daniel-gordon/biopharma-dive huggingface dataset:
Per deal terms Embark shareholders could receive up to 456 million euros in additional payments based on achievement of certain development regulatory and commercial goals. The startups staff will continue their research under a new company called Embark Laboratories
Amyloid protein clumps together to form plaques between neurons in the brains of people with Alzheimers. Ultragenyx will spin out a new company to take advantage of research it claims could lead to a new way of treating Alzheimers disease
Arialys bought the medicine from Astellas Pharma where Matsumoto previously served as head of the Japanbased companys neuroscience research unit
The success of GLP1 drugs in managing diabetes and weight loss has also fueled billions of dollars in sales for Lilly, which makes Mounjaro, and Novo Nordisk the maker of Ozempic and Wegovy
The patient was able to sit unassisted for the first time in over a decade and she demonstrated the ability to unclasp her hands and hold an object steadily for the first time since infancy
Biotechnology company Triveni Bio launched Thursday with 92 million in Series A funding and a focus on what it calls geneticsinformed precision medicine approach to immune and inflammatory diseases
Benefits of FTD to programs include early and frequent interactions with the FDA during the clinical development process and if relevant criteria are met the FDA may also review portions of a marketing application before the sponsor submits the complete application
This is twice the dystrophin expression compared to the 20 mgkg cohort at week 12 mean expression of 306 and eight times that of the eteplirsen comparison group mean expression of 082
BrainStorm submitted an approval application even though NurOwn had failed to meet any of the treating ALS or amyotrophic lateral sclerosis. Notably the FDA had initially declined to review BrainStorms application
Precede has used the money to develop a research-use-only platform that enables biopharma companies to see the activity of genes and pathways in tissues using 1mL plasma samples
Former Novartis executive Tsai to lead UK heart drug startup John Tsai previously Novartisʼ chief medical oﬃcer has been named CEO of Forceﬁeld Therapeutics a new biotech working on a treatment for heart attacks
The medicines make up a regimen that is being studied in multiple tumor types. Agenus could seek an approval in colorectal cancer next year. By zeroing in on botensilimab and balstilimab we expect to expedite regulatory approval and availability for healthcare providers and patients in need said Chairman and CEO Garo reflects our commitment to the success of these programs while optimizing shareholder value The company had 175 million in cash at the end of the second quarter enough to fund operations for about a year
Today we are taking hard but necessary steps to streamline our team and optimize our RD approach and cost structure. The layoffs will affect 176 employees 2Seventy said
Fundraising for private rounds can take on average six months so Bioluminescence aims to move similarly quickly on its end
Researchers have long focused on clearing amyloid plaques in the brain as a way of battling Alzheimers even though some experts have questioned the approach . Despite decades of work and billions of dollars  there is no cure for the disease
The company is also temporarily suspending all of its preclinical and clinicalstage research so it can pool its resources towards its most advanced work a combination of two cancer immunotherapies known as botensilimab and balstilimab
Initially the function of these Molecular glues was uncovered somewhat by chance. But scientists in recent years have intentionally designed them into a class of medicines also called targeted protein degraders  to destroy diseasecausing proteins
Kapellers company Rome Therapeutics on Tuesday revealed it has raised another 72 million to advance its research drawing in new investors Johnson  Johnson and Bristol Myers Squibb into a Series B extension round
As pills meanwhile TYK2 drugs could be more convenient alternatives to injectable drugs like AbbVies Humira or Amgens Enbrel. With Mondays results Ventyx now appears to be out of the TYK2 race in two indications
We are pleased that VOXZOGO is now available for children of all ages with achondroplasia said Hank Fuchs MD president of Worldwide Research and Development at BioMarin
Dee Datta the cofounder and CEO of Switch Therapeutics  said some companies need to be more disciplined when it comes to research and development. Not every new innovation can become a startup company and neither can each new innovation become a product she said
In line with previously reported clinical data no clinically relevant complement activation was observed in these 11 patients
While we saw exceptional expression after only a few initial doses our models predict that we will exceed dystrophin expression levels of 10 of normal or greater over time with SRP5051 said Doug Ingram president and chief executive officer Sarepta
About Duchenne Muscular Dystrophy DMD is an Xlinked rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death
Ultragenyx to spin out new company around Alzheimerʼs gene therapy CEO Emil Kakkis says the companyʼs ﬁndings are too exciting to ignore but the highrisk highreturn venture needs to be pursued outside the organization
And that means that we need is often very complicated. Goldsteins vision is to pick rare disease drug targets that are also relevant to conditions affecting larger groups of people
Right now it still remains a market at least on the IPO side from a demand perspective thats very much driven by the specialist investors Eisele said
Adlai Nortye has focused on buparlisibs potential in different settings and in combination with other drugs. The furthest along is a latestage study testing the drug and chemotherapy in people whose head and neck cancers have progressed after a commonly used immunotherapy
Sarepta Therapeutics Announces Advisory Committee Meeting will be Held for SRP9001 31623  Advisory committee meeting to be held in advance of target action date  Company will hold conference call today at 430 pm Eastern time CAMBRIDGE Mass
Epigenic joins wave of startups raising cash to edit the epigenome. The China-based company is one of at least four young biotechs in a competitive race to use CRISPR tools to alter gene expression without changing DNA
The announcement comes less than a month after the FDA convened an outside group of advisers to evaluate NurOwn which is made from a patients own stem cells and designed to help protect and grow neurons
Moderna aims for the stars again with ambitious drug development plan. The biotech is telling investors it expects to launch as many as 15 new products from which it is forecasting billions of dollars in annual sales by 2028
To date OrbiMed has invested in almost 200 startups  including recent investments in biotechs like Star Therapeutics  Upstream Bio and Rampart Bio a company that launched Tuesday
Their attempts to go public will test investor demand for biotech offerings in a slow year for IPOs
Although it is passed down from parents to children about 13 of cases are caused by a spontaneous mutation a new mutation that was not inherited
Three biotechs Lexeo Therapeutics Abivax and Kairos Pharma on Friday filed plans to go public. All three have at least one drug in earlystage human testing matching analysts predictions about which companies would be in the strongest position to launch an IPO
Seraxis plans to implant its cell therapy dubbed SR02 in humans who will be taking drugs to keep their bodies from rejecting a foreign substance similar to the treatment a patient would get after receiving a full organ transplant
The fresh funding will allow OrbiMed to continue to back new companies across life sciences. Investors contributing to the raise foundations pension funds and sovereign wealth funds OrbiMed said
Administration for permission to begin human testing of its most advanced program an experimental medicine known as ART5803
It is not known if VOXZOGO passes into breast milk
Other tissuebased diagnostics test for markers such as BRAF and KRAS
While the FDA isn't required to follow the advice of its advisory committees, it usually does
Arialys debuts with 58 million from a group of life sciences investors that includes the biotechs founding backers  Avalon BioVentures, Catalys Pacific, and MPM BioImpact, along with Alexandria Venture Investments and Johnson  Johnsons venture arm
Flagship expands into Asia Pacific wi th new Si ngapore hub The biotech company creator hopes its new oﬃce will help its portfolio companies grow and strike partnerships in the region
Biotech IPOs have slowed considerably amid a sectorwide slowdown dating back to last year
What should you tell the doctor before or during taking VOXZOGO
At the Sept 27 meeting FDA staff expressed concern not only with the safety and effectiveness of NurOwn but also BrainStorms data analysis and conclusions
Initially BARDA will fund up to 10 million over a base period that runs through the first quarter of 2024
While some patients benefit greatly from CART therapy  companies like Gilead have recently received expanded clearances for their CART drugs based on strong data, not all do. Others relapse months after treatment
As a result Alnylam no longer plans to seek an expanded approval for patisiran which is approved in the US as Onpattro for people with ATTR amyloidosis who have polyneuropathy or nerve damage
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The link to the live webcast will also be available on the Investors  News section of the Ascendis Pharma website at httpsinvestorsascendispharmacom
The round also comes during a prolonged downtown in the biotech stock market that has made raising money more the larger funds that help move startups closer to an initial public offering  an important milestone for many young biotechs
This development a month from the agencys PDUFA action date may lead to a delay in the FDAs final regulatory decision on the TransCon PTH NDA  said Jan Mikkelsen Ascendis Pharmas President and Chief Executive Officer
Even though regulatory situations are inherently opaque Burnett said he still expects Vertex and CRISPRs option to ultimately win FDA approval
Approved in Europe in 2020 as Libmeldy the gene therapy has been used in patients from six different countries since its commercial launch there
The increase is primarily due to increasing demand for the Companys products and the writeoffs of certain batches of EXONDYS 51 not meeting the Companys quality specifications for the six months ended June 30 2021 with no similar activity during the same period of 2020
Launched on Tuesday Arialys Therapeutics says it was founded on recent scientific discoveries that point to the immune system as a main culprit behind some of these disorders
At 12 weeks posttreatment compared to baseline the study met its primary biological endpoint of microdystrophin protein expression P00001
The decrease is primarily driven by cash used to fund the Companys ongoing operations during 2021
Amortization of inlicensed rights For each of the three months ended June 30 2021 and 2020 the Company recorded amortization of inlicensed rights of approximately 02 million
After years of ups and downs the company in June finally won US approval for Roctavian  the first with payers have made it difficult to move patients onto the therapy which has a list price of 29 million in the US and is also approved in Europe
Rather than blocking cancerdriving proteins as many targeted small-molecule drugs do protein degradation tries to break them down
Regulators cleared the treatment called Lantidra for patients who suffer from repeated severe episodes of low blood sugar while trying to manage their diabetes
While CART therapies approved by the Food and Drug Administration for lymphoma target a protein called CD19 CRG022 is aimed at a different dont respond to or have evaded CD19-directed treatment
Many of these would-be therapies take advantage of the proteasome which functions like the bodys garbage disposal system for unnecessary or damaged proteins. In effect these treatments are meant to tag a diseasecausing protein for destruction by the proteasome
Emerging biotech Lessons from a biotech downturn Funding challenges an IPO dry spell and what to expect in 2024 Data is king and itʼs something that is really standing true in this market said Kevin Eisele managing director at investment bank William Blair
Obviously we do that on the consumer side in returning the reports but we also felt like we needed to make our best attempt at saying lets see if we can actually solve these problems
The Food and Drug Administration has recently stepped up scrutiny of PI3K inhibitors due to safety concerns that emerged during testing in certain blood cancers
Otsuka partners with Shape in deal to design eye gene therapies The companies will work together to pair Shapeʼs adeno associated virus technology with Otsukaʼs genetic payloads
With its filing Neumora is one of four biotechs to set plans for an IPO since late July following closely after another high profile startup RayzeBio filed to do so earlier this week
UniQure lays off 20 of staff cuts research to ʻsignificantlyʼ lower costs The biotech is discontinuing work on a Parkinsonʼs drug candidate among other programs as it focuses on its experimental Huntingtonʼs treatment
Although kidney toxicity was not observed in the clinical studies with AMONDYS 45 kidney toxicity including potentially fatal glomerulonephritis has been observed after administration of some antisense oligonucleotides
There were two so-called serious adverse events but each was deemed unrelated to treatment
Laidoff staff can land new jobs at other companies in quick succession  particularly if they have specialized skills. And while new funding has been harder to obtain venture capital firms continue to raise new funds  suggesting they see plentiful opportunities for investment. That said venture investors appear to be shifting how they deploy those funds  insisting on clearer timelines to milestones that create value
More recently it partnered with a Japanese company called Heartseed to develop a heart failure treatment that has entered clinical trials and with the Canadian company Aspect Biosystems for bioprinting of cell therapies for Type 1 diabetes and obesity
The company's most advanced program has already entered human testing as a potential therapy for drugresistant seizure disorders
At RD Day in June 2023  Taysha provided an overview of new comprehensive data analysis and development of diseaseprogression model DPM which the Company believes has the potential to address FDA feedback regarding the heterogeneity of GAN and effortdependent nature of MFM32 as the primary endpoint in an unblinded study New comprehensive data analysis utilizing the DPM submitted as meeting request to the FDA feedback for a potential regulatory pathway for TSHA120 expected in Q3 2023 FDA feedback on CMC module 3 amendment concluded that the analytical data is sufficient to support the comparability of pivotal lot and release for use in clinical studies Second Quarter 2023 Financial Highlights Research and Development Expenses Research and development expenses were 198 million for the three months ended June 30 2023  compared to 235 million for the three months ending June 30 2022
Concentras bid filed Friday proposes to acquire all outstanding shares from Rain for 125 per share plus a type of security known as a contingent value right that promises Rain shareholders 80 of the proceeds from any license or sale of the companys programs
Rampart which takes its name from a word meaning to fortify hopes to create a treatment that could be more longlasting and effective for those patients
Many of the medicines used to treat Parkinsons symptoms revolve around dopamine a chemical messenger that plays a role in a variety of essential functions including motor function. Bluerocks therapy targets this chemical as well but in a way different from currently available treatments as its meant to replace the dopaminemaking nerve cells that are lost in Parkinsons patients
About Ascendis Pharma AS Ascendis Pharma is applying its innovative platform technology to build a leading fully integrated  global biopharma ceutical company focused on making a meaningful difference in patients lives
News of Verves and Intellias regulatory progress could give a lift to a field thats been battered this year by a down market for biotech
Developers of bloodbased multicancer screening tests such as Illuminas Grail use genomic sequencing and other approaches to detect signals from cellfree DNA methylation mutations and analytes including proteins
With Tuesdays approval of Xphozah Ardelyx is the rare company to overcome such a hurdle some two years after it received a complete response letter from the agency in July 2021
Earlier this week gene therapy biotech Lexeo Therapeutics priced an offering that came below its expected share price raising 100 million for experimental cardiovascular and neurodegenerative disease drugs
Along with JJ and Bristol Myers Squibb its investors include Sanofi Eurofarma Arch Ventures and Mass General Brigham Ventures
The Danish firm is looking to strengthen its hold through dealmaking with the latest Canadian biotechnology company Inversago Pharma
We are pleased to receive FTD from the FDA which underscores the significant unmet medical need in patients with Rett syndrome and the potential of TSHA102 to serve as a meaningful treatment option said Sukumar Nagendran  MD President and Head of RD of Taysha
Turning off PCSK9 could powerfully lower LDL cholesterol high levels of which are closely intertwined with cardiovascular disease
A weekly diabetes treatment Ozempic followed which it sells in weightloss as Wegovy although Ozempic is widely used offlabel to treat overweight people. These drugs regulate blood sugar by activating a hormone called GLP1
Despite these regimens many people continue to experience breakthrough bleeds resulting in progressive and debilitating joint damage which can have a major impact on their quality of life
To access the call please register online at httpsregisterveventcomregisterBI6ac74b5406af42c5980ba67c6f9e419d httpsctsbusinesswirecomctCT
The biotech previously secured a partnership with Johnson  Johnson on an experimental treatment for an inherited eye disorder known as retinitis pigmentosa
The FDA had granted valoctocogene roxaparvovec Priority102023 651 PM BioMarin Receives Complete Response Letter CRL from FDA for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A  Au httpsinvestorsbiomarincom20200819BioMarinReceivesCompleteResponseLetterCRLfromFDAforValoctocogeneRoxaparvovecGeneTherapyforSe 36Review status and Breakthrough Therapy and Orphan Drug designations
Rapports work revolves around RAPs or receptorassociated proteins. These proteins unlike other kinds are local to specific areas of the brain and central nervous system. By targeting RAPs Rapport believes it can create precision drugs that are either more effective or safer than other treatment options
Although the approach was successful  the company sold almost 55 million doses of its mpox vaccine to the US government during the surge of cases last summer  the business model is lumpy CEO Paul Chaplin said
We believe these data take us one step closer to achieving our Alnylam Px25 vision of becoming a leading biopharma company
Obesity drugs Novo continuing deal streak buys another obesity drug startup, Founders of Embark Biotech have researched proteins that regulate energy and the breakdown of fatty tissues
Ned Pagliarulo Lead Editor Courtesy of US Food and Drug Administration Food and Drug Administration scientists have major concerns with an application filed by biotechnology company BrainStorm Cell Therapeutics for approval of a personalized stem cell treatment for amyotrophic lateral sclerosis documents posted online Monday show
The company believes the path forward will include a Phase 3b clinical trial so its actively exploring the development of a protocol for an additional study
You may report side effects to BioMarin at 18669066100
We are pleased with the progress we have made this quarter in the clinical evaluation of our two lead investigational programs
The process of streamlining Sangamos pipeline has been accelerated within todays challenging economic environment and we have had to make difficult decisions to defer further investments and seek collaboration partners or direct investment in both our Fabry gene therapy and CARTreg cell therapy programs said Sandy Macrae CEO of Sangamo in the companys statement
In late 2019 Michael Ehlers the companys former research and development head left to join the life sciences venture capital firm Apple Tree Partners
Biotechnology company Rain Oncology disclosed Monday that it has received a buyout offer from Concentra Biosciences via Concentras owner Tang Capital
While we are disappointed that we must communicate a change in decision after our prior statement on the topic we are not disappointed with the decision to hold an advisory committee
Ascendis does not assume any obligation to update any forward looking statements except as required by law
The Food and Drug Administration has approved two drugs designed to slow Alzheimers progression by targeting amyloid plaques. The first Biogens Aduhelm was embroiled in controversy amid questions about how much it benefits patients. The second a drug called Leqembi developed by Eisai and Biogen may prove more successful after initial pushback from insurers before its full approval in July . The data supporting it are clearer in showing a benefit to treatment
The company has been investing in the country since 1997 and the expansion was part of a fiveyear plan to invest up to 500 million into its Singapore headquarters
Shares in Madrigal fell by about 10 on news of Sibolds appointment potentially reflecting investors resetting their expectations on whether the company will be bought by a larger drugmaker
The Lexington Massachusetts-based biotech plans to cut just over 85 jobs in the restructuring a move that is expected to save the company about 40 million by the end of the year
23andMes growth has not been without controversy. From data privacy concerns to the possibility of discrimination based on genetic information the company has had to navigate ethical concerns. Its currently embroiled in a breach of customer user data thats drawn scrutiny from the Connecticut attorney general
Another 18 patients from the trial have been treated but hadn't been followed for longer than 12 months when the data were analyzed
Notably the FDA found that at study completion more trial participants in the arm receiving BrainStorms drug had died than in the group who got a placebo
Elsewhere Biogen is in the early stages of launching three products. One Qalsody was approved in April for a small segment of ALS patients who have specific genetic mutations. Another Zurzuvae is a new postpartum depression drug that Biogen secured rights to through a multibilliondollar collaboration agreement with Sage Therapeutics
Among participants with treatmentrelated adverse events 82 were mild or moderate in severity and 4 participants experienced serious treatmentrelated adverse events including 3 participants in the treatment group 2 cases of rhabdomyolysis 2 transaminase elevations and 1 participant in the placebo rhabdomyolysis
Analytics company Global Data estimates that pharma companies signed roughly 16 billion worth of antibodydrug conjugate licensing deals in 2022
Bluebirds announcement Wednesday doesn't change the overall timeline for the experimental treatment. The company won a priority review with the FDA promising an answer on the application for lovocel by Dec 20
In Europe Ascendis submitted it s Marketing Authorisation Application MAA for TransCon PTH in hypoparathyroidism to the European Medicines Agency in November 2022
Developing GB0669 from its start to clinical testing took a year and a half  a pretty darn fast turn-around thanks to Generates platform