sentence
stringlengths 16
4.72k
| label
int64 0
2
|
|---|---|
Samsara raised 37 million prior to the Series A round announced Tuesday | 2 |
When developing new medicines drugmakers typically try to make treatments that are more effective durable or safer than whats already available. Rampart Bioscience a new biotechnology company eyeing a way to make better DNAbased medicines is striving to hit all three | 1 |
Genentech takes up a deal to make ʻmolecular glueʼ drugs Through a collaboration with Orionis Biosciences the Roche subsidiary is eyeing targeted protein degradation medicines for cancer and neurodegenerative conditions | 1 |
Biologic drugs like Humira Stelara and Entryvio can be used to treat both ulcerative colitis and Crohns while pills like Xeljanz and Rinvoq are cleared for the former condition. Shares in Teva fell by 2 in Wednesday morning trading | 0 |
Beam Lilly will now own optin rights to jointly develop and commercialize with Verve gene editing medicines for heart disease that are aimed at several wellknown drug targets. The new deal brings the companies closer and allows Lilly to closely track Verves progress on programs aimed at other genes important to heart disease risk | 2 |
The safety profile of SRP9001 observed in the first 11 participants is consistent with the safety seen in earlier studies using clinical manufacturing process material | 1 |
For more information about our people science and pipeline please visit wwwalnylamcom httpsctsbusinesswirecomctCT | 1 |
Rapport which was built around a discovery platform from Johnson Johnsons research unit officially launched early this year with backing from JJs strategic venture capital arm as well as the investment firms Third Rock Ventures and Arch Venture Partners | 1 |
In line with previously reported clinical data no clinical complement activation was observed | 1 |
The pharmaceutical company also revealed plans to split off its consumer health business next year a move similar to divestments done by other large drugmakers | 1 |
In the United States Ascendis submitted its NDA for TransCon PTH in hypoparathyroidism in August 2022 in October 2022 the FDA accepted the NDA for Priority Review and set a PDUFA action date of April 30 2023 | 1 |
Internet Posting of Information We routinely post information that may be important to investors in the For Investors section of our website at wwwsareptacom | 1 |
We are confident in valoctocogene roxaparvovec gene therapy and its potential to redefine the treatment paradigm for people with hemophilia A | 2 |
The company has decided to delay its SG299 therapy for patients with blood cancer. As of June 30 Sana had a cash position of 3259 million, down from 434 million at the end of December. With the layoffs announced Tuesday the company expects its cash burn rate to be below 200 million next year. Sana is certainly not alone in shedding staff amid a downturn in biotech financing | 2 |
The multiyear deal also offers 2 billion worth of socalled biobucks or future payments contingent on hitting certain development and commercialization milestones | 1 |
The deal involves three separate multiyear collaborations between the companies to discover and develop biologic therapies for cancer and inflammatory diseases using Tentarixs drugmaking technology. Gilead will also hold options to acquire up to three Tentarix subsidiaries built around each of the collaborations | 2 |
A replay of the webcast will be available on this section of our website shortly after conclusion of the event for 30 days | 1 |
Actual results could materially differ from those stated or implied by these forwardlooking statements as a result of such risks and uncertainties | 1 |
The cuts announced Thursday include the closure of a lab in Lexington Massachusetts and discontinuation of an Parkinsons drug candidate among other unnamed programs | 2 |
The biotech provided Beam Lilly with an early look at those results as part of the pharma companys due diligence for the deal Verve, the CEO said | 1 |
The severe form of hypophosphatasia affects an estimated 1 in 100000 babies but milder cases likely occur more frequently and later in life. Especially for those who have diseases where it impacts their quality of life helping them fortify their own bodies is a great step | 2 |
The Waltham Massachusetts startup formed as a merger between two companies Amagma Therapeutics and Modify Therapeutics, and is backed by a syndicate of blue chip investors coled by Atlas Venture and Cormorant Asset Management | 1 |
Sanofi buys into Teva gut disease drug The French drugmaker will pay 500 million to collaborate with Teva on an antiTL1A therapy for ulcerative colitis and Crohnʼs disease | 2 |
Rapport Therapeutics a young biotechnology company developing precision drugs for brain and nervous system disorders has raised another 150 million from a collection of existing and new investors | 1 |
Data from a Phase 1 trial were released this June and showed positive signs with regard to safety and tolerability | 1 |
Settlement and license charges In February 2021 the Company recognized a 100 million settlement charge related to contingent settlement payments to BioMarin Pharmaceutical Inc BioMarin as a result of the approval of AMONDYS 45 in the United States | 1 |
Cash and cash equivalents As of June 30 2023 Taysha had 451 million in cash and cash equivalents | 1 |
The nonGAAP loss is defined by the Company as GAAP net loss excluding interest expense net income tax benefit expense depreciation and amortization expense stockbased compensation expense and other items | 1 |
The program will also advance a program for the liver and lung condition alpha1 antitrypsin deficiency and conduct an initial clinical trial at certain US sites for a treatment for glycogen storage disease 1a | 1 |
In it the CEO sees a promise to develop a drug that resembles a traditional biologic that would appear more familiar to patients | 1 |
Over the past few years Novo has broken out of character to ink a string of acquisitions including for biotechnology companies Dicerna Pharmaceuticals and Forma Therapeutics that flesh out its drug pipeline in other metabolic and rare blood conditions | 2 |
In these studies TransCon PTH has been generally well tolerated with no discontinuations related to study drug | 1 |
Novo Nordisk has built a reputation on developing drugs for diabetes and more recently obesity | 1 |
Verge Genomics a biotechnology company using artificial intelligence to develop therapies for diseases of the nervous system announced on Friday a four-year partnership with AstraZenecas rare disease unit Alexion to identify new drug targets. As part of the agreement Verge will receive up to 42 million in upfront equity and nearterm payments | 2 |
Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency eg | 1 |
A potential new way to attack targets once thought undruggable molecular glues have become the focus of a number of collaborations between smaller biotechnology companies and larger pharmaceutical firms including Sanofi and Astellas | 1 |
Molecular glues, so named for their ability to forcibly pull together two proteins that wouldn't otherwise interact with each other have come a long way since they were discovered around three decades ago | 2 |
Obesity affects a large swathe of the US adult population and the many health issues particularly the metabolic disorders that priority | 2 |
Study 102 uses clinical process SRP9001 and has two primary endpoints microdystrophin expression at 12 weeks and change in NSAA total score at 48 weeks compared to placebo | 1 |
If a persistent increase in serum cystatin C or proteinuria is detected refer to a pediatric nephrologist for further evaluation | 1 |
While lowering its forecast for the year BioMarin said reimbursement issues for Roctavian are looking up in the US and other parts of the world | 1 |
PTC Therapeutics has agreed to sell a big chunk of its remaining royalty stream from the spinal muscular atrophy drug Evrysdi to Royalty Pharma for 1 billion | 1 |
Apellis won't start any new development on a systemically administered version of pegcetacoplan the medicine it sells as Syfovre for geographic atrophy and Empaveli for a rare blood disorder | 1 |
Acelyrins stumble could help rival MoonLake Immunotherapeutics which is also developing an IL17 treatment for hidradenitis suppurativa and psoriatic arthritis Cantor Fitzgerald analyst Prakhar Agrawal said in a note to clients | 0 |
Most of the shots are either in early stages of testing or are still preclinical but the company has pushed an experimental shot for cytomegalovirus into Phase 3 | 1 |
The test is designed to identify epigenetic changes that signal the start of tumor development | 1 |
The panel nonetheless chose to back patisiran citing the drugs manageable side effects and modest benefit | 1 |
The job cuts announced Tuesday will affect about 225 employees | 0 |
Sana Biotechnology which just two years ago was one of the most wellfinanced startups in the industry plans to make a second round of layoffs in less than 11 months to save cash | 0 |
We are surprised and disappointed that the FDA introduced new expectations for the first time in the Complete Response Letter | 0 |
But roughly 70 of the estimated 850 or so GPCRs in the human body are currently considered undruggable according to Superluminal. That leaves plenty of room for young biotechs to try to develop new ways of targeting those receptors | 1 |
RayzeBios private backers included Viking Global Capital VenBio Versant Ventures Samsara Biocapital and Sofinnova Investments | 1 |
The theme of starting with a more homogeneous disease setting and then moving out into a broader population is certainly something thats been around for decades said Goldstein | 1 |
Superluminal was cofounded by former RA Capital Management executive Ajay Yekkirala and former Evotec executive Cony Dcruz. Insight Partners Nvidia and Gaingels joined RA Capital in the seed round | 2 |
Nimbus next act is led by a medicine dubbed NDI101150. Like Nimbus other medicines the drug is a small molecule | 1 |
Such risks may be amplified by the impacts of the COVID19 pandemic | 1 |
About SRP9001 delandistrogene moxeparvovec SRP9001 delandistrogene moxeparvovec is an investigational gene transfer therapy designed to address the underlying cause of DMD through the targeted production of functional components of dystrophin in muscle tissue | 1 |
With a singular focus on developing curative medicines we aim to rapidly translate our treatments from bench to bedside | 2 |
Biogen leadership has said that as part of the research overhaul there will be greater investment in the areas of immunology neuropsychiatry and rare disease. To that end in July the company announced the biggest deal in its history by unadjusted dollar terms a planned 73 billion acquisition of rare disease drugmaker Reata Pharmaceuticals | 2 |
The data in major depressive disorder were more mixed however. A key study failed in 2019 leading Sage to redraw its development plans | 0 |
FTD is designed to help treatments reach patients faster by facilitating the development and expediting the review of therapies with potential to address unmet medical needs for a serious or lifethreatening condition | 1 |
ForwardLooking Statements This press release contains forwardlooking statements | 1 |
If the patient is breastfeeding or plans to breastfeed | 1 |
The event will be webcast live under the investor relations section of Sareptas website at httpsinvestorrelationssareptacomevents presentations and following the event a replay will be archived there for one year | 1 |
NonGAAP selling general and administrative expenses were 540 million and 551 million for the three months ended June 30 2021 and 2020 respectively a decrease of 11 million | 1 |
The drug came to Aiolos via Chinas Jiangsu Hengrui Pharmaceuticals from which the startup licensed AIO001 in August | 1 |
Fresh off 4B deal Nimbus gears up for ʻthird chapterʼ with new fundraise. The Boston biotech has sold two experimental drugs a liver disease medicine and a TYK2 inhibitor to Gilead and Takeda respectively | 2 |
But our particular approach to doing that is to develop a framework that really can effectively start with 4000 different Mendelian disease genes and pull out the ones that really represent the best opportunities According to Goldstein the pace of research into Mendelian diseases single gene disorders like cystic fibrosis and sickle cell has moderated after a period of rapid discoveries enabled by genetic sequencing | 1 |
The move will affect 29% of Sanas workforce or about 123 employees based on the companys headcount of 424 fulltime employees at the end of June | 0 |
Still the moves are a notable step back for a company that launched two years ago with an approved cancer cell therapy a builtout drug pipeline and hundreds of millions of dollars. At the time Bluebird was struggling to turn research progress in cell and splitting the company as a better way forward with 2Seventy inheriting the companys oncology work and a resized Bluebird focusing on gene therapies for sickle cell disease and beta thalassemia | 1 |
We anticipate a European Commission decision on our MAA during the fourth quarter of 2023 | 1 |
Its treatment dubbed VGA039 is designed to restore production of a bloodclotting enzyme by targeting a molecule known as protein S The company is testing it in a Phase 1 trial for the blood disorder von Willebrand disease | 2 |
Their treatment called exacel is difficult to make requiring a patients own blood stem cells to be extracted and then engineered using CRISPR gene editing technology. Its also likely to come at an extremely high price possibly creating a sticking point with insurers | 2 |
From there we scheduled and have now completed a very productive and informative endofphase2 meeting with the FDA and on that basis we anticipate initiating Study SRP9001301 EMBARK our pivotal trial for SRP9001 in September of this year in the United States and globally | 2 |
The company recently disclosed Phase 1 data from a drug targeting another protein known as gamma secretase thats related to amyloid buildup | 0 |
CART biotech Cargo Therapeutics prices 281M IPO The offering is one of the largest for a biotech company in 2023 a year that has otherwise been sluggish for drugmaker IPOs | 0 |
Its focusing on an ongoing Phase 3 trial of pegcetacoplan next year | 1 |
Gilead is adding another earlystage biotechnology company to its rolodex of partners announcing Tuesday a multipart research alliance with the San Diegobased Tentarix Biotherapeutics | 2 |
BrainStorms share price dropped 5 Wednesday trading at 17 cents | 0 |
Emerging biotech Laronde Senda Biosciences merge to create new Flagship company Sail Biomedicines will combine Larondeʼs work on endless RNA with Sendaʼs research on programmable nanoparticles | 2 |
Todays positive results advance our goal to establish an industry leading TTR franchise which currently includes ONPATTRO and AMVUTTRA for the polyneuropathy of hereditary ATTR amyloidosis | 2 |
Affected children may experience developmental delays such as difficulty in walking climbing stairs or standing from a sitting position | 1 |
Originally submitted for FDA review in 2019 the therapys application was derailed by the partial shutdown of the federal government that year. Approval when granted came with strings attached as the FDA ordered Aimmune to sell Palforzia through a risk management program designed to protect people from inadvertent allergic reactions | 1 |
Other AIfocused companies like Recursion Pharmaceuticals, Exscientia, BenevolentAI, and Insitro have drawn similar large drugmaker partnerships too | 1 |
The enthusiasm and continued support of our existing investors combined with the significant commitment from this new slate of top tier funds provides validation for our circuitbased discovery platform approach the quality of our clinical assets and the strong capabilities of our team said Christopher Kroeger the companys founder and CEO in a statement | 2 |
Histogen plans to wind down operations after failing to find a partner The San Diego-based drugmaker will delist its shares on the Nasdaq stock exchange and sell its assets | 0 |
They noted how some subgroups did not seem to experience much benefit on one or both of the trials measures including women, Black participants, and those taking tafamidis at the trials start | 1 |
ProQR sale of eye drugs comes undone over employment contracts. The Dutch company said its focus will remain on an RNA editing platform called Axiomer | 1 |
The patisiran and placebo arms had similar frequencies of adverse events AEs 912 percent and 944 percent respectively and serious adverse events SAEs 337 percent and 354 percent respectively | 1 |
For JJ which records almost 7 billion in annual sales from brain drugs like Concerta and Invega Sustenna Arialys is another bet on neuroscience | 2 |
A few months later the company announced plans to lay off as many as 500 employees as it cut costs to conserve cash for operations. Aprocitentan is the companys best hope for a major cash influx | 0 |
The increase in cost of sales is primarily due to increased demand for the Companys products | 1 |
Genesis work has drawn the attention of major pharmaceutical companies Eli Lilly and Roche which partnered with the startup in 2022 and 2020 respectively | 1 |
Currently there are no approved diseasemodifying therapies that treat the genetic root cause of the disease | 1 |
This press release contains forward looking statements that involve substantial risks and uncertainties | 1 |
Taysha expects to receive gross proceeds of 150 million from the Private Placement which is expected to close August 16 2023 before deducting placement agent commissions and offering expenses | 1 |
ALS patients lose the ability to perform essential tasks like talking eating and breathing and typically live just two to five years after diagnosis, disease is fatal and often fast-progressing patients and advocates have pressured the FDA to adopt more flexible policies that would allow easier and quicker access to treatments | 0 |
Shares in the company nearly quadrupled in value and took the companys valuation to more than 7 billion sparking an investor rush to find other companies working on NASH drugs | 2 |
The foundation said establishment of its plant should help concentrate cell therapy expertise in one place to create a largescale production facility | 1 |
Verge is currently developing a drug for ALS known as VRG50635. The small molecule drug is meant to restore degenerated neurons by inhibiting PIKfyve, an enzyme that research indicates performs various important cellular functions. It advanced into human testing last year | 2 |