Aankondiging • Jul 24
Prime Medicine Announces U.S. FDA Clearance of Investigational New Drug Application for PM577a in H1069Q-Mutated Wilson Disease Prime Medicine, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for PM577a, an investigational in vivo Prime Editor for Wilson disease (WD). With the IND cleared, PM577a may proceed to clinical study in the United States. Together with the Company’s previously announced New Zealand Clinical Trial Application (CTA) clearance, the IND clearance establishes a global Phase 1/2 program and opens participation to patients in the United States, where H1069Q is the single most common pathogenic variant causing WD. The Phase 1/2 clinical trial is an open-label, global, first-in-human study designed to evaluate the safety, tolerability, biological activity, and efficacy of ascending doses of PM577a in adults and adolescents with WD. The study will initially enroll adults who are clinically stable on standard-of-care therapy. Biological activity and efficacy assessments may include copper efflux by 64Cu PET, serum ceruloplasmin, non-ceruloplasmin bound copper, 24-hour urinary copper excretion, and hepatic copper by biopsy. PM577 is a family of LNP-formulated Prime Editing products designed to correct pathogenic ATP7B mutations in hepatocytes via a single intravenous infusion. Prime Medicine’s initial candidate, PM577a, targets the ATP7B H1069Q allele, which accounts for approximately 30–50% of WD-associated variants in the United States and Europe. The Company intends to develop additional products to address the majority of pathogenic variants on a global basis. Currently, a follow-on candidate is in pre-clinical development targeting R778L, the most common mutant allele in East Asian populations. New Risk • Jul 09
New minor risk - Share price stability The company's share price has been volatile over the past 3 months. It is more volatile than 75% of American stocks, typically moving 11% a week. This is considered a minor risk. Share price volatility indicates the stock is highly sensitive to market conditions or economic conditions rather than being sensitive to its own business performance, which may also be inconsistent. It also increases the risk of potential losses in the short term as the stock tends to have larger drops in price more frequently than other stocks. Currently, the following risks have been identified for the company: Major Risks Less than 1 year of cash runway based on free cash flow trend (-US$159m free cash flow). Earnings are forecast to decline by an average of 3.8% per year for the foreseeable future. Shareholders have been substantially diluted in the past year (38% increase in shares outstanding). Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$217m net loss in 3 years). Share price has been volatile over the past 3 months (11% average weekly change). Revenue is less than US$5m (US$4.0m revenue). Aankondiging • Jun 22
Prime Medicine Receives U.S. Fda Regenerative Medicine Advanced Therapy Designation for Pm359 for the Treatment of Chronic Granulomatous Disease Prime Medicine, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to PM359, an investigational autologous Prime Edited hematopoietic stem cell therapy for the treatment of p47phox-deficient chronic granulomatous disease (CGD). RMAT designation was granted based on Phase 1/2 clinical data, including data previously published in The New England Journal of Medicine, and will provide the benefits of intensive FDA guidance and expedited review through the program’s development. The combination of RMAT, Fast Track, Orphan Drug, and Rare Pediatric Disease Designations underscores the seriousness of CGD and the need for transformative therapies that deliver durable benefit, while positioning us to engage with the FDA on the most efficient path to a Biologics License Application. RMAT designation was established under the 21st Century Cures Act to expedite the development and review of regenerative medicine therapies intended to treat serious or life-threatening diseases when preliminary clinical evidence indicates the therapy has the potential to address unmet medical need. RMAT designation provides all the benefits of Fast Track and Breakthrough Therapy Designations, including intensive FDA interaction, discussions on surrogate or intermediate endpoints that may support accelerated approval, and eligibility for rolling and priority review of a future Biologics License Application. The initial clinical trial results were reported in the December 2025 publication in The New England Journal of Medicine. The data demonstrate that a single dose of PM359 drove rapid engraftment and a durable, clinically meaningful restoration of immune cell function in both treated patients, with neutrophil activity well above the level associated with clinical benefit and a safety profile consistent with busulfan-based conditioning alone. The findings support the potential of PM359 to deliver a one-time, disease-modifying treatment for patients with p47phox-deficient CGD. PM359 is an investigational autologous hematopoietic stem cell therapy designed to correct the delGT mutation in NCF1, the most prevalent disease-causing mutation in p47phox-deficient CGD. PM359 is being evaluated in an ongoing Phase 1/2, multinational, first-in-human trial. In addition to RMAT designation, PM359 has received Fast Track, Orphan Drug, and Rare Pediatric Disease Designations from the FDA. Chronic granulomatous disease (CGD) is a rare inherited hematologic disorder characterized by susceptibility to severe, difficult-to-treat infections and inflammatory and autoimmune complications. CGD is caused by mutations in any one of the subunits comprising the NADPH oxidase complex, which is required for phagocytic cells, in particular neutrophils, to destroy many invasive microorganisms. CGD causative mutations are estimated to occur in between one in 100,000 and one in 200,000 births in the United States, with the p47phox form accounting for approximately 25% of cases. Current standard of care relies on lifelong antimicrobial prophylaxis and, in select cases, allogeneic hematopoietic stem cell transplantation, which carries significant morbidity and is not accessible to all patients. There are no approved gene editing therapies for CGD. Aankondiging • Jun 19
Prime Medicine, Inc. Receives New Zealand Clearance For Clinical Trial Application For PM577a In H1069Q-Mutated Wilson Disease Prime Medicine, Inc. announced that the New Zealand Medicines and Medical Devices Safety Authority (Medsafe) has cleared the Company’s Clinical Trial Application (CTA) for PM577a, an investigational Prime Editor for Wilson Disease (WD). The clearance represents the first clinical authorization for an in vivo Prime Editing therapy from Prime Medicine and enables the initiation of the Company’s global Phase 1/2 study. The Phase 1/2 clinical trial is an open-label, global, first-in-human study designed to evaluate the safety, tolerability, biological activity, and efficacy of ascending doses of PM577a in adults and adolescents with WD. The study will initially enroll adults who are clinically stable on standard-of-care therapy. Biological activity and efficacy assessments may include copper efflux by 64Cu PET, serum ceruloplasmin, non-ceruloplasmin bound copper, 24-hour urinary copper excretion, and hepatic copper by biopsy. PM577 is a family of LNP-formulated Prime Editing products designed to correct pathogenic ATP7B mutations in hepatocytes via a single intravenous infusion. Prime Medicine’s initial candidate, PM577a, targets the ATP7B H1069Q allele, which accounts for approximately 30–50% of WD-associated variants in the United States and Europe. The Company intends to develop additional products to address the majority of pathogenic variants on a global basis. Currently, a follow-on candidate is in pre-clinical development targeting R778L, the most common mutant allele in East Asian populations. Aankondiging • Apr 25
Prime Medicine, Inc., Annual General Meeting, Jun 05, 2026 Prime Medicine, Inc., Annual General Meeting, Jun 05, 2026. Aankondiging • Apr 16
Prime Medicine, Inc. Appoints Svetlana Makhni as Chief Financial Officer Prime Medicine, Inc. announced the appointment of Svetlana Makhni as Chief Financial Officer (CFO). Ms. Makhni will oversee Prime Medicine’s financial operations and strategy, including investor relations, financial planning and analysis, and corporate development. Ms. Makhni brings over 20 years of experience across multiple biotechnology and healthcare CFO and investment banking roles. Prior to joining Prime Medicine, she served as CFO of Marengo Therapeutics, a clinical-stage immuno-oncology company, where she led all aspects of the finance organization, including business development, investor relations and corporate development. In this role, Ms. Makhni played a key role in the execution of multiple high-value strategic partnerships, including transactions with Ipsen and Gilead Sciences. Previously, Ms. Makhni served as CFO of Escient Pharmaceuticals and CFO and Head of Operations at Bierman ABA. Earlier in her career, Ms. Makhni spent over a decade in investment banking and financial services at BMO Capital Markets, Goldman Sachs, Westbrook Partners and The Blackstone Group, advising public and private companies on equity and debt financings, mergers and acquisitions, and other strategic transactions. Ms. Makhni holds an M.B.A. from Harvard Business School and a B.S. from the Wharton School of the University of Pennsylvania. Aankondiging • Nov 07
Prime Medicine, Inc. has filed a Follow-on Equity Offering in the amount of $200 million. Prime Medicine, Inc. has filed a Follow-on Equity Offering in the amount of $200 million.
Security Name: Common Stock
Security Type: Common Stock
Transaction Features: At the Market Offering Aankondiging • Nov 04
Prime Medicine, Inc. Appoints Matthew Hawryluk as Chief Business Officer Prime Medicine, Inc. announced the appointment of Matthew Hawryluk, Ph.D., M.B.A. as Chief Business Officer (CBO). Dr. Hawryluk will lead Primes corporate and business development initiatives, corporate strategy, and alliance management functions, advancing the companys efforts to expand the reach of Prime Editing through strategic partnerships and collaborations. Dr. Hawryluk joins Prime Medicine with nearly two decades of leadership experience spanning business development, strategy, and corporate operations in the biotechnology industry. Most recently, he served as Chief Business Officer at AIRNA Corporation, where he led corporate and business development, finance, and investor relations, and played a key role in securing an oversubscribed $155 million Series B financing. Dr. Hawryluk served for nearly a decade as Executive Vice President and Chief Business Officer at Gritstone bio, Inc., guiding its growth from early stage to a public company and spearheading its expansion into infectious diseases. He led transactions totaling more than $2.5 billion in potential value, including landmark collaborations with Gilead Sciences, bluebird bio, and multiple government and nonprofit organizations such as BARDA, CEPI, NIAID, and the Bill &Melinda Gates Foundation. Earlier in his career, Dr. Hawryluk held senior roles at Foundation Medicine, where he was instrumental in forging the companys transformative strategic partnership and majority acquisition by Roche/Genentech. His experience also includes business development roles at Thermo Fisher Scientific and work in venture capital and translational science. Dr. Hawryluk holds a Ph.D. in Cell Biology and Protein Biochemistry from the University of Pittsburgh School of Medicine, an M.B.A. from the Carnegie Mellon University Tepper School of Business, and a B.S. in Biochemistry from the University of Notre Dame. Dr. Hawryluk serves on the Boards of Directors of Predictive Oncology Inc. and OuroTech, Inc. (Pear Bio). Aankondiging • Aug 01
Prime Medicine, Inc. has completed a Follow-on Equity Offering in the amount of $125.4 million. Prime Medicine, Inc. has completed a Follow-on Equity Offering in the amount of $125.4 million.
Security Name: Common Stock
Security Type: Common Stock
Securities Offered: 38,000,000
Price\Range: $3.3
Discount Per Security: $0.198 Aankondiging • Jul 31
Prime Medicine, Inc. has filed a Follow-on Equity Offering. Prime Medicine, Inc. has filed a Follow-on Equity Offering.
Security Name: Common Stock
Security Type: Common Stock Aankondiging • May 21
Prime Medicine, Inc. Announces Breakthrough Clinical Data Showing Rapid Restoration of DHR Positivity After Single Infusion of PM359 Prime Medicine, Inc. announced positive initial data from the first patient dosed in its ongoing Phase 1/2 clinical study of PM359 in Chronic Granulomatous Disease (CGD). Preliminary results from the first patient demonstrated that PM359 was well-tolerated, showed rapid engraftment and restored NADPH oxidase activity to well above the threshold for clinical benefit, as measured by the dihydrorhodamine (DHR) assay. Going forward, Prime Medicine will focus its resources on advancing its in vivo liver franchise, where the Company is advancing programs to cure two of the largest genetic liver diseases, Wilson's Disease and Alpha-1 Antitrypsin Deficiency (AATD). Prime Medicine will also continue its in vivo Cystic Fibrosis program with support from the Cystic Fibrosis Foundation, and its efforts to develop Prime Edited CAR-T products for hematology, immunology and oncology in partnership with Bristol Myers Squibb. In addition, Prime Medicine will continue to pursue additional business development opportunities to accelerate innovation, ensure the broadest application of Prime Editing, and further bolster its financial resources. PM359 comprisesautologous hematopoietic stem cells (HSCs) modified ex vivo using Prime Editors that have been designed to correct a high percentage of cells containing the disease-causing mutation. PM359 has received rare pediatric drug designation and orphan drug designation from the U.S. Food and Drug Administration. Chronic Granulomatous disease (CGD) is a rare inherited hematologic disorder characterized by susceptibility to severe, difficult-to-treat infections, and inflammatory/autoimmune complications. Aankondiging • Apr 23
Prime Medicine, Inc., Annual General Meeting, Jun 04, 2025 Prime Medicine, Inc., Annual General Meeting, Jun 04, 2025. Aankondiging • Mar 18
Prime Medicine, Inc. Unveils Program for the Treatment of Alpha-1 Antitrypsin Deficiency Prime Medicine, Inc. unveiled a preclinical program for the treatment of alpha-1 antitrypsin deficiency (AATD), the next program within its liver franchise. Prime Medicine expects to file an investigational new drug (IND) and/or clinical trial application (CTA) in mid-2026. Prime Medicine’s Approach to AATD: Prime Medicine’s program leverages the Company’s universal liver lipid nanoparticle (LNP) to edit the E342K (PiZ) mutation in the SERPINA1 gene, the prevalent disease-causing mutation in AATD, restoring the mutated protein sequence back to wild-type M protein, with the potential to treat both lung- and liver-associated disease. In Prime Medicine’s initial in vivo data, LNP delivery of Prime Editors targeting the PiZ (E324K) mutation demonstrated up to 72% precise correction of the SERPINA1 gene in the hepatocytes of fully humanized mice. Importantly, this restored over 95% of serum AAT to the corrected isoform, with healthy AAT (M-AAT) protein in the serum at levels well above 20µM, indicating restoration of M-AAT to normal levels in a humanized mouse model. Prime Medicine’s universal LNP contains a GalNAc-targeting ligand (GalNAc-LNP), a validated component for liver-specific delivery of gene editors. In preclinical studies, delivery of Prime Editors using a GalNAc-LNP has demonstrated increased potency, and both an improved safety profile and biodistribution when benchmarked against other LNPs that have gone into the clinic. Preclinical studies using unoptimized surrogate Prime Editors for genetic diseases in non-human primate (NHP) models showed greater than 50% editing, with an excellent safety profile, and no detectable off-target edits or unintended edits at the target site. Based on these data, Prime Medicine believes Prime Editing has the ability to correct disease-causing mutations with high efficiency, without introducing off-target or bystander edits. Prime Medicine is advancing its AATD program through the final stages of lead optimization and expects to file an IND and/or CTA filing in mid-2026. AATD is a progressive, genetic disorder caused by mutations in the SERPINA1 gene; these mutations lead to decreased levels of circulating AAT protein in the blood, as well as the build-up of toxic mutant AAT protein in the liver. Because the primary function of AAT is to protect lungs from inflammation caused by infection and inhaled irritants, low levels of circulating AAT can result in lung-related symptoms, including shortness of breath, wheezing, chronic cough and frequent chest colds. Additionally, the build-up of abnormal AAT in the liver can cause jaundice, ascites, and cirrhosis. There are currently no disease-modifying or curative treatments approved for the approximately 200,000 people in the United States and European Union with AATD, and many patients ultimately progress to liver failure or severe lung disease, eventually resulting in premature death. Aankondiging • May 01
Prime Medicine, Inc., Annual General Meeting, Jun 12, 2024 Prime Medicine, Inc., Annual General Meeting, Jun 12, 2024, at 14:00 US Eastern Standard Time. Agenda: To elect Wendy Chung, M.D., Ph.D., Kaye Foster, Keith Gottesdiener, M.D., and Jeffrey Marrazzo as Class II Directors, each to serve until the 2027 annual meeting of stockholders, and until his or her respective successor shall have been duly elected and qualified, or until his or her earlier death, resignation or removal; to approve an amendment to third amended and restated certificate of incorporation to limit the liability of certain of officers as permitted by Delaware law; to ratify the appointment of PricewaterhouseCoopers LLP as independent registered public accounting firm for the fiscal year ending December 31, 2024 and to transact such other business as may properly come before the Annual Meeting or any continuations, adjournments, and postponements thereof. Aankondiging • Apr 30
Prime Medicine Announces FDA Clearance of Investigational New Drug (IND) Application for PM359 for the Treatment of Chronic Granulomatous Disease (CGD) Prime Medicine, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for PM359, submitted on March 29, for the treatment of chronic granulomatous disease (CGD), enabling the Company to initiate its global Phase 1/2 clinical trial in the United States. The Phase 1/2 clinical trial is a multinational, first-in-human trial designed to assess the safety, biological activity and preliminary efficacy of PM359 in adult and pediatric study participants. Initial study participants will be adults with stable disease. If safety and biological activity are demonstrated in this cohort, the study is designed to enroll participants with active infection or severe inflammation as well as adolescent and pediatric participants. Participants will be followed for safety, including engraftment and reconstitution of the hematopoietic system, early biological markers of restored immune function, and long-term resolution and prevention of infectious and inflammatory complications of CGD. Prime Medicine expects to report initial clinical data from the study in 2025. Aankondiging • Jan 07
Prime Medicine Announces Chief Financial Officer Changes, Effective January 17, 2024 Prime Medicine, Inc. announced the appointment of Allan Reine, M.D., as the Company’s Chief Financial Officer, effective January 17, 2024. Dr. Reine, age 49, a seasoned financial executive with over twenty years’ experience in the biotechnology industry, Dr. Reine will be responsible for the company’s financing strategy and investor relations, and will oversee all financial operations as Prime Medicine begins its transition into a clinical company. Prior to joining Prime Medicine, Dr. Reine was Chief Financial Officer at Foghorn Therapeutics and, before that, at Pieris Pharmaceuticals. Dr. Reine also serves as Chairman of the Board of ONK Therapeutics. Previously, Dr. Reine managed various healthcare portfolios primarily focused on biotechnology and pharmaceutical companies at Lombard Odier Asset Management, Citi Principal Strategies, SAC Capital, Trivium Capital and Alexandra Investment Management. He started his career at CIBC World Markets where he worked in both biotechnology investment banking and biotechnology equity research. Dr. Reine received his M.D. from the University of Toronto, and his Bachelor of Science in statistical sciences from the University of Western Ontario. Effective January 17, 2024, Carman Alenson will transition from Interim Chief Financial Officer and Chief Accounting Officer to Senior Vice President, Finance and Chief Accounting Officer. She will step down from the role of principal financial officer and will continue in her role of principal accounting officer. Aankondiging • Jan 06
Prime Medicine, Inc. Announces Executive Changes, Effective January 17, 2024 On January 4, 2024, the Board of Directors of Prime Medicine, Inc. appointed Allan Reine as the principal financial officer of the company effective as of January 17, 2024. Dr. Reine, age 49, has over 20 years of experience in the biotechnology sector. Prior to joining the Company, he was Chief Financial Officer of Foghorn Therapeutics, Inc., a biotechnology company, from September 2019 to January 2024 and Chief Financial Officer of Pieris Pharmaceuticals, Inc. from August 2017 to September 2019. Previously, Dr. Reine managed various healthcare portfolios primarily focused on biotechnology and pharmaceutical companies at Lombard Odier Asset Management, Citi Principal Strategies, SAC Capital, Trivium Capital and Alexandra Investment Management. He started his career at CIBC World Markets where he worked in both biotechnology investment banking and biotechnology equity research. Dr. Reine is currently chairman of the board of directors of ONK Therapeutics, Inc. where he has served on the board of directors since March 2022. Dr. Reine received his M.D. from the University of Toronto and his B.S. in Statistical Sciences from the University of Western Ontario. Effective January 17, 2024, Carman Alenson will transition from Interim Chief Financial Officer and Chief Accounting Officer to Senior Vice President, Finance and Chief Accounting Officer. She will step down from the role of principal financial officer and will continue in her role of principal accounting officer. Price Target Changed • Dec 08
Price target decreased by 10% to US$19.71 Down from US$22.00, the current price target is an average from 7 analysts. New target price is 132% above last closing price of US$8.50. Stock is down 50% over the past year. The company is forecast to post a net loss per share of US$1.94 next year compared to a net loss per share of US$4.19 last year. Aankondiging • Oct 28
Prime Medicine, Inc. Presents First-Ever Prime Editing Data in Non-Human Primates Demonstrating Highly Efficient Ability of Prime Editors to Precisely Correct Disease-Causing Mutation of Gsd1b Prime Medicine, Inc. reported new preclinical data demonstrating the ability of liver-targeted Prime Editors to efficiently and precisely correct one of the most prevalent disease-causing mutations of glycogen storage disease 1b (GSD1b) in non-human primates (NHP) and mouse models. The data were presented at the European Society of Gene and Cell Therapy (ESGCT) 2023 Congress in Brussels, Belgium. GSD1b is a rare, serious progressive disease that causes impaired glycogen metabolism and affects approximately 1,500 patients. It results from mutations in the glucose-6-phosphate transporter (G6PT), which is encoded by the gene SLC37A4. Deficiencies in this transporter result in hypoglycemia, or low blood glucose levels, which can be fatal if patients do not adhere to a strict dietary regimen, including consuming slow-release glucose and overnight feeding. P.L348fs and p.G339C mutations are known to be the most prevalent disease-causing mutations and are found in approximately 46-52% of the GSD1b patient population. According to scientific literature and Prime Medicine research, correcting SLC37A4 gene mutations in fewer than 10% of liver cells may be sufficient to reverse many manifestations of this disease. To address the underlying genetic cause of GSD1b, Prime Medicine is advancing Prime Editors that are delivered to the liver by single intravenous infusion and designed to enable a precise correction of the disease-causing mutations, restoring G6PT protein expression and glucose homeostasis. The Prime Editors are composed of a Prime Editor guide RNA (pegRNA) targeting the respective mutations, a nick-guide RNA (ngRNA) and a messenger RNA (mRNA) packaged in Prime’s universal lipid nanoparticle (LNP) formulation that includes a ligand targeting the LNP to hepatocytes. Through high-throughput screening and subsequent optimization, Prime researchers identified pegRNAs that precisely corrected the p.L348fs and p.G339C mutations in liver cells, which were then evaluated in vitro, demonstrating average editing of 77% and 37%, respectively. In today’s presentation at ESGCT, Prime Medicine highlighted data from in vivo rodent and NHP studies with its Prime Editor targeting the p.L348fs mutation. Key findings from the studies showed: Up to 50% whole liver precise editing of p.L348 in NHPs at day 14 without significant on-target unintended edits. Up to 83% of the key target cells, liver hepatocytes, were estimated to have both alleles precisely edited by this single LNP administration. Up to 56% whole liver precise correction of the p.L348fs mutation in a GSD1b humanized mouse model with on-target unintended editing of less than 0.2% across dose levels evaluated. Prime Editing of up to 44% led to restored levels of G6PT protein expression of up to 46%, with the extent of correction directly correlating with the extent of G6PT protein restoration in the humanized mouse model. Redosing of the universal LNP in non-naïve animals was tolerated similarly to naïve animals with no infusion reactions, no body weight changes, and transient, modest liver function changes that resolved by day 7; minimal transient cytokine abnormalities were observed. No detectable off-target edits were observed in patient-derived induced pluripotent stem cells (iPSCs) following a comprehensive off-target screening analysis, consistent with what has been observed to date across Prime Medicine’s extensive off-target analyses for each of its programs. These findings provide important proof-of-concept for Prime Medicine’s LNP liver-targeted delivery approach, and support the further advancement of the Company’s Prime Editors targeting the p.L348fs and p.G339C mutations in GSD1b, as well as its additional liver-targeted programs. Aankondiging • Oct 25
Prime Medicine, Inc. Presents First in Vivo Proof-Of-Concept Prime Editing Data Demonstrating Ability of Prime Editors to Treat Ophthalmological Diseases Prime Medicine, Inc. reported new preclinical data demonstrating the ability of Prime Editors to efficiently and precisely correct the predominant mutations that cause rhodopsin associated autosomal dominant retinitis pigmentosa (RHO adRP). The data were presented at the International Symposium on Retinal Degeneration 2023 Congress (RD2023) in Costa del Sol, Spain. RHO adRP is a rare inherited retinal disease that causes progressive vision loss in early adolescence, leading to eventual blindness in adulthood due to photoreceptor degeneration. It results from mutations in the gene RHO, which encourages rhodopsin, the light-sensitive G protein-coupled receptor involved in phototransduction in rods, a type of photoreceptor, and leads to the progressive loss of rods and, subsequently, cones in the retina. Specifically, today's data highlight the ability of two Prime Editors to correct the predominant mutations causing RHO adRP -- one to correct p.P23H, the most common disease-causing mutation in RHO in the U.S., and one to correct 18 different mutations at a mutational hotspot in RHO, including p.V345L and p.P347L, which are the most prevalent mutations in Europe. New Risk • Oct 18
New minor risk - Share price stability The company's share price has been volatile over the past 3 months. It is more volatile than 75% of American stocks, typically moving 9.5% a week. This is considered a minor risk. Share price volatility indicates the stock is highly sensitive to market conditions or economic conditions rather than being sensitive to its own business performance, which may also be inconsistent. It also increases the risk of potential losses in the short term as the stock tends to have larger drops in price more frequently than other stocks. Currently, the following risks have been identified for the company: Major Risks Earnings are forecast to decline by an average of 8.2% per year for the foreseeable future. Revenue is less than US$1m. Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$213m net loss in 3 years). Share price has been volatile over the past 3 months (9.5% average weekly change). Aankondiging • May 12
Prime Medicine, Inc. Appoints Jeff Marrazzo to Its Board of Directors Prime Medicine, Inc. announce announced appointment of Jeff Marrazzo, co-founder and former Chief Executive Officer of Spark Therapeutics, Inc., to its Board of Directors. Jeff founded and built Spark Therapeutics from an idea incubated within the Children’s Hospital of Philadelphia (CHOP) into the world’s first fully integrated, commercial gene therapy company with over 850 employees at the time of his departure. Under Jeff’s leadership, Spark developed and launched LUXTURNA® for a rare blinding disorder, the first U.S. Food and Drug Administration-approved gene therapy for a genetic disease in the U.S., spearheading the creation of novel reimbursement models to ensure patient access to genetic medicines. While at Spark, Jeff shepherded multiple gene therapies into the clinic for patients with conditions ranging from inherited retinal diseases to rare bleeding and neuromuscular disorders, raising $1 billion in capital and establishing major partnerships with Pfizer and Novartis. Jeff’s orchestration of Spark’s $4.8 billion sale to Roche in 2019 marked a 100-fold increase in the company’s market value over six years. Board Change • Nov 16
High number of new and inexperienced directors There are 11 new directors who have joined the board in the last 3 years. The company's board is composed of: 11 new directors. 1 experienced director. No highly experienced directors. Independent Director David Schenkein is the most experienced director on the board, commencing their role in 2019. The following issues are considered to be risks according to the Simply Wall St Risk Model: Lack of board continuity. Lack of experienced directors. Board Change • Oct 22
High number of new and inexperienced directors There are 11 new directors who have joined the board in the last 3 years. The company's board is composed of: 11 new directors. 1 experienced director. No highly experienced directors. Independent Director David Schenkein is the most experienced director on the board, commencing their role in 2019. The following issues are considered to be risks according to the Simply Wall St Risk Model: Lack of board continuity. Lack of experienced directors.