1M+ people live with SYNGAP1-related conditions, which can include severe seizures.
For the first time in mice models, gene therapy has reversed symptoms, even after onset. The breakthrough was achieved in collaboration with BioMarin.
🧠📈 Read the study: www.cell.com/molecular-th...
For the first time in mice models, gene therapy has reversed symptoms, even after onset. The breakthrough was achieved in collaboration with BioMarin.
🧠📈 Read the study: www.cell.com/molecular-th...
October 3, 2025 at 9:24 PM
1M+ people live with SYNGAP1-related conditions, which can include severe seizures.
For the first time in mice models, gene therapy has reversed symptoms, even after onset. The breakthrough was achieved in collaboration with BioMarin.
🧠📈 Read the study: www.cell.com/molecular-th...
For the first time in mice models, gene therapy has reversed symptoms, even after onset. The breakthrough was achieved in collaboration with BioMarin.
🧠📈 Read the study: www.cell.com/molecular-th...
The real question is why a medication (developed by the US pharmaceutical company BioMarin) even costs 1 million dollars. BioMarin has been criticized in the past for the exorbitant costs of treatments.
July 14, 2025 at 4:40 AM
The real question is why a medication (developed by the US pharmaceutical company BioMarin) even costs 1 million dollars. BioMarin has been criticized in the past for the exorbitant costs of treatments.
This is why we need a land value tax.
BioMarin is sitting on whole city block in San Rafael and has put a project to develop additional office space there on indefinite pause.
www.marinij.com/2026/04/26/b...
BioMarin is sitting on whole city block in San Rafael and has put a project to develop additional office space there on indefinite pause.
www.marinij.com/2026/04/26/b...
BioMarin pauses expansion of San Rafael complex
The pharmaceutical company says it could be years before it resumes efforts to redevelop 999 Third St. with buildings for offices and labs.
www.marinij.com
April 27, 2026 at 4:49 PM
This is why we need a land value tax.
BioMarin is sitting on whole city block in San Rafael and has put a project to develop additional office space there on indefinite pause.
www.marinij.com/2026/04/26/b...
BioMarin is sitting on whole city block in San Rafael and has put a project to develop additional office space there on indefinite pause.
www.marinij.com/2026/04/26/b...
BioMarin Sees Voxzogo Nearing $1.05B as Retention Holds
BioMarin Pharmaceutical's flagship therapy Voxzogo is on track to surpass $1.0 billion in revenue for 2026, driven by a 20% surge in global…
Read more: https://embers.news/article/biomarin-sees-voxzogo-nearing-105b-as-retention-holds-77476
BioMarin Pharmaceutical's flagship therapy Voxzogo is on track to surpass $1.0 billion in revenue for 2026, driven by a 20% surge in global…
Read more: https://embers.news/article/biomarin-sees-voxzogo-nearing-105b-as-retention-holds-77476
BioMarin Sees Voxzogo Nearing $1.05B as Retention Holds
BioMarin Pharmaceutical's flagship therapy Voxzogo is on track to surpass $1.0 billion in revenue for 2026, driven by a 20% surge in global patient demand, executives said at the Citi Biopharma Summit. The projected full-year 2026 revenue of $1.0 billion to $1.05 billion highlights sustained growth
embers.news
September 27, 2026 at 8:35 PM
BioMarin Sees Voxzogo Nearing $1.05B as Retention Holds
BioMarin Pharmaceutical's flagship therapy Voxzogo is on track to surpass $1.0 billion in revenue for 2026, driven by a 20% surge in global…
Read more: https://embers.news/article/biomarin-sees-voxzogo-nearing-105b-as-retention-holds-77476
BioMarin Pharmaceutical's flagship therapy Voxzogo is on track to surpass $1.0 billion in revenue for 2026, driven by a 20% surge in global…
Read more: https://embers.news/article/biomarin-sees-voxzogo-nearing-105b-as-retention-holds-77476
Malachia Hoover’s passion for biomedical sciences fuels her perseverance, even when faced with difficulties. Currently, a postdoctoral fellow at BioMarin Pharmaceutical Inc, she hopes to inspire other minorities to pursue careers in the biomedical sciences. buff.ly/sDknjgw #MemberMonday #WomenInSTEM
March 17, 2025 at 4:01 PM
Malachia Hoover’s passion for biomedical sciences fuels her perseverance, even when faced with difficulties. Currently, a postdoctoral fellow at BioMarin Pharmaceutical Inc, she hopes to inspire other minorities to pursue careers in the biomedical sciences. buff.ly/sDknjgw #MemberMonday #WomenInSTEM
BioMarin to buy Amicus Therapeutics for $4.8 billion in rare disease bet reut.rs/4pXXD0g
BioMarin to buy Amicus Therapeutics for $4.8 billion in rare disease bet
BioMarin Pharmaceutical said on Friday it would acquire Amicus Therapeutics for about $4.8 billion, in the drugmaker's second deal this year to expand its presence in rare diseases.
reut.rs
December 19, 2025 at 7:10 PM
BioMarin to buy Amicus Therapeutics for $4.8 billion in rare disease bet reut.rs/4pXXD0g
New data shows a drug called VOXZOGO significantly increased growth in kids with hypochondroplasia (a genetic disorder affecting bone growth). This could be huge for kids with this condition!
BioMarin Announces Positive Phase 3 Pivotal Study Results for VOXZOGO® (vosoritide) in Children with
BioMarin Pharmaceutical Inc. announced positive results from its Phase 3 CANOPY-HCH-3 study evaluating VOXZOGO (vosoritide) in children with hypochondroplasia. The study met its primary goal by demons
www.biomarin.com
May 21, 2026 at 8:04 AM
New data shows a drug called VOXZOGO significantly increased growth in kids with hypochondroplasia (a genetic disorder affecting bone growth). This could be huge for kids with this condition!
BioMarin to acquire Alesta Therapeutics for $275 million for potential rare bone disease treatment
BioMarin to acquire Alesta Therapeutics for $275 million for potential rare bone disease treatment
BioMarin will acquire Alesta Therapeutics for $275 million upfront, bolstering BioMarin's unit focused on rare skeletal conditions.
cnb.cx
August 19, 2026 at 3:24 PM
BioMarin to acquire Alesta Therapeutics for $275 million for potential rare bone disease treatment
BioMarin Raises VOXZOGO Sales Outlook to $1 Billion Midpoint
BioMarin Pharmaceutical is expanding its rare-disease portfolio as VOXZOGO revenue growth accelerates. This performance led the c…
Read more: https://embers.news/article/biomarin-raises-voxzogo-sales-outlook-to-1-billion-midpoint-67196
BioMarin Pharmaceutical is expanding its rare-disease portfolio as VOXZOGO revenue growth accelerates. This performance led the c…
Read more: https://embers.news/article/biomarin-raises-voxzogo-sales-outlook-to-1-billion-midpoint-67196
BioMarin Raises VOXZOGO Sales Outlook to $1 Billion Midpoint
BioMarin Pharmaceutical is expanding its rare-disease portfolio as VOXZOGO revenue growth accelerates. This performance led the company to raise its full-year sales guidance for the treatment to a $1 billion midpoint. The results reflect expansion within the firm's metabolic conditions segment, whic
embers.news
September 22, 2026 at 10:31 AM
BioMarin Raises VOXZOGO Sales Outlook to $1 Billion Midpoint
BioMarin Pharmaceutical is expanding its rare-disease portfolio as VOXZOGO revenue growth accelerates. This performance led the c…
Read more: https://embers.news/article/biomarin-raises-voxzogo-sales-outlook-to-1-billion-midpoint-67196
BioMarin Pharmaceutical is expanding its rare-disease portfolio as VOXZOGO revenue growth accelerates. This performance led the c…
Read more: https://embers.news/article/biomarin-raises-voxzogo-sales-outlook-to-1-billion-midpoint-67196
Despite turning a profit and having secured multiple drugs in over a quarter of a century, BioMarin is getting revamped with the help of ex-Roche dealmaker.
BioMarin turns to ex-Roche dealmaker James Sabry to build ‘next version’ of 27-year-old company
SAN FRANCISCO — Not many biotechs survive beyond 25 years, let alone get multiple drugs approved and turn a profit. BioMarin Pharmaceutical has done all that, but finds itself navigating ...
endpts.com
January 23, 2025 at 8:51 PM
Despite turning a profit and having secured multiple drugs in over a quarter of a century, BioMarin is getting revamped with the help of ex-Roche dealmaker.
BioMarin vets lead new biotech with $82M for rare disease drugs.
endpoints.news/biomarin-vet...
endpoints.news/biomarin-vet...
BioMarin vets spearhead new biotech with $82M for rare disease drugs
Mendra launches with $82M Series A to acquire and develop rare disease drugs, led by CEO Joshua Grass with backing from OrbiMed, 8VC, and 5AM Ventures.
endpoints.news
January 22, 2026 at 4:52 PM
BioMarin vets lead new biotech with $82M for rare disease drugs.
endpoints.news/biomarin-vet...
endpoints.news/biomarin-vet...
“Great companies go through versions,” Sabry said. “They go through building their fundamental business in one direction. And then over time, these things change. That happened at Genentech. It’s happened to Roche, and it will happen to BioMarin as well.”
endpts.com/biomarins-ja...
endpts.com/biomarins-ja...
BioMarin turns to ex-Roche dealmaker James Sabry to build ‘next version’ of 27-year-old company
SAN FRANCISCO — Not many biotechs survive beyond 25 years, let alone get multiple drugs approved and turn a profit. BioMarin Pharmaceutical has done all that, but finds itself navigating ...
endpts.com
January 23, 2025 at 3:24 PM
“Great companies go through versions,” Sabry said. “They go through building their fundamental business in one direction. And then over time, these things change. That happened at Genentech. It’s happened to Roche, and it will happen to BioMarin as well.”
endpts.com/biomarins-ja...
endpts.com/biomarins-ja...
"Though BioMarin is a global organization... the U.S. remains a vital source of innovative research that pharma companies ultimately turn into medicines. We're hoping that pipeline of good ideas, which often comes from NIH-funded research, doesn't dry out.” Thank you for your courage BioMarin.
As BioMarin searches for 'clever science,' its R&D chief warns innovation could be hit by NIH fund drought
A slimmed-down BioMarin started the new year with a bang, hitting the annual J.P. | A slimmed-down BioMarin started the new year open for business, seeking to leverage its unique skillset to strike de...
www.fiercebiotech.com
February 24, 2025 at 3:10 PM
"Though BioMarin is a global organization... the U.S. remains a vital source of innovative research that pharma companies ultimately turn into medicines. We're hoping that pipeline of good ideas, which often comes from NIH-funded research, doesn't dry out.” Thank you for your courage BioMarin.
BioMarin has announced a new €60m investment to expand its Shanbally, Co Cork facility
BioMarin announces €60m Cork expansion
The expansion will result in a new four-story laboratory facility enabling the company to increase its production capacity.
www.siliconrepublic.com
December 9, 2024 at 8:23 AM
BioMarin has announced a new €60m investment to expand its Shanbally, Co Cork facility
Wow. That’s really heartbreaking.
Failing to find a buyer, BioMarin pulls Roctavian in gene therapy's biggest disappointment
Failing to find a buyer, BioMarin pulls Roctavian in gene therapy's biggest disappointment
Failing to find a buyer, BioMarin pulls Roctavian in gene therapy's biggest disappointment
For all its blockbuster hopes and high-profile status in the gene therapy field, Roctavian's commercial saga has ended.
buff.ly
February 25, 2026 at 12:50 PM
Wow. That’s really heartbreaking.
Failing to find a buyer, BioMarin pulls Roctavian in gene therapy's biggest disappointment
Failing to find a buyer, BioMarin pulls Roctavian in gene therapy's biggest disappointment
@Reuters: BioMarin Pharma gets DOJ subpoena on sponsored testing programs for two therapies https://t.co/UeCQWFZmbw https://t.co/SlwPQHGOBu
February 26, 2024 at 11:07 PM
@Reuters: BioMarin Pharma gets DOJ subpoena on sponsored testing programs for two therapies https://t.co/UeCQWFZmbw https://t.co/SlwPQHGOBu
#apaperaday Back to back papers were published in Nucleic Acid Therapeutics, the journal of the oligonucleotide therapeutic society, on preclinical research done to optimize and test BMN-351, the BioMarin exon 51 skipper currently tested in clinical trials. DOI: 10.1089/nat.2024.0049
February 13, 2025 at 10:36 AM
#apaperaday Back to back papers were published in Nucleic Acid Therapeutics, the journal of the oligonucleotide therapeutic society, on preclinical research done to optimize and test BMN-351, the BioMarin exon 51 skipper currently tested in clinical trials. DOI: 10.1089/nat.2024.0049
Next: The Rare Disease AI Inflection Point, with
Arpit Davé at BioMarin.
Moderated by Rick Berke.
AD: BioMarin focuses on rare diseases. Our North Star is to bring therapies to patients who need it the most. AI can help with this.
#STATBreakthrough
Arpit Davé at BioMarin.
Moderated by Rick Berke.
AD: BioMarin focuses on rare diseases. Our North Star is to bring therapies to patients who need it the most. AI can help with this.
#STATBreakthrough
May 19, 2026 at 11:56 PM
Next: The Rare Disease AI Inflection Point, with
Arpit Davé at BioMarin.
Moderated by Rick Berke.
AD: BioMarin focuses on rare diseases. Our North Star is to bring therapies to patients who need it the most. AI can help with this.
#STATBreakthrough
Arpit Davé at BioMarin.
Moderated by Rick Berke.
AD: BioMarin focuses on rare diseases. Our North Star is to bring therapies to patients who need it the most. AI can help with this.
#STATBreakthrough
BioMarin's EVP & Chief Business Officer James Sabry spoke w/ @endpts.com @andrewedunn.bsky.social about their BD focus ranging from preclinical to late-stage clinical programs & their priorities: enzyme therapies, skeletal conditions & genetically defined diseases. endpts.com/biomarins-ja... #biosky
BioMarin turns to ex-Roche dealmaker James Sabry to build ‘next version’ of 27-year-old company
SAN FRANCISCO — Not many biotechs survive beyond 25 years, let alone get multiple drugs approved and turn a profit. BioMarin Pharmaceutical has done all that, but finds itself navigating ...
endpts.com
January 24, 2025 at 7:23 PM
BioMarin's EVP & Chief Business Officer James Sabry spoke w/ @endpts.com @andrewedunn.bsky.social about their BD focus ranging from preclinical to late-stage clinical programs & their priorities: enzyme therapies, skeletal conditions & genetically defined diseases. endpts.com/biomarins-ja... #biosky
Thank you to Biomarin bsky.app/profile/biom... for their bronze sponsorship of #WMS2025. We look forward to seeing you in Vienna for our 30th anniversary Congress.
September 26, 2025 at 9:55 AM
Thank you to Biomarin bsky.app/profile/biom... for their bronze sponsorship of #WMS2025. We look forward to seeing you in Vienna for our 30th anniversary Congress.
US company BioMarin to invest €60m in Cork facility
US company BioMarin to invest €60m in Cork facility
Picture: IDA Ireland
www.businesspost.ie
December 9, 2024 at 8:37 AM
US company BioMarin to invest €60m in Cork facility
Are you a biopharma C-suite exec or investor dissecting rare disease playbooks?
Then this one's for you.
www.linkedin.com/posts/mattkr...
Then this one's for you.
www.linkedin.com/posts/mattkr...
#raredisease #biotechstrategy #biomarin | Matt Krebs
Are you a biopharma C-suite exec or investor dissecting rare disease playbooks?
Then this one's for you.
BioMarin Pharmaceutical Inc. just made its 1st acquisition in 10+ yrs.
The $270M price ...
www.linkedin.com
May 19, 2025 at 2:07 PM
Are you a biopharma C-suite exec or investor dissecting rare disease playbooks?
Then this one's for you.
www.linkedin.com/posts/mattkr...
Then this one's for you.
www.linkedin.com/posts/mattkr...