Ra Pharmaceuticals, Inc.

→ The top leadership at Lexicon Pharmaceuticals is still undergoing a major makeover as president and COO Jeff Wade heads for the exit on Sept. 30 after 25 years with the Texas-based biotech. Wade won’t be around for the outcome of Lexicon’s latest attempt at an FDA nod in type 1 diabetes for sotagliflozin , which has a Dec. 20 decision date. The drug has already been approved for heart failure and is marketed as Inpefa . Lexicon laid off about 75 people in its sales and commercial teams in August “to right-size our promotional efforts around Inpefa for heart failure and reallocate resources to prepare for our potential launch” in type 1 diabetes and chronic kidney disease, CEO Mike Exton said in a statement. Exton succeeded Lonnel Coats as CEO a month earlier. → Sarepta Therapeutics has appointed Genmab chair Deirdre Connelly to the board of directors. Connelly had a 26-year career with Eli Lilly before pivoting to GSK in 2009 as president, North America pharmaceuticals until her retirement in 2015. She’s also a board member at Macy’s and Lincoln Financial Group . Like Janet Woodcock before him, CBER Director Peter Marks helped Sarepta cross the goal line with a label expansion in June for its Duchenne muscular dystrophy treatment Elevidys — overruling his own staff and befuddling experts. → Mitchell Chan has replaced Vit Vasista as CFO of Regenxbio at a time when the company plans to start a pivotal study for its own Duchenne gene therapy. It’s another big change for Regenxbio after Ken Mills became executive chairman and Curran Simpson succeeded Mills as CEO on July 1. Chan spent a year with Catalio Capital Management as operating partner after he was elevated to the CFO post at Viela Bio. Vasista had been finance chief since 2009 and will be an advisor until Jan. 3. → Here’s a look at some CEO news that we covered earlier this week: Former Kite Pharma commercial chief Warner Biddle has succeeded Peter Maag as CEO of Kyverna Therapeutics , which also added ex-Kite CEO Christi Shaw to the board. Shares $KYTX of Kyverna have taken a precipitous fall since its $319 million IPO in February. Ochre Bio co-founder Jack O’Meara is calling it quits after five years as CEO, but he tells Lei Lei Wu that he has an Alzheimer’s startup in the works. In a LinkedIn post announcing his departure, O’Meara also said that former F-star CEO and current Levicept chief Eliot Forster will be interim executive chair. And Shilpa Sambashivan has been promoted to CEO of Nura Bio , which stapled an additional $68 million to its Series A — bringing its total to $141 million. Sambashivan was a project team lead at Amgen before becoming a founding member at Nura Bio. → Matt Kane is taking the top spot at Celyad Oncology , a Belgian biotech that has seen considerable turnover at the top. Celyad co-founder Michel Lussier filled in as interim CEO for a second time after Georges Rawadi ’s sudden exit last December; he also took over when Filippo Petti stepped down in 2022. Kane co-founded Precision BioSciences with Derek Jantz and Jeff Smith , leading the company until he joined epigenome editing startup Tune Therapeutics as CEO in 2021. Kane’s LinkedIn page indicates that he left Tune in April, a year after he hired Jantz as CSO. → Ex- Prometheus Biosciences chief Mark McKenna has found a CFO at his new I&I bet, Mirador Therapeutics . Gregg Gilbert had been a managing director in the healthcare investment banking group for Bank of America Securities since 2021 and worked at Bank of America Merrill Lynch for 15 years. After selling Prometheus and its inflammatory bowel disease drug to Merck for $10.8 billion , McKenna wasted little time getting Mirador off the ground with a $400 million Series A in March, one of the largest private financing rounds of 2024. → GLP-1 competitor Structure Therapeutics has promoted Blai Coll to CMO and welcomed Ashley Hall as chief development officer. Coll, who replaces Mark Boch , had led clinical development for Raymond Stevens ’ crew since May 2022 and was cardiovascular and metabolic platform lead, medical affairs at the end of a six-year stint with Amgen. Hall is an Amgen vet in her own right who held the role of development chief at Reneo Pharmaceuticals . Structure’s stock price soared in June $GPCR when it produced efficacy data with its oral GLP-1 GSBR-1290 that was on par with Eli Lilly’s obesity drug orforglipron . → Novartis and Gilead veteran Matthias Will has succeeded Edgar Braendle as chief development officer at Autolus Therapeutics . Will had led clinical development at CRISPR Therapeutics and CytomX before his most recent gig as CMO of Dren Bio . The FDA is expected to make a decision by Nov. 16 on Autolus’ CD19-directed CAR-T obecabtagene autoleucel , or obe-cel . → According to an SEC filing , Crinetics chief commercial officer Jim Hassard will pack his bags on Oct. 14 in what’s described as a “termination without cause.” The 17-year Amgen vet came to Crinetics from Arrowhead Pharmaceuticals in March 2022. Phase 3 successes with its acromegaly drug paltusotine have propelled Crinetics to an NDA filing “later this year” and “an expected market launch in 2025,” CEO Scott Struthers said in the San Diego biotech’s second-quarter report. CFO Mark Wilson cited personal reasons for his own upcoming departure, which was also included in Crinetics’ Q2 update. → Allosteric drug discovery shop HotSpot Therapeutics has introduced Alison O’Neill as CMO. O’Neill is the former medical chief at Surface Oncology , which was acquired by Coherus BioSciences last year for $65 million. She had a senior leadership role in the global oncology division at Sanofi and was VP of clinical development for Radius Health . → Ex- Icosavax CEO Adam Simpson has landed at Frazier Life Sciences as a venture partner on its company creation team and will work out of Frazier’s office in San Diego. Simpson sold the vaccine developer to AstraZeneca last December, and his previous company — celiac disease specialist PvP Biologics — was purchased by Takeda in 2020. → Ruben Tommasi has been named CSO of Entact Bio , a Watertown, MA-based biotech led by ex- Ribon Therapeutics CEO Victoria Richon that is developing enhancement-targeting chimeric molecules, or ENTACs. Tommasi has also held this role at Dunad Therapeutics and AstraZeneca spinout Entasis Therapeutics . Entact Bio has kept a relatively low profile since its $81 million Series A in 2022, but it did show up in Peer Review this spring when it recruited fellow Entasis vet Michael Gutch as COO. → Third Rock ’s Treg developer Abata Therapeutics has resurfaced in Peer Review for the first time in more than a year, naming Joanne Beck as chief technical officer. The Shire and Celgene alum is the ex-technical chief at Aerium Therapeutics , the Covid biotech led by Rajeev Venkayya that recently shut down because of a surge in new variants. In April 2023, Abata poached Sonoma Biotherapeutics CMO Leonard Dragone for the same role . → Led by ex- Teva R&D chief Michael Hayden , neuro biotech Prilenia has tapped Rob Lauzen as CFO and Jason Marks as chief legal officer. Lauzen was VP, investor relations and head of financial planning & analysis at Dyne Therapeutics , which made a CMO change and lost its COO and CBO earlier this month. Marks just had a short stint with Teva as a senior legal advisor to global transactions and global finance. The Bausch Health alum has also been chief legal and compliance officer at Amarin . → San Francisco eye disease biotech Ray Therapeutics has selected Christopher Whitmore as CFO. Whitmore had held the same position with Immune-Onc Therapeutics since July 2022, and he was VP, finance & administration for Harpoon Therapeutics from 2018-21. Ray Therapeutics, which hauled in a $100 million Series A last year, will test its gene therapy RTx-015 in patients with retinitis pigmentosa. → Tris Pharma has promoted VP of marketing Manesh Naidu to the role of chief commercial officer. Naidu joined the New Jersey-based company in 2023 after a stint as chief marketing officer of Althera Pharmaceutical . Naidu’s résumé also includes stints at McKinsey , Mallinckrodt , Novartis, Pfizer and Bellerophon Therapeutics . In May, Tris snagged the first liquid non-stimulant ADHD drug approval in the US. Onyda XR will be used to treat ADHD in children at least 6 years of age. → Backed by Jeff Bezos , Seattle-based Nautilus Biotechnology has named Kentaro Suzuki as chief marketing officer. Suzuki most recently served as VP and general manager of Agilent Technologies’ mass spectrometry division. Before that, he was with Takeda and Hewlett-Packard . → San Francisco-based 89bio has appointed Teresa Perney as chief regulatory and quality officer. Perney held this title at EQRx after five years with Myovant Sciences as SVP of regulatory and quality. Madrigal Pharmaceuticals finally broke through with an FDA approval for metabolic dysfunction-associated steatohepatitis (also referred to as NASH), and 89bio is enrolling patients for late-stage studies of pegozafermin — one for non-cirrhotic MASH and another for MASH with compensated cirrhosis. “I’m eager to leverage my experience to navigate the regulatory landscape and help bring this innovative therapy to patients worldwide,” Perney said in a statement . → There’s also a new regulatory chief at Disc Medicine : Steve Caffé spent five years with CRISPR Therapeutics as head of regulatory affairs until his departure last December. Earlier, he had numerous stops as a regulatory exec with such companies as Ra Pharmaceuticals , Sucampo Pharmaceuticals , AMAG Pharmaceuticals and MedImmune . Shares of Disc $IRON fell sharply in early April after mixed data with its drug for a rare blood disorder called erythropoietic protoporphyria, but have rebounded since. → Although AI-designed drugs haven’t exactly lived up to the hype, companies like BPGbio are trying to flip the narrative. This week, GSK alum John Beeler has joined the team as SVP of business development. Beeler comes to BPGbio from Bristol Myers Squibb , where he took on the role of business development search & evaluation lead. → Macrocycle startup Circle Pharma has brought in Marie Evangelista as SVP and head of cancer biology, and promoted Constantine Kreatsoulas to SVP and head of discovery technology sciences. Evangelista spent the last two years as VP of oncology at one of the most ambitious AI drug developers , Recursion . The longtime Genentech vet also had a brief tenure with Frontier Medicines as senior director of translation medicine. Kreatsoulas had a nine-year run at GSK and was the pharma giant’s interim head, computational toxicology before coming to Circle in 2021 as head of research and VP, research informatics and computational chemistry. Circle disclosed its $54 million Series D in an SEC filing this summer. → South San Francisco-based Ideaya Biosciences has recruited Douglas Snyder as general counsel. Snyder most recently served as chief legal officer and secretary of GW Pharmaceuticals . Earlier in his career, Snyder was general counsel and secretary at Actelion and Eisai . Back in July, Ideaya inked a $406.5 million deal with Biocytogen . → Deerfield Group has rolled out the welcome mat for two new execs: Guy Varady as EVP, integrated marketing and Bob Josefsberg as EVP, communications. Before this, Varady was SVP, management supervisor for DDB Health and held a number of roles at IPG Health and Omnicom Health Group . Meanwhile, Josefsberg was VP, corporate communications at Karuna Therapeutics and managing director at Chamberlain Healthcare Public Relations . → Tempest Therapeutics , which revealed early PFS and OS data for its liver cancer combo last October, has brought on Sheldon Mullins as VP, regulatory affairs. Mullins joins the team after gigs at Arrowhead Pharmaceuticals and Ardelyx . Earlier in his career, Mullins was with Anacor Pharmaceuticals , Onyx Pharmaceuticals and Genentech. In addition to Mullins’ appointment, Darrin Bomba and Henry Johnson have been promoted to the titles of VP, development operations and VP, CMC & medicinal chemistry, respectively. → Entrada Therapeutics CEO Dipal Doshi has picked up a seat on the board of directors at āshibio , a California biotech that will push its ex-Gilead drug andecaliximab into a Phase 2/3 study for patients with an ultra-rare bone disorder called fibrodysplasia ossificans progressiva (FOP). Āshibio came out of stealth mode this summer with a $40 million raise . → Akari Therapeutics , which is developing PAS-nomacopan for geographic atrophy, has elected former Epizyme CEO Rob Bazemore to the board of directors. Bazemore is also a board member at Ardelyx and Nuvation Bio .

Dr. Steve Caffé is an accomplished industry executive with over 25 years of expertise in global product development and regulatory affairsWATERTOWN, Mass., Sept. 19, 2024 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today announced the appointment of Steve Caffé, MD as the company’s Chief Regulatory Officer. Dr. Caffé is an experienced biotech executive with significant expertise in global regulatory leadership across a wide range of therapeutic areas, including hematology, oncology, and rare diseases. “We are excited to welcome an executive as accomplished as Steve to Disc, where his depth of regulatory expertise and experience in global product development will be integral to the company’s growth,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “Steve’s track record of successful drug approvals across multiple disease areas and geographies will be a significant advantage for Disc as we move into the later stages of development across our portfolio.” “I am excited to be joining Disc at this pivotal moment in the company’s transition to a late-stage hematology company,” said Dr. Caffé. “With significant trial initiations coming in the next year for all three pipeline programs, I look forward to leading global regulatory interactions to support Disc’s mission of delivering novel treatments to patients with high unmet need. I am excited to work with the talented team at Disc and build on the excellent work done to date.” Dr. Caffé has more than 25 years of experience in global product development and regulatory affairs, having held senior leadership positions at several top biotechnology companies. Most recently, he served as Head of Regulatory Affairs at CRISPR Therapeutics where he was involved in the development and approval of Casgevy® (exagamglogene autotemcel) for sickle cell disease and beta thalassemia. Prior to joining CRISPR, Dr Caffé was the Senior Vice President leading Regulatory Affairs, Pharmacovigilance, Quality, and Patient Advocacy at Ra Pharmaceuticals. Before this, Dr. Caffé held senior-level regulatory positions at a number of other biopharmaceutical companies, including Sucampo Pharmaceuticals, AMAG Pharmaceuticals, MedImmune (Biologics Division of AstraZeneca), Baxter, Sanofi-Aventis and Merck. Across these experiences, Dr. Caffé has contributed to over 40 new drug approvals and major new indications worldwide in a wide range of therapeutic areas. Steve received his M.D. at the Université Pierre et Marie Curie in Paris, France. In connection with Dr. Caffé’s appointment, on September 16, 2024, Disc granted to Dr. Caffé an inducement equity award outside of Disc’s Amended and Restated 2021 Stock Option and Incentive Plan in accordance with Nasdaq Listing Rule 5635(c)(4), comprised of (i) an option to purchase 55,000 shares (the “Option Award”) of Disc’s common stock (“Common Stock”), at an exercise price equal to the closing price of the Common Stock on the date of grant, and (ii) a restricted stock unit award for 36,666 shares of Common Stock (the “RSU Award” and, together with the Option Award, the “Inducement Award”). The Option Award shall vest 25% on September 16, 2025, with the remainder vesting in 36 equal monthly installments thereafter. The RSU Award shall vest in equal installments on each of the first, second, third, and fourth anniversaries of the vesting date set by Disc’s company vesting policy. The Inducement Award was approved by the Compensation Committee of Disc’s Board of Directors. About Disc Medicine Disc Medicine (NASDAQ:IRON) is a clinical-stage biopharmaceutical company committed to discovering, developing, and commercializing novel treatments for patients who suffer from serious hematologic diseases. We are building a portfolio of innovative, potentially first-in-class therapeutic candidates that aim to address a wide spectrum of hematologic diseases by targeting fundamental biological pathways of red blood cell biology, specifically heme biosynthesis and iron homeostasis. For more information, please visit www.discmedicine.com. Disc Cautionary Statement Regarding Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding Disc’s expectations with respect to the chief regulatory officer position, upcoming trial initiations in the next year, and Disc’s clinical development plans and related regulatory interactions. The use of words such as, but not limited to, “believe,” “expect,” “estimate,” “project,” “intend,” “future,” “potential,” “look forward,” “continue,” “may,” “might,” “plan,” “will,” “should,” “seek,” “anticipate,” or “could” or the negative of these terms and other similar words or expressions that are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Disc’s current beliefs, expectations and assumptions regarding the future of Disc’s business, future plans and strategies, clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Disc may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and investors should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of a number of material risks and uncertainties including but not limited to: Disc’s expectations regarding the chief regulatory officer position; Disc’s expectations regarding leadership and future growth; Disc’s expectations regarding its research and development programs; Disc’s expectations of entering late-stage development; and the other risks and uncertainties described in Disc’s filings with the Securities and Exchange Commission, including in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2023, and in subsequent Quarterly Reports on Form 10-Q. Any forward-looking statement speaks only as of the date on which it was made. None of Disc, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law. Media Contact Peg RusconiDeerfield Grouppeg.rusconi@deerfieldgroup.com Investor Relations Contact Christina TartagliaPrecision AQchristina.tartaglia@precisionaq.com

Following achievements in Asia, Roche's PNH treatment drug PIASKY (crovalimab) received its first FDA approval. This approval has spiked the competition between Roche and current market leader AstraZeneca. Additionally, the other pharma companies also geared up to launch their respective lead assets. LAS VEGAS, Sept. 2, 2024 /PRNewswire/ -- Paroxysmal nocturnal hemoglobinuria (PNH), a rare disorder, shows a triad of features: intravascular hemolysis, thromboembolic events, and cytopenia. Manifestations vary among patients, making classification based on typical presentations challenging due to the disease's unpredictable nature. In 2023, the Paroxysmal Nocturnal Hemoglobinuria diagnosed prevalent cases were ~12,000 cases in the 7MM, which might reach ~13,000 cases by 2034. In the 7MM, the highest number of paroxysmal nocturnal hemoglobinuria diagnosed prevalent cases were observed in the US. Until 2007, PNH was a devastating disease without treatment for hemolysis and thrombosis, the leading cause of death in patients. However, the last decade saw a revolutionary shift with the advent of the anti-C5 agent eculizumab. It significantly reduced hemolysis, decreased transfusion dependency, and, crucially, lowered the thrombosis rate. The disease-modifying therapeutic strategy for paroxysmal nocturnal hemoglobinuria includes complement inhibition therapy, with drugs like SOLIRIS, ULTOMIRIS, and EMPAVELI approved by the FDA, and considered the gold standard. SOLIRIS, the pioneering therapy for PNH, was succeeded by ULTOMIRIS, both developed by Alexion Pharmaceuticals, offering C5 inhibitors as essential therapeutic options. Recent approvals of factor B and D inhibitors like FABHALTA (iptacopan) in the US and VOYDEYA (danicopan) in Japan aim to offer improved treatment with fewer side effects. Learn more about the FDA-approved paroxysmal nocturnal hemoglobinuria drugs @ Drugs for Paroxysmal Nocturnal Hemoglobinuria Treatment The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended crovalimab for treating PNH in adults and adolescents aged 12 and above in June 2024, based on findings from the global Phase III COMMODORE 2 clinical trial. The FDA accepted a biologics license application for crovalimab in September 2023 and subsequently approved it in June 2024 for treating PNH in adults and adolescents aged 13 years and older. Genentech, a subsidiary of Roche, has made the drug available in the US. In Japan, Chugai Pharmaceutical submitted a new drug application for PiaSky to the Japan Ministry of Health, Labour, and Welfare (MHLW) in June 2023. The drug was approved in March 2024 and launched in May 2024 for patients with PNH aged 12 years and above. In February 2024, China's National Medical Products Administration (NMPA) approved crovalimab for treating PNH in adults and adolescents aged 12 years and older who have not previously received complement inhibitor treatment, following a priority review that began in August 2022. PiaSky is also undergoing review by other regulatory authorities, including those in Europe and Taiwan. To know more about paroxysmal nocturnal hemoglobinuria treatment options, visit @ New Treatment for Paroxysmal Nocturnal Hemoglobinuria The paroxysmal nocturnal hemoglobinuria market is crowded with so many companies working in the domain. Emerging PNH therapies include Pozelimab (REGN3918) + Cemdisiran, OMS906, NM8074 (ruxoprubart), and others, reflecting a dynamic evolution in treatment strategies. Discover which therapies are expected to grab major paroxysmal nocturnal hemoglobinuria market share @ Paroxysmal Nocturnal Hemoglobinuria Market Report Pozelimab is a novel, fully human monoclonal antibody currently under investigation. It aims to inhibit complement factor C5, thereby preventing the destruction of red blood cells (hemolysis) responsible for symptoms in conditions like Paroxysmal Nocturnal Haemoglobinuria (PNH) and other diseases influenced by complement pathway activity. This IgG4 antibody binds tightly to both wild-type and variant forms of human C5, effectively blocking its function. In its ongoing development, pozelimab is also being studied in combination with Alnylam's cemdisiran, a siRNA C5 inhibitor. This investigational combination therapy is aimed at treating various complement-mediated disorders, including PNH and myasthenia gravis. OMS-906 is currently being developed to treat paroxysmal nocturnal hemoglobinuria (PNH), complement 3 glomerulopathy (C3G), idiopathic immune complex-mediated glomerulonephritis (ICGN), and arthritis. It can be administered either subcutaneously or intravenously. This drug candidate is a monoclonal antibody designed to target mannan-binding lectin serine protease 3 (MASP-3) to exert its therapeutic effects. Discover more about drugs for paroxysmal nocturnal hemoglobinuria in development @ Paroxysmal Nocturnal Hemoglobinuria Clinical Trials The anticipated launch of these emerging therapies for paroxysmal nocturnal hemoglobinuria are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the paroxysmal nocturnal hemoglobinuria market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that in 2023, the total paroxysmal nocturnal hemoglobinuria market size in the 7MM was USD ~1.4 billion, which is expected to reach USD ~2.5 billion by 2034. This growth can be attributed to the advances in disease mechanisms that drive new diagnostics and therapeutics, fueling drug development. Additionally, the PNH market foresees positive growth, propelled by increasing cases and upcoming therapies in the forecast period. DelveInsight's latest published market report titled as Paroxysmal Nocturnal Hemoglobinuria Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the paroxysmal nocturnal hemoglobinuria country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The paroxysmal nocturnal hemoglobinuria market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Diagnosed Prevalent Cases of PNH Gender-specific Cases of PNH The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM paroxysmal nocturnal hemoglobinuria market. Highlights include: 11-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this paroxysmal nocturnal hemoglobinuria market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the paroxysmal nocturnal hemoglobinuria market. Also, stay abreast of the mitigating factors to improve your market position in the paroxysmal nocturnal hemoglobinuria therapeutic space. Related Reports Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted paroxysmal nocturnal hemoglobinuria epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Paroxysmal Nocturnal Hemoglobinuria Pipeline Paroxysmal Nocturnal Hemoglobinuria Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key paroxysmal nocturnal hemoglobinuria companies, including Biocad, AKARI Therapeutics, Amgen, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, among others. Aplastic Anemia Market Aplastic Anemia Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key aplastic anemia companies, including Pfizer, BioLineRx Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, Hangzhou Zede Pharmaceutical Technology, Cellenkos, Hemogenyx Pharmaceuticals, among others. Aplastic Anemia Pipeline Aplastic Anemia Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key aplastic anemia companies, including Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP