daniel-gordon/biopharma-dive huggingface dataset:
In the absence of dystrophin which is required to strengthen and protect muscles muscles become weakened and damaged
Eventually increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support and cardiac dysfunction can lead to heart failure
The plan announced Monday to look at different doses of the two treatments was welomed by some analysts
We encourage investors and potential investors to consult our website regularly for important information about us
These forwardlooking statements include without limitation statements relating to our future operations business plans priorities research and development programs SRP9001s potential for accelerated approval the Companys plans to continue working with the FDA as they complete their review of the SRP9001 BLA the potentially transformative benefits of SRP9001 and that the FDA is not bound by the advisory committee recommendation but takes its advice in consideration when reviewing applicationsActual results could materially differ from those stated or implied by these forwardlooking statements as a result of such risks and uncertainties
Gilead allies with targeted drug startup Tentarix in latest research bet As part of the deal Gilead will hold options to acquire up to three Tentarix subsidiaries built around drug programs the companies discover and develop
A third similarly acting drug called donanemab and from Eli Lilly could receive an FDA approval by the end of the year
The drug also received a vote of confidence in March when Royalty Pharma bought a portion of royalty rights held by PureTech Health which had originally advanced KarXT
Gene editing Verve gets FDA green light to run base editing study in US The trial which is ongoing in the UK and New Zealand has been on hold in the US since late last year as the FDA sought more details from Verve
Tentarix claims its drugs will be able to engage more than two targets at a time and work in a broader array of cellular settings. The company raised $50 million in funding from Versant Ventures and Samsara Biocapital in October 2021 and has now drawn Gileads attention
Some of the Series C round announced Tuesday will support one of the spinouts Star has already launched a company called Vega Therapeutics. The rest will be used to develop new drug candidates and subsequently the startups it creates around them
At the September advisory committee meeting BrainStorm highlighted these approvals and the regulatory flexibility exercised by the FDA
Ventyx along with others in the field hope to match or surpass Bristol Myers Squibbs firsttomarket therapy Sotyktu  which the pharmaceutical company forecasts will earn 4 billion in annual sales
ProQR CEO said his company was disappointed the deal fell apart. Still the focus at ProQR will remain on a number of earlystage RNA editing programs that use the companys Axiomer technology he said
Alternatively use a laboratory test that does not use the reagent pyrogallol red as this reagent has the potential to cross react with any VYONDYS 53 that is excreted in the urine and thus lead to a false positive result for urine protein
Reporter: You said your company was set up from the beginning as a research database. What role has that played in the companys growth, anything that surprised you? CEO: We had a really good set of advisers early on and we spent a lot of time on ethics. Its unusual because as a startup tech company we had consent forms right away and we spent lots of time soliciting feedback on consent and on getting input
Syfovre got off to a hot start far surpassing analyst expectations for sales in its first months on the market
PTC worked with Roche and the Spinal Muscular Atrophy Foundation to develop Evrysdi which was approved by the Food billion Swiss francs or about 12 billion last year and continues to have strong growth  making it an attractive target for Royalty
On Wednesday Moderna said that it is currently resizing its manufacturing footprint and supply base to improve its margins toward a longerterm target of 75 to 80
About Rett Syndrome Rett syndrome is a rare neurodevelopmental disorder caused by mutations in the Xlinked MECP2 gene which is a gene thats essential for neuronal and synaptic function in the brain
Any of the foregoing risks could materially and adversely affect the Companys business results of operations and the trading price of Sareptas common stock
The Boston biotechnology company has become one of the sectors most successful privately-held companies generating large returns for its investors through deals with Gilead Sciences and most recently Takeda
A replay of the call will be available two hours aer the call and archived on the same web page for six months
ROCTAVIAN is not intended for administration in women
Reducing the pathogenic protein leads to a reduction in amyloid deposits in tissues
NonGAAP selling general and administrative expenses were 1056 million and 1096 million for the six months ended June 30 2021 and 2020 respectively a decrease of 40 million
Just 14 companies have completed an IPO so far this year compared to 22 last year and 104 in 2021
The committees positive vote is based on the evaluation of the totality of evidence including the SRP9001 product design as well as biological and empirical data
During treatment monitor urine dipstick every month and serum cystatin C and urine proteintocreatinine ratio UPCR every three months
Adverse Reactions The most common adverse reactions that occurred in patients treated with ONPATTRO were upper respiratory tract infections 29 and infusionrelated reactions 19
These nonGAAP measures are not intended to be considered in isolation or to replace the presentation of the Companys financial results in accordance with GAAP
Adverse reactions in DMD patients N8 treated with EXONDYS 51 30 mg or 50 mgkgweek by intravenous IV infusion with an incidence of at least 25 more than placebo N4 Study 1 24 weeks were EXONDYS 51 placebo balance disorder 38 0 vomiting 38 0 and contact dermatitis 25 0
Valoctocogene roxaparvovec maintains Breakthrough Therapy and Orphan Drug designations
Almost 17 of those treatment naive patients experienced brain swelling after receiving subcutaneous Leqembi while 22 experienced the bleeding
Companies that are further along or have drugs in clinical testing have a better chance according to Jordan Saxe senior managing director at Nasdaq
Our vast pipeline is driven by our multiplatform Precision Genetic Medicine Engine in gene therapy RNA and gene editing
Developed by the biotechnology company Aimmune Therapeutics Palforzia won Food and Drug Administration approval in 2020
Part A of MOMENTUM is now complete and Sarepta will work with great urgency to discuss the results with regulatory agencies and gain their insights including the development path to support an accelerated approval of SRP5051 in the United States Results from the 30 mgkg dose cohort In biopsies taken at a median of week 12 30 mgkg of SRP5051 dosed monthly resulted in mean exon skipping of 1079 n4
When its shares fell by almost 20 after the company adjusted its financial outlook alongside third quarter earnings
Kinnate Biopharma a biotechnology company focused on targeted cancer therapies is reducing its workforce by 70 leaving only 28 fulltime employees as it adjusts research priorities
Analysts and investors expect the pace of public offerings to pick up in the coming months
Other midstage assets include two antibody drugs that target another protein implicated in Alzheimers disease called tau
Forbion used money from its new Growth Opportunities Fund II, which closed in April with 600 million euros committed to colead VectorYs Series A
The increase in research and development expenses primarily reflects the following 189 million increase in upfront and milestone expenses primarily due to a 287 million increase of an accrued sublicense fee to Nationwide Childrens Hospital Nationwide and a 30 million expense incurred as a result of a milestone achievement in a research and license agreement during the three months ended June 30 2021 offset by 120 million of upfront payments as a result of the execution of certain research and license agreements during the same period of 2020 166 million increase in manufacturing expenses primarily due to a continuing rampup of the Companys gene therapy programs 98 million increase in clinical trial expenses primarily due to increased patient enrollment for the Companys ESSENCE and MOMENTUM programs as well as certain startup activities for the Companys SRP9001 microdystrophin program 60 million increase in research and other primarily driven by an increase in sponsored research with academic institutions during the three months ended June 30 2021 40 million increase in facility and technologyrelated expenses due to the Companys continuing expansion efforts 32 million increase in preclinical expenses primarily due to an increase of toxicology studies in the Companys PPMO platforms 21 million increase in compensation and other personnel expenses primarily due to a net increase in headcount 17 million increase in stockbased compensation expense primarily driven by increases in headcount and changes in stock price10 million decrease in collaboration cost sharing with Genethon on its microdystrophin drug candidate and 93 million increase in the offset to expense associated with a collaboration reimbursement from Roche primarily due to continuing development of the Companys microdystrophin gene therapy
Eczema is believed to result from both skin barrier dysfunction and immune dysregulation, trials  are focused on soothing the inflammation caused by the immune system
Against these lower sales estimates Sage this week announced that it will lay off roughly 40 of its workforce as part of a larger restructuring meant to save money and prioritize certain research programs
Continued approval may be contingent upon verification of a clinical benefit in confirmatory trials
The company estimates that approximately 100000 people in the US have the condition. No cure exists for AATD instead doctors treat the symptoms of the disorder or patients can receive weekly injections of the alpha1 antitrypsin protein. Airna is looking to develop a therapy that can correct the mutation that produces the errant protein
Stockholders are urged not to place undue reliance on forwardlooking statements which speak only as of the date hereof
After IPO success Acelyrin hits trial setback with inflammation drug. The companyʼs lead drug izokibep missed its goal in a latestage trial though Acelyrin said it sees some hope in the results
Genesis raises 200 million for AI drug discovery research Led by Andreessen Horowitz and an unnamed life sciences investor the funding will help Genesis move its ﬁrst drug candidates into clinical testing
Net loss Net loss for the three months ended June 30 2023 was 246 million or 038 per share as compared to a net loss of 341 million or 085 per share for the three months ended June 30 2022
Late last year Nestlé admitted it was reviewing options for the business and subsequently wrote down much of Aimmunes value
Rome Therapeutics based in Boston is exploring ways to study the socalled dark genome
Now with another 150 million, Rapport is extremely well positioned to advance our ongoing clinical programs in seizure and psychiatric disorders and to expand our pipeline by harnessing our unique capability to leverage receptorassociated proteins for precision neuromedicine Ceesay said in a statement Wednesday
Mariana based in Cambridge Massachusetts plans to use the new money to advance a pipeline of radiopharmaceutical drugs into clinical testing including its lead candidate for small cell lung cancer. While radiation therapy is a mainstay of cancer care it can come with harsh side effects and can inadvertently damage healthy tissue. By comparison radiopharmaceuticals introduce radiation directly into tumors by pairing a radioactive isotope with a targeting compound
We work under a corporate strategy where its really important to help people living with obesity diabetes and cardiovascular diseases. Our primary focus is to find nextgeneration therapeutics
Milestone payments are made when certain development regulatory and sales milestone events are achieved
AbbVies Rinvoq and Pfizers Cibinqo treat psoriasis by suppressing proteins that regulate inflammation for instance
Though the study assessed just one of the potential uses for izokibep the high placebo response raised questions among analysts about whether the trial was well executed
Gene editing Vertex gearing up for launch as sickle cell therapy review advances Executives at the biotech say theyʼre trying to get ahead of the payer and production challenges that will face their exacel therapy which is made through an exhaustive and expensive process
The Company plans to meet with the Agency in the coming weeks to align on the next steps to obtain approval
In a controlled clinical study 19 of ONPATTROtreated patients experienced IRRs compared to 9 of placebotreated patients
This was a result of a settlement and license agreement with BioMarin in July 2017
It's approach use a stem cell line generated from human pancreas to produce groups of cells known as organoids that can replicate the work of an organ
Limited treatment adoption hurt by the pandemic and complicated dosing led to the 2022 strategic review and in February Nestlé revealed it had taken an impairment charge of 19 billion Swiss francs on Palforzia and Aimmune
BioMarin to replace CEO Bienaimé with Genentech veteran. The rare disease drug developer is turning to former Genentech head Alexander Hardy as the launch of its Roctavian gene therapy stumbles
Alfasigma will pay Intercept investors 19 per share in cash an 82 premium from Intercepts closing price on Monday
The company recorded losses of 38 million over the same period
And I realized the world was focused on revenue optimization and not on dramatic changes to keep you change from within. If you want to change healthcare you have to change it from outside
Additionally the transaction fee related to the Roche transaction is nonrecurring and is excluded from its nonGAAP results
A closely held company controlled by the family of its founder Alfasigma markets drugs like Zelnorm and rifaximin for irritable bowel syndrome and sulodexide for postthrombotic syndrome. It also provides contract manufacturing services as well as nutritional supplements. Its factory in Shreveport Louisiana manufactures nutritional supplements for the US market
Even at an early timepoint of 12 weeks and after as few as only three doses these data confirm the potential of Sareptas nextgeneration PPMO platform to be a step order improvement over our current PMO platform and to profoundly impact the course of Duchenne
Adverse reactions observed in at least 20 of patients treated with AMONDYS 45 and at least 5 more frequently than in the placebo group were AMONDYS 45 placebo upper respiratory tract infections 65 55 cough 33 26 pyrexia 33 23 headache 32 19 arthralgia 21 10 and oropharyngeal pain 21 7
Harbinger will use the Series B funding to complete a 10000 person study of its test expand its commercial team in preparation for launch and start additional trials in highrisk populations
Examples of such statements include but are not limited to statements relating to i Ascendis plans to seek to address the deficiencies identifi ed by the FDA  ii the possibility of a delay in the FDAs final regulatory decision on the TransCon PTH NDA  iii Ascendis intent to continue to make TransCon PTH available to patients participating in its ongoing clinical trials as well as to physicians through its US EAP iv Ascendis expectations regarding a n EAP in Germany  v the timing and results of a European Commission decision on the Marketing Authorisation Application for TransCon PTH vi Ascendis expectations regarding the timing of potential commercial launch of TransCon PTH in Europe if approved  vii Page 3 of 3 Ascendis ability to apply its platform technology to build a leading fully integrated  global biopharmaceutical company and  viii Ascendis use of its TransCon technologies to create new and potentially best inclass therapies
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The new target population is much smaller than Fulcrum previously envisioned. But some analysts expressed optimism in the drugs prospects. We believe there could be a need especially among those with severe disease that could benefit from reduced recurring events andor a functional cure wrote Leerink Partners analyst Joseph Schwartz in a note to investors Tuesday
The cash raised will help the company fund clinical studies in obesity diabetes nonalcoholic steatohepatitis hypertriglyceridemia and other liver and kidney conditions
In UniQures Phase 12 trial mutant huntingtin levels in trial participants declined by half in results collected one year after treatment. But data released in June showed those levels had rebounded by the twoyear mark
About RNAi RNAi RNA interference is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today
A devastating and incurable muscle wasting disease DMD is associated with specific errors in the gene that codes for dystrophin a protein that plays a key structural role in muscle fiber function
Sibold recently departed Sanofi where he had served since 2017 as head of the pharmaceutical companys specialty medicine division formerly known as Genzyme
Moderna is also developing vaccines for other viruses including HIV and Epstein Barr for a market it estimates is worth between 10 billion and 25 billion
Flagship Pioneeringlaunched startups Laronde and Senda Biosciences are merging to form a new biotechnology company called Sail Biomedicines the companies announced Thursday
In this case researchers tested the drug in patients with a skin condition known as hidradenitis suppurativa
We caution investors not to place considerable reliance on the forwardlooking statements contained in this press release
Just days before the Reata announcement Biogen disclosed plans to eliminate 1000 jobs as part of an initiative meant to save 1 billion in annual operating expenses
PeptiDream will use its peptide discovery platform system to identify and develop candidates for potential cancer drugs against targets Genentech will focus on
You may report side effects to the FDA at 800 FDA1088 or wwwfdagovmedwatch
Mariana Oncology a biotechnology company developing radiopharmaceuticals for cancer has raised 175 million in a Series B round
Until now it has only been approved in the US for children aged 5 and older with open growth plates said William Wilcox MD professor of human genetics at Emory University
Hibben a veteran of a number of biotech executive suites will lead external business and commercial initiatives the company said
Despite an upanddown market life sciences investors have raised billions of dollars this year
In early May Apellis shares soared above 93
Another participant a 28 year-old male had a nearly fivehourlong episode after taking Sages drug during which he was unresponsive to pain stimuli documents said
Words such as anticipates believes expects intends projects plans and future or similar expressions are intended to identify forwardlooking statements