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A Chinese biotechnology startup promising breakthrough medicine with the use of a hot new gene modulation technology has raised 32 million in a Series A financing | 2 |
The company will stop work on a very early-stage program targeting hepatitis B and seek a partner for that as well as its ex vivo CART cell therapies | 1 |
The contract does give some validation for Gritstone company's approach and is certainly a nice signal of continued government support for COVID research Miller continued. Shares in the company rose by onefourth in Thursday morning trading | 2 |
But that chapter closed quite some time ago and weve written a completely new chapter for Laronde that builds on a lot of scientific foundation that was established earlier Its merger with Senda which launched three years ago and raised 266 million in private financing before the merger creates a biotech that can draw on two Flagship platforms | 2 |
The initial round of layoffs was expected to impact about 8 of its workers and came after the study failure of an experimental drug called vatiquinone in Friedreich ataxia an inherited disorder affecting the nerves | 2 |
Its discovery has been heralded as a major scientific breakthrough that happens once every decade or so and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine | 2 |
For the entire population while we saw separation at every time point between the active and placebo cohorts Study 102 did not achieve statistical significance on the primary functional endpoint | 0 |
Transthyretin-mediated ATTR amyloidosis is a rare rapidly progressive debilitating disease caused by misfolded transthyretin TTR proteins which accumulate as amyloid fibrils in multiple tissues including the nerves heart and gastrointestinal GI tract | 0 |
Now however Eisais Biogenpartnered drug Leqembi is approved in the US and Lillys donanemab is under Food and Drug Administration review. Roche hasnt fully ended its antiamyloid drug work yet continuing tests of one called crenezumab in people who have an inherited form of Alzheimers | 0 |
The CEO called the finding too important not to pursue, but the work and funding needed to develop an Alzheimers drug is well outside of Ultragenyxs rare disease focus. So Ultragenyx is forming a new company called Amlogenyx to capitalize on the highrisk high return discovery. The spinout will be cash neutral for Ultragenyx the CEO said | 1 |
We have our immunooncology programs in house and we have a dedicated discovery team focused on inflammation and immunology and looking at how we can go through datasets pull out interesting and novel insights and translate those into meaningful discoveries and cures potentially for our customers | 1 |
The company was incubated at GV which joined in the Series B extension financing and where Kapeller is a fellow | 1 |
One other Phase 3 study is ongoing for pamrevlumab in pancreatic cancer. Initial results are expected by the end of the year according to a federal database | 1 |
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 | 2 |
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 | 2 |
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 | 2 |
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 | 2 |
In the case of a serious or lifethreatening IRR the infusion should be discontinued and not resumed | 1 |
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 | 1 |
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 | 1 |
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 | 1 |
If a hypersensitivity reaction occurs institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy | 1 |
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 | 2 |
Kidney function should be monitored in patients taking VYONDYS 53 | 1 |
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 | 0 |
Its data platform comes from a collaboration with Nashville Biosciences a subsidiary of Vanderbilt University Medical Center | 1 |
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 | 2 |
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 | 2 |
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 | 1 |
The Swiss drugmaker has fallen behind rivals Eisai Biogen and Eli Lilly which have benefited from recent clinical development successes | 0 |
The company expects to complete the study in 2025 according to a federal database of clinical trials | 1 |
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 | 1 |
While the Axiomer program remains in early stages ProQR does have a heavyweight partner in Eli Lilly for that research | 0 |
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 | 1 |
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 | 1 |
Words such as anticipates believes expects intends projects plans and future or similar expressions are intended to identify forwardlooking statements | 1 |
Neumora a richly funded brain drug developer readies for an IPO The Archbacked startup has raised more than 600 million in venture 铿乶ancing to fund testing of neurological disease therapies | 2 |
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 | 1 |
ForwardLooking Statements This press release contains forwardlooking statements Any statements that are not statements of historical fact may be deemed to be forward looking statements | 1 |
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 | 1 |
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 | 2 |
Those who would like to participate may access the live webcast here or register in advance for the teleconference here | 1 |
The decrease of 39 million was due to reduced general and administrative compensation as a result of lower headcount consulting and professional fees | 1 |
Ultimately Moderna sees the future in respiratory vaccine combinations which may be more attractive to patients than any one inoculation alone | 1 |
The disorder is characterized by intellectual disabilities loss of communication seizures slowing andor regression of development motor and respiratory impairment and shortened life expectancy | 0 |