| Date | Title | Description |
| 19.08.2026 | BioMarin to acquire Alesta Therapeutics for $275 million for potential rare bone disease treatment | BioMarin will acquire Alesta Therapeutics for $275 million upfront, and up to an additional $215 million based on milestones.
Alesta Therapeutic’s lead drug is a potential treatment for hypophosphatasia (HPP).
The deal bolsters BioMarin’s u... |
| 16.06.2026 | DIMERIX EXCLUSIVELY LICENSES DMX-200 IN GREATER CHINA, SOUTH KOREA AND SOUTHEAST ASIA TO EVEREST MEDICINES | Everest Medicines licenses exclusive rights to commercialise DMX-200 for the treatment of Focal Segmental Glomerulosclerosis (FSGS) in Greater China, South Korea and Southeast Asia
500,000-1 million people are estimated to be living with FS... |
| 07.05.2026 | Rare disease families find roadmap to drug development at bootcamps | Rare disease families are increasingly driving research efforts to find treatments for their children.
Ultragenyx’s Rare Bootcamp gives advocates a roadmap for navigating drug development.
The program has helped connect families with resear... |
| 17.04.2026 | World Hemophilia Day 2026 - April 17, 2026 - "Diagnosis: First step to care." | MONTREAL, April 17, 2026 /PRNewswire/ -- On April 17, 2026, the global inherited bleeding disorders community will come together on World Hemophilia Day to advocate for all inherited bleeding disorders. This year's theme of "Diagnosis:... |
| 08.04.2026 | Swiss startups appoint new CEOs and senior hires across biotech, AI and proptech |
Anaveon, a pre-clinical biotechnology company developing immune-modulating therapies for autoimmune and inflammatory disorders, has appointed Thaminda Ramanayake as Chief Executive Officer. Ramanayake succeeds Andreas Katopodis, who led th... |
| 25.01.2026 | Mendra Revolutionizes Rare Disease Therapy with $82M AI-Driven Launch | Mendra, a San Francisco biopharmaceutical firm, launched with an oversubscribed $82 million Series A funding round. This capital infusion propels its mission. Mendra focuses on rare disease therapies. The company leverages advanced AI. It t... |
| 23.01.2026 | Mendra Launched With $82 Million Series A To Build AI-Enabled Rare Disease Therapeutics Portfolio | Mendra, a newly launched biopharmaceutical company focused on rare diseases, has closed an oversubscribed $82 million Series A to acquire, develop, and commercialize therapies targeting high-unmet-need indications. The financing was co-led ... |
| 16.01.2026 | Making U.S. biotech more competitive with China’s could help rare disease patients, experts say | To share your story, or find out how to get involved with CNBC Cures, email CNBC.cures@cnbc.com
China is pumping money into its biotech industry, helping launch a new wave of labs and incubators in the country.
Experts say this could slow d... |
| 11.06.2025 |
Cramer’s Lightning Round: Union Pacific is a buy | Monday - Friday, 6:00 - 7:00 PM ET
It’s that time again! “Mad Money” host Jim Cramer rings the lightning round bell, which means he’s giving his answers to callers’ stock questions at rapid speed.
BioMarin Pharmaceutical: “I’m getting tired... |
| 17.04.2025 | World Hemophilia Day 2025 - April 17, 2025 - "Access for all: Women and girls bleed too" | MONTREAL, April 17, 2025 /PRNewswire/ -- On April 17, 2025, the global bleeding disorders community will come together under the theme "Access for all: Women and girls bleed too" and celebrate World Hemophilia Day. Today, women an... |
| 10.12.2024 | Resilience and Growth: The Pulse of Ireland's SMEs and BioPharma Sector | In the heart of Ireland, small and medium-sized enterprises (SMEs) pulse with confidence. A recent survey by ACCA reveals that 79% of these businesses feel optimistic about the future. This is not just a flicker of hope; it’s a beacon of re... |
| 09.12.2024 | SME confidence high despite concerns about costs - ACCA | Eight in 10 (79%) small and medium-sized enterprises are either as confident or more confident about the future compared with this time last year.
A survey by professional accountancy body ACCA, that sought the views of SMEs on their econom... |
| 09.12.2024 | BioMarin Pharmaceutical Inc announce €60m investment in Cork plant | BioMarin Pharmaceutical Inc. has announced a €60m investment in the expansion of its state-of-the-art facility in Shanbally, Co. Cork.
The funding is being used to build a four-story laboratory facility that will enable the company to incre... |
| 07.06.2024 | KKR, CrowdStrike Holdings and GoDaddy Set to Join S&P 500; Others to Join S&P MidCap 400 and S&P SmallCap 600 | NEW YORK, June 7, 2024 /PRNewswire/ -- S&P Dow Jones Indices ("S&P DJI") will make the following changes to the S&P 500, S&P MidCap 400, and S&P SmallCap 600 indices effective prior to the open of trading on Mo... |
| 17.04.2024 | World Hemophilia Day 2024"Equitable access for all: recognizing all bleeding disorders" | MONTREAL, April 17, 2024 /PRNewswire/ -- On April 17, 2024, the global bleeding disorders community will come together to celebrate World Hemophilia Day. The theme of the event this year is "Equitable access for all: recognizing all bl... |
| 09.02.2022 | BioMarin lands $110m sale of priority review voucher; Hybridize licenses out BK virus therapy | BioMarin announced that it has sold its priority review voucher for $110 million, but it hasn’t announced who the buyer is just yet.
The California biotech won the voucher in November when it gained approval for Voxzogo, a prescription medi... |
| 09.01.2022 | BioMarin Announces Stable and Durable Annualized Bleed Control in the Largest Phase 3 Gene Therapy Study in Adults with Severe Hemophilia A; 134-Participant Study Met All Primary and Secondary Efficac... | SAN RAFAEL, Calif., Jan. 9, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced positive results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for ... |
| 28.07.2021 | BioMarin Announces Second Quarter 2021 Financial Results and Corporate Updates | |
| 21.07.2021 | BioMarin Announces Oral Presentation at International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress with 5 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Rox... | |
| 19.07.2021 | BioMarin Announces Oral Presentation of Positive One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A at International Society on... | |
| 02.07.2021 | BioMarin Announces 12 Presentations at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress | |
| 25.06.2021 | BioMarin Pharmaceutical : Receives Positive CHMP Opinion in Europe for Vosoritide for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close | SAN RAFAEL, Calif., June 25, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization... |
| 25.06.2021 | BioMarin Receives Positive CHMP Opinion in Europe for Vosoritide for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close | |
| 12.05.2021 | BioMarin Announces 6 Presentations at American Society of Gene and Cell Therapy (ASGCT) Virtual 2021 Annual Meeting | |
| 29.04.2021 | BioMarin Announces First Quarter 2021 Financial Results and Corporate Updates | |
| 29.04.2021 | Is the Rally Ready to Begin for BioMarin Pharmaceutical? | April 29, 2021 4 min read This story originally appeared on MarketBeat
Shares of BioMarin Pharmaceutical (NASDAQ:BMRN) are trading at light volume as the company prepares to deliver its first quarter 2021 earnings report on April 29, 2021. ... |
| 15.04.2021 | BioMarin Announces New and Updated Data at 2021 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting Demonstrating Commitment to Understanding Achondroplasia and P... | |
| 20.03.2021 | BioMarin Announces Oral Presentation at ENDO2021, the Endocrine Society's Annual Meeting, with Data Demonstrating 2 Years of Treatment Benefit in Children with Achondroplasia Treated with Vosoritide | |
| 20.03.2021 | BIOMARIN PHARMACEUTICAL INC.
BioMarin Pharmaceutical : Announces Oral Presentation at ENDO2021, the Endocrine Society's Annual Meeting, with Data Demonstrating 2 Years of Treatment Benefit in Childre... | SAN RAFAEL, Calif., March 20, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that data from the open-label long-term extension of the Phase 3 study of 15 µg/kg dose of vosoritide was presented at an oral pr... |
| 20.03.2021 | BioMarin Pharmaceutical : Announces Oral Presentation at ENDO2021, the Endocrine Society's Annual Meeting, with Data Demonstrating 2 Years of Treatment Benefit in Children with Achondroplasia Treated ... | SAN RAFAEL, Calif., March 20, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that data from the open-label long-term extension of the Phase 3 study of 15 µg/kg dose of vosoritide was presented at an oral pr... |
| 08.03.2021 | BioMarin Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to Valoctocogene Roxaparvovec, Investigational Gene Therapy for Hemophilia A | |
| 03.03.2021 | BioMarin Completes Full Enrollment in Phase 2 Study of Vosoritide for Treatment of Infants and Young Children with Achondroplasia | |
| 25.02.2021 | BioMarin Announces Fourth Quarter and Record Full-year 2020 Financial Results and Corporate Updates | |
| 10.01.2021 | BioMarin Announces Positive Phase 3 Gene Therapy Trial Results in Adults with Severe Hemophilia A; Study Met All Primary and Secondary Efficacy Endpoints in One-Year Data Set | |
| 21.12.2020 | BioMarin Announces Benefit Maintained for Over Two Years in Children with Achondroplasia Treated with Vosoritide in Phase 3 Extension Study | |
| 05.11.2020 | BioMarin Announces Third Quarter 2020 Total Revenues of $477 Million | |
| 02.11.2020 | Food and Drug Administration Accepts BioMarin's New Drug Application for Vosoritide to Treat Children with Achondroplasia | |
| 07.10.2020 | BioMarin, Pioneer in Rare Disease Treatments for Phenylketonuria (PKU), Receives FDA Approval of Label Expansion to Allow Maximum Dose of 60 mg for Palynziq® (pegvaliase-pqpz) Injection for Treatment ... | |
| 05.10.2020 | Scientific Luminary and MacArthur Fellowship Winner, Kevin Eggan, Ph.D., Joins BioMarin as Head of Research and Early Development | |
| 02.10.2020 | BioMarin, Pioneer in Phenylketonuria (PKU) and Gene Therapy, Receives FDA Fast Track Designation for PKU Investigational Gene Therapy, BMN 307 | |
| 08.09.2020 | BioMarin Announces The Lancet Publishes Detailed Vosoritide Phase 3 Data Demonstrating Statistically Significant Increase in Annualized Growth Velocity (AGV) Over 52 Weeks in Children with Achondropla... | |
| 19.08.2020 | BioMarin Receives Complete Response Letter (CRL) from FDA for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A | |
| 06.05.2020 | DiNAQOR and BioMarin to develop gene theapies for rare heart diseases | DiNAQOR was founded in 2019 by several leading pharmaceutical and biotechnology executives academics with deep cardiology and gene therapy expertise. The company is focused on developing gene therapies for the heart, the company's lead prec... |
| 06.05.2020 | DiNAQOR and BioMarin to develop gene theapies for rare heart diseases | |
| 28.05.2019 | BioMarin to take Phase III hemophilia gene therapy data to regulators, announce filing timeline later this year | Both studies showed significant increases in Factor VIII activity, while median annual bleeding episodes and number of Factor VIII infusions were greatly reduced, to as low as zero. In GENEr8-1, eight patients in a 20-patient cohort achieve... |
| 13.10.2016 | The compassionate use poll: A lopsided vote favors posting policies, with plenty of fretting | We ran a snap poll yesterday on compassionate use policies, and there’s clearly plenty of positive sentiment among our readers backing some sort of easily accessible, online statement explaining each biotech’s polici... |
| 25.08.2015 | Morning Read: Hatteras Venture Partners raises $90M for fifth fund, BioMarin in $570M breast cancer drug deal | PAYERS-PROVIDERS
A series of pilot programs in Ventura and Los Angeles counties are using some paramedics for non emergency, preventive care for areas like congestive heart failure and tuberculosis. The hope is that preventive care will sav... |
| - | BioMarin to take Phase III hemophilia gene therapy data to regulators, announce filing timeline later this year | A company developing a gene therapy for hemophilia plans to meet with US and European regulators to discuss potential filings for approval later this year after releasing interim Phase III data.
San Rafael, California-based BioMarin Pharmac... |
| - | A history of the advancements for PKU after almost two decades of research | For twelve-year-old Kellen Brown, preparing for a sleepover is far more challenging than for most boys his age. He must prepare several servings of his special dietary formula ahead of time, along with small portions of low-protein snacks.
... |
| - | Morning Read: Hatteras Venture Partners raises $90M for fifth fund, BioMarin in $570M breast cancer drug deal | TOP STORIES
Hatteras Venture Partners has raised $90 million as part of its first close for its fifth venture fund. According to its website the company hopes to raise $150 million for the fund — which would be its largest to date. It will ... |
