim8 in Children With Haemophilia A With or Without Inhibitors. Last accessed May 2025. Available at Novo Nordisk is a registered trademark of Novo Nordisk A/S. © 2025 Novo Nordisk All rights reserved. US25HRBD00174 June 2025 View original content to download multimedia: SOURCE Novo Nordisk Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Daily Mail
13-05-2025
- Health
- Daily Mail
DC man suffers terrifying rare bleeding disorder after Covid vaccine booster
A Washington DC man was left excessively bleeding due to a rare side effect linked to his Covid vaccine booster. The unidentified man who is in his 70s went to the hospital after he saw blood in his stool - believing it was due to the colonoscopy he had the day before. But while examining him, doctors also noticed large, dark bruises on both of his arms that had developed within the span of a few days. Tests revealed he was suffering from hemophilia A, where the body prevents blood from clotting, causing excessive bleeding from wounds for days at end. This prolonged bleeding can cause blood to accumulate beneath the skin, forming a bruise and make the body more susceptible to injuries. In this particular case, since the patient had sustained wounds from a colorectal procedure, they were mostly bleeding internally and the blood was only visible in their stool. The patient had no family history of the condition and was not actively bleeding, according to a medical report about his case. However, they traced his symptoms back a week after the elderly patient had received his Moderna Covid vaccine booster - marking it as his fifth shot. The experts theorized that it was possible the shot triggered his immune system to produce antibodies against his body's clotting process and caused the disorder. Typically, mRNA vaccines work by prompting the immune system to produce SARS-CoV-2 spike proteins to attack the Covid virus in the body through a piece of genetic code. However, in this case doctors believe that it may be possible that the vaccine also made the body stop recognizing its own cells as safe and began developing antibodies against the clotting process. Experts have noted that adverse reactions to Covid vaccines are extremely rare and that the benefits of getting the shot outweigh potential complications After his diagnosis, the experts reviewed 21 cases of AHA developing in patients after receiving Covid vaccines between 2020 to 2022 and discovered that they usually were in their 70s and symptoms tend to show 14 days after receiving the shot. They also noted that the condition can be fatal in about 10.3 percent of cases which is why early diagnosis and treatment are crucial for survival. AHA can cause bleeding in the skin, muscles, and soft tissues but more concerningly, it can also lead to internal bleeding, including in the brain and gastrointestinal tract - which if not controlled, can cause the body to start bleeding out and in certain cases, lead to death. It remains unclear exactly how the vaccine triggered specific clotting antibody production in the patients' bodies. Most cases OF AHA occurred after people received their first or second dose of the Covid vaccine - when the immune response is highest. However, this particular man developed the bleeding disorder after his fifth dose - making his case distinct. When the elderly man initially went to the doctors, his white blood cell count, platelets count, liver and kidney function all appeared normal during tests. But much to their surprise, the bruising on his arms continued to worsen and he developed large marks on his left knee two weeks later - prompting doctors to order more intensive blood tests which included a plasma test. Results showed that while his white cell and platelets counts were normal, they were certain antibodies present in his plasma that were not allowing the body's clotting process to function. Further testing showed that his body's FVIII, crucial protein involved in forming blood clots to stop bleeding, levels were only at one percent in his body. As a result, he was diagnosed with acquired hemophilia A - which is known to primarily target the FVIII protein in the body. Typical symptoms of this condition include nosebleeds, bruising throughout the body, solid swellings of congealed blood, blood in the urine and gastrointestinal bleeding. Consequently, patients also develop also complications associated with abnormal, uncontrolled bleeding into the muscles, skin and soft tissue that can occur spontaneously, during surgery or following trauma. While it is usually treatable, AHA can potentially cause life-threatening bleeding complications in certain cases. Experts estimate that the condition affects about two people per a million in the US - meaning that it is diagnosed in nearly 1,000 people in a population of 340 million people. As for treatment, the elderly man was asked to visit a haematology clinic every week and began taking cyclophosphamide (an immunosuppressant medication used to treat autoimmune diseases). The doctors progressively decreased his cyclophosphamide dosage as his FVIII levels improved and he showed signs of recovery. Along with this, he was also given medications to prevent fungal infections and protect the stomach.
Technical.ly
04-04-2025
- Business
- Technical.ly
Spark Therapeutics files notice to lay off 300 employees this year
Cell and gene therapy standout Spark Therapeutics is undergoing its second shakeup in a month, with plans to lay off about half of its workforce. Several hundred people will be affected. A WARN notice, which companies file to provide advance notice of layoffs, reported 298 eliminated positions in the Philadelphia region. A Spark Therapeutics spokesperson told the Philadelphia Business Journal on Thursday it would be laying off 337 of its almost 650 employees. These changes are expected to occur in three waves: in May, July and at the end of 2025. The remaining 310 employees will be incorporated into parent company Roche, a multinational pharmaceutical company. Spark first announced on January 30 the decision to integrate more of its work into Roche, spokesperson Denise Bradley told The impacted employees will be eligible for severance, outplacement services and will be able to apply for other roles within Roche. Spark's plans for its University City-based Gene Therapy Innovation Center in Philadelphia have not changed, Bradley said. Last month, Roche classified the former startup as a financial loss following the end of its trial for a hemophilia A gene therapy treatment, the Philadelphia Inquirer reported. The company is still working on a new Hemophilia A gene product, Spark previously told The layoff announcement comes less than a year after Spark's previous workforce reduction, when it let less than 50 of its employees go in July 2024. At the end of last year, the company welcomed Roche veteran Sylke Poehling as its new CEO, replacing Ron Phillip, who had been in the role since 2022. Spark's year of downsizing Recent struggles at Spark, which was founded by Jeffrey Marrazzo in 2013 and was considered a big Philadelphia success story, indicate the need for the gene therapy sector to make manufacturing more cost effective, Rebecca Grant, senior director of life sciences and innovation for the city's Department of Commerce, previously told But the company itself heavily contributed to the development of the industry as a whole. 'They really created a lot of recognition for gene therapy and innovation,' Grant said. 'Now more people understand what gene therapy means and how it can literally cure disease.' In 2021, the company announced plans for a 500,000-square-foot Gene Therapy Innovation Center in University City. At the time, Spark said the new site would house over 500 jobs. The Innovation Center is still expected to be completed next year, Spark spokesperson Bradley previously told The Penn spinout is known for developing the first FDA-approved gene therapy, Luxturna. Pharma giant Roche acquired Spark in 2019 for $4.8 billion, the largest VC-backed exit in Philadelphia at the time. 'Gene therapy is not a huge sector, and Spark was a trailblazer,' Dean Miller, president of the Philadelphia Alliance for Capital and Technologies, previously said. '[It's] never easy when your trailblazer starts to disappear a little further.' Sarah Huffman is a 2022-2024 corps member for Report for America, an initiative of The Groundtruth Project that pairs young journalists with local newsrooms. This position is supported by the Lenfest Institute for Journalism.
