daniel-gordon/biopharma-dive huggingface dataset:
Inhibiting PRC2 has been associated with a risk of developing blood cancer, an issue US regulators flagged when stopping testing of FTX6058 in February. The FDA asked Fulcrum to narrow testing to a group where the potential benefits outweighed the risks leading to the study design the company will carry forward
In the second quarter our net product revenue stood at 1418 million a 27 increase over the same quarter last year stated Doug Ingram Sareptas president and chief executive officer
102023 715 PM US Food and Drug Administration Approves BioMarins ROCTAVIAN valoctocogene roxaparvovecrvox the First and Only Gene Thera httpsinvestorsbiomarincom20230629USFoodandDrugAdministrationApprovesBioMarinsROCTAVIANTMvaloctocogeneroxaparvovecrvoxtheF 111Clinical Trials Contact Us Careers US Food and Drug Administration Approves BioMarins ROCTAVIAN valoctocogene roxaparvovecrvox the First and Only Gene Therapy for Adults with Severe Hemophilia A       Jun 29 2023 ROCTAVIAN is a OneTime SingleDose Treatment Available for Adults with Severe Hemophilia A to Control Bleeds ROCTAVIANs Approval was Based on Durability Efficacy and Safety Results from the Largest and Longest Phase 3 Study for a Gene Therapy for Hemophilia Majority of Patients Continued to Respond to ROCTAVIAN Treatment over 3 Years Conference Call and Webcast to be Held at 430 pm Eastern Time on Thursday June 29 2023 SAN RAFAEL Calif June 29 2023 PRNewswire  BioMarin Pharmaceutical Inc Nasdaq BMRN a global biotechnology company dedicated to transforming lives through genetic discovery today announced that the United States Food and Drug Administration FDA approved ROCTAVIAN valoctocogene roxaparvovecrvox gene therapy for the treatment of adults with severe hemophilia A congenital factor VIII FVIII deficiency with FVIII activity upper levels of normal ULN
The NurixSeagen deal will have Nurix develop protein degraders against a number of targets set by Seagen that could be conjugated to a Seagen antibody
In the case of a serious or lifethreatening IRR the infusion should be discontinued and not resumed
2Seventy shares equally with Bristol Myers in the profit and loss of Abecma in the US The macro environment for oncology cell therapy companies and the nearterm headwinds we have seen in our own business have led us to examine how we pursue our mission said Leschly in 2Seventys statement
But Histogen would eventually enter a reverse merger with Conatus Pharmaceuticals which had developed emricasan and was reeling from the failure of a medicine for nonalcoholic steatohepatitis or NASH
Its not just the availability of options for patients it provides much more specificity to their symptoms and some of the aspects of their own disease progression Rampart is also developing an unspecified number of other programs
While linear strands of RNA often dont last long the round shape of eRNA in theory could prevent cellular enzymes from degrading the blueprints leading to longer protein expression
Kidney function should be monitored in patients taking VYONDYS 53
Beam Therapeutics the base editing biotechnology company on Thursday said it will lay off 100 employees pause some research and look for partners on a few of its drug development programs
Its data platform comes from a collaboration with Nashville Biosciences a subsidiary of Vanderbilt University Medical Center
The DanaFarber Cancer Institute spinout has raised 57 million from investors including 5AM Ventures Binney Street Capital Lilly Asia Ventures Illumina Ventures and Bristol Myers Squibb
Pharmaceutical firms had cast brain drugs aside  opting instead to devote resources to less risky often more lucrative areas of research like oncology and immunology. But recent scientific and technological breakthroughs along with the approvals of new medicines for Alzheimers and ALS have helped renew investor interest in neuroscience
FibroGens rebound plans leaned heavily on the companys only other medicine in advanced testing pamrevlumab which is in Phase 3 studies for a variety of different diseases from Duchenne to idiopathic pulmonary fibrosis and cancer
The Swiss drugmaker has fallen behind rivals Eisai Biogen and Eli Lilly which have benefited from recent clinical development successes
The company expects to complete the study in 2025 according to a federal database of clinical trials
The company has been an active dealmaker in both fields over the past several years striking modestsized alliances as well as spending 21 billion to acquire Immunomedics
While the Axiomer program remains in early stages ProQR does have a heavyweight partner in Eli Lilly for that research
Amortization expense primarily associated with inlicensed rights as well as patent costs are amortized over a period of several years after acquisition or patent application or renewal and generally cannot be changed or influenced by management
We have 100 people doing hypothesis early research to filing an IND and actually in human studies with a Phase 2a program 23andMes pipeline includes two clinical programs both in immunooncology
Neumora a richly funded brain drug developer readies for an IPO The Archbacked startup has raised more than 600 million in venture ﬁnancing to fund testing of neurological disease therapies
Near the end of the last decade Novartis had two similar drugs advancing through clinical development. Known as PI3 kinase inhibitors because of the enzyme they target the drugs steadily moved through human testing
PPMO is Sareptas nextgeneration chemistry platform designed around a proprietary cellpenetrating peptide conjugated to the PMO backbone with the goal of increasing tissue penetration increasing exon skipping and significantly increasing dystrophin production
There are many groups that have been working for specific rare diseases on genetic therapies that potentially would be a oneand patients this provides more flexibility for doctors
The decrease of 39 million was due to reduced general and administrative compensation as a result of lower headcount consulting and professional fees
Ultimately Moderna sees the future in respiratory vaccine combinations which may be more attractive to patients than any one inoculation alone