daniel-gordon/biopharma-dive huggingface dataset:
But the magnitude of eﬃcacy was less than targeted leading the biotech to end the study
The Series B round was led by Cormorant Asset Management and saw participation from other socalled crossover investors including divisions of Fidelity and Goldman Sachs
While venture companies like OrbiMed have remained active in backing new startups established biotechs have had a harder time raising funds and face a harder path to initial stock offerings
Recent investments include Ascend Cell and Gene Therapies  Entact Bio and Wugen
FibroGen plans to dig through the Duchenne data before determining the next steps for the program. Full results will be presented at an upcoming medical meeting
Orums lead drug called ORM5029 is in a Phase 1 clinical trial and targets HER2expressing solid tumors in patients with breast cancer
While the actual opportunity for FTX6058 will remain a point of interest the lifting of the clinical hold marks an important win for the new management team and instills confidence they can right a ship that has undergone a series of mishapsadversities wrote Stifel analyst Dae Gon Ha in a note to investors on Tuesday
As part of the workforce reduction the company will separate from all employees at Kinnjiu Biopharma its whollyowned Chinese subsidiary
TSHA120 has received Orphan Drug and Rare Pediatric Disease designations from the FDA and has been granted Orphan Drug designation from the European Commission
In psoriasis pills like Bristol Myers Squibbs Sotyktu have joined mainstay biologics such as Amgens Enbrel and AbbVies Humira spurring the entry of new competitors attempting to develop safer and more effective medicines
We undertake no obligation to update forwardlooking statements based on events or circumstances after the date of this press release
Seagen is committing 60 million to develop a new type of cancer drug striking a multiyear alliance with biotechnology company Nurix that aims to combine their respective technologies
However because there was a signal in the major depressive disorder studies and we cannot rule out an effect we will include suicidal thought and behavior warning language in the drugs potential label
Cleared for nonsense mutation, Duchenne muscular dystrophy Translarna has had conditional authorization from the European Medicines Agency since 2014
The Shanghaibased biotech is focused on epigenetic editing  an approach built on CRISPR technology. Its pipeline includes potential treatments for cardiovascular metabolic ocular and rare diseases as well as viral hepatitis
Government contracts are difficult to predict and the timing is difficult to explain to analysts and investors he said
The company also recently struck a deal with Freeline Therapeutics to license use of that companys adenoassociated conditions
In total the deal could be worth up to 1 billion more for Teva if certain development or launch milestones are met
By raising a large fund Bioluminescence intends to play a large role in investing in young biotechs including leading future private financing rounds
Part of the responsibility we have to our customers in dramatically accelerating the pace of research is being able to leverage the insights we have for their good
The company held cash and shortterm investments of about 128 million at the end of June and expects to record around 75 million in revenue from Ibsrela sales this year
Gritstone wins ʻProject NextGenʼ funding to run head-to-head COVID vaccine study. The planned trial would enroll 10000 people and compare Gritstoneʼs experimental mRNA vaccine against an approved coronavirus shot
NonGAAP research and development expenses are defined by the Company as GAAP research and development expenses excluding depreciation and amortization expense stockbased compensation expense and other items
ProQR initially had high hopes for entering the ophthalmology business and advanced two drugs called sepofarsen and ultevursen past earlystage research
But Agenus dwindling cash reserves have left it without the resources to fund other work. The companys partnered research isnt affected by the restructuring, but after speaking with management Phipps added that other inhouse programs will be reevaluated in six to 12 months to determine which might be restarted following what the company assumes will be a potential influx of capital in the coming months
In July Neumora announced results showing that patients given Neumoras oral treatment experienced meaningful improvements in depression symptoms
It has also launched new efforts to bring more drug discovery research into its orbit
In May the company announced plans to cut 65 of its workforce as well as pare back development of its lead drug candidate milademetan after it failed in Phase 3 testing. Rain shares dropped sharply following the news
Results from the Phase 1 trial could come by the end of the year
The ongoing valoctocogene roxaparvovec clinical trials will continue while BioMarin is exploring next steps to obtain approval
The startup is scheduled to discuss its first scientific data in an oral presentation at the European Society for Medical Oncology Congress in October. Precede will simultaneously publish the data in a medical journal and platform for research use enabling them to see if the technology can reveal the vulnerability of tumors to certain mechanisms of action
Emerging biotech Bioluminescence Ventures debuts with 477M to speed up drug discovery Led by former Andreessen Horowitz investor Kouki Harasaki Bioluminescence is laserfocused on cutting down the amount of time it takes to develop new medicines
The company which operates in China and the US through subsidiaries owned by a Cayman Islands holding company will report findings next year. It claims it could file for an accelerated approval afterwards if results are positive
The reinvention wont surprise anyone who has paid close attention to the companys moves since it launched in 2006 such as when it hired RD veteran Richard Scheller as chief scientific officer in 2015 or when it got an OK in 2017 to market the first FDAauthorized directtoconsumer tests to determine genetic predisposition to certain medical diseases or conditions or just last month when the company extended a drug discovery collaboration with GSK
The firm initially sought 300 million for its Clinical Co-Development Co-Investment Fund which was ultimately oversubscribed. With the new fund Abingworth has now raised nearly 1 billion for life sciences investing since 2021
The US approval of Bristol Myers TYK2 drug Sotyktu in psoriasis sparked a drug development race that has raised the profile of companies like Ventyx
Apellis is continuing to investigate the cases trying to reassure doctors and avoid a repeat of the pullback in use that occurred for Novartis when side effects hobbled its eye drug Beovu in 2020
The company notified the Securities and Exchange Commission earlier this year of plans to raise 175 million in equity financing
QA CEO Anne Wojcicki on turning 23andMe into a ʻfullfledged biotechʼ 23andMe always had larger aims than athome genetics testing
The agreement that the company won, which is managed by the Biomedical Advanced Research and Development Authority or BARDA, is worth up to 433 million
Now Trivenis next goal is to bring TRV509 through to a mid stage human trial. The company said it will keep its focus on that and the other two immune disease programs
To fulfill this postmarketing requirement BioMarin intends to use the ongoing openlabel extension studies compared to available natural history
Novo Nordisk parent backs cell therapy manufacturing facility as cell therapies for cancer and diabetes grow the Novo Nordisk Foundation will spend up to 136 million to build a plant at a Danish university
Histogen reaches the end of its road after hiring Roth Capital Partners in July to explore strategic alternatives. In the end no possibility existed that would enhance stockholder value said the current CEO in a statement
Instead the company will turn its focus to a followon medicine called vutrisiran which is currently being tested in a study called HELIOSB for the cardiomyopathy form of the disease and look forward to sharing topline results in early 2024 said the CEO in the company's statement
Sarepta executes licensing agreement for gene therapy program from Nationwide Childrens Hospital to treat limbgirdle muscular dystrophy type 2A Today Sarepta announced that upon completion of a number of preclinical and safety studies the Company executed its licensing agreement to an investigational gene therapy candidate calpain 3 CAPN3 to treat limbgirdle muscular dystrophy type 2A LGMD2A
For PTC the deal offers a chance to clear a significant overhang on the stock Cantor Fitzgerald analyst Kristen Kluska wrote in a note to clients
The Series C funds will give the company enough cash for several years paving the way for future clinical testing of GB0669 and other drugs to emerge from a pipeline that now includes 17 research programs
The European Commission decision on the Companys MAA is anticipated during the fourth quarter of 2023
The biotechnology company is also closing its current headquarters in Brisbane California early next year and transitioning to a location in Richmond California
Forward Looking Statements This press release contains forward looking statements that involve substantial risks and uncertainties
The biotechs research focuses on autoantibodies  proteins created by the immune system that attack the bodys own tissues
We are committed to sharing all learnings from this trial with the Duchenne community and hope that there are insights that may help future efforts to develop treatments for this devastating disease the CEO said in a statement
The company's most recent round was led by Sofinnova Investments with participation from new and existing investors such as Qatar Investment Authority Westlake Village BioPartners and OrbiMed
Romes lead drug candidate targets a virallike protein known as the LINE1 reverse transcriptase which when activated appears involved in cellular signaling that drives autoimmune diseases like lupus
Cargo announced it had raised a 200 million Series A round in March backed by notable biotech investors such as Samsara BioCapital and Third Rock Ventures
Alexion will have the option to license potential targets to advance them through the development and commercialization
Sarepta announced positive 12week expression and safety results for SRP9001103 the first results from a clinical trial using SRP9001 commercially representative material In May 2021 the Company announced results from the first 11 participants enrolled in Study SRP9001103 an openlabel study known as ENDEAVOR being conducted in partnership with Roche
The FDA based its decision to expand use of Neurocrine drug based on results from two clinical trials one of which remains ongoing. The studies have shown the severity of chorea significantly improves in patients who receive Ingrezza for a few months
More recently Nido Biosciences emerged from stealth mode with $109 million in funding. The startup was incubated by venture firm 5AM Ventures and attracted investment from Eli Lilly among others
The US FDA cleared the IND application for TSHA102 in pediatric patients with Rett syndrome and the Company expects to dose the first pediatric patient in the first quarter of 2024
Palforzia never caught on however and sales remained slow
Neurocrines drug will now compete more directly with Austedo a Teva Pharmaceutical medicine that like Ingrezza is approved to treat chorea as well as another neurological condition called tardive dyskinesia
In fact there are some benefits obviously for convenience and for the waits for infusion centers Hersch noted too the enthusiasm for subcutaneous Leqembi from investigators and patients in the study
In 2018 the FDA approved Onpattro to treat the polyneuropathy form of the condition which is less common than the cardiomyopathy type
News of the deal with MeiraGTx gave a much greater boost to that company sending shares in the biotech higher by nearly half at one point Monday morning
By looking for common epigenetic changes rather than focus solely on genetic biomarkers the startup aims to overcome challenges related to the genetic divergence and evolution of tumors
The FDA is currently reviewing an ex vivo treatment from Vertex Pharmaceuticals and CRISPR Therapeutics that could become the first CRISPRbased medicine approved by the agency
The venture capital group is owned by investment firm Carlyle and has offices in London Boston and Menlo Park California
The company maintains that the two forms of its drug have similar ARIA rates
Called resmetirom, the drug could become the first treatment approved in the US for the fatty liver disease which is thought to affect millions of people
Risks regarding our business are described in detail in our Securities and Exchange Commission SEC filings including in our Annual Report on Form 10K for the fullyear ended December 31 2022 and our Quarterly Report on Form 10Q for the quarter ended March 31 2023  both of which are available on the SECs website at wwwsecgov
The primary endpoint of APOLLOB is the change from baseline in the 6MWT at 12 months compared to placebo
A cohort of startups such as Arvinas and Magnet Biomedicine are also dedicated to this field of research. Rocheowned Genentech is the latest to invest in the space. In a deal announced Wednesday  Genentech will pay drugmaker Orionis Biosciences 47 million upfront in a research collaboration conditions
85 of participants in the treatment group experienced at least one treatmentrelated adverse event compared to 43 in the placebo group
Patients with Alzheimers disease may have another treatment option in the nottoodistant future as newly released data appear to support a more convenient version of the closely watched medicine Leqembi
I am delighted that VOXZOGO is now approved for younger children where we hope to have potentially greater impact by starting treatment earlier and as a result a much longer treatment window
Approximately 1 in 10000 people have hemophilia A102023 715 PM US Food and Drug Administration Approves BioMarins ROCTAVIAN valoctocogene roxaparvovecrvox the First and Only Gene Thera httpsinvestorsbiomarincom20230629USFoodandDrugAdministrationApprovesBioMarinsROCTAVIANTMvaloctocogeneroxaparvovecrvoxtheF 611People living with hemophilia A lack sufficient functioning FVIII protein to help their blood clot and are at risk for painful andor potentially lifethreatening bleeds from even modest injuries
You may also report side effects to BioMarin at 18669066100102023 715 PM US Food and Drug Administration Approves BioMarins ROCTAVIAN valoctocogene roxaparvovecrvox the First and Only Gene Thera httpsinvestorsbiomarincom20230629USFoodandDrugAdministrationApprovesBioMarinsROCTAVIANTMvaloctocogeneroxaparvovecrvoxtheF 911Please see the ROCTAVIAN full Prescribing Information for additional Important Safety Information
Stockholders are urged not to place undue reliance on forwardlooking statements which speak only102023 805 PM US Food and Drug Administration Approves BioMarins VOXZOGO vosoritide for Children Under 5 Years with Achondroplasia  Oct 20  httpsinvestorsbiomarincom20231020USFoodandDrugAdministrationApprovesBioMarinsVOXZOGORvosoritideforChildrenUnder5Yearswith 1011Navigate Our MotivationAbout Our Historyas of the date hereof
Tissuebased tests which Precede said are often poorly performed or not performed at all are required to show if a patient is eligible for treatment with many therapies
Ascendis is headquartered in Copenhagen Denmark and has addi tional facilities in Germany  Heidelberg Berlin and Munich  and the United States  Palo Alto and Redwood City California  and Princeton New Jersey
In addition any forwardlooking statements represent Alnylams views only as of today and should not be relied upon as representing its views as of any subsequent date
There are two dierent types of ATTR amyloidosis  Hereditary ATTR hATTR amyloidosis caused by a TTR gene variant and Wildtype ATTR amyloidosis wtATTR which occurs without a TTR gene variant
Please specify to the operator that you would like to join the Sarepta Second Quarter 2021 Earnings Call The conference call will be webcast live under the investor relations section of Sareptas website at wwwsareptacom and will be archived there following the call for 90 days
Results from the postpartum study were generally positive showing the medicine quickly and significantly improved depressive symptoms