daniel-gordon/biopharma-dive huggingface dataset:
By early December the Food and Drug Administration will decide on approval of a genetic medicine for sickle cell disease potentially offering people with the rare blood condition a new soughtafter treatment option
The Companys programs and research focus span several therapeutic modalities including RNA gene therapy and gene editing
Seraxis also announced Thursday the hiring of Ted Hibben as chief corporate development officer
Novo is beefing up its diabetes and weightloss pipeline where it has around nine novel projects in clinical trials
In a statement Tuesday Fulcrum said it plans to restart patient enrollment in a Phase 1b trial of the drug known as FTX6058 and one of two medicines the Cambridge Massachusettsbased biotechnology company has in human testing
After long journey Ardelyx gets FDA OK for kidney disease drug The biotech appealed a 2021 rejection of the therapy now called Xphozah and late last year secured the backing of FDA advisers for its eventual approval
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
VectorYs experimental drug now in preclinical testing is meant to target aggregates of a misfolded protein called TDP43. Clearing these defective protein clumps from the cytoplasm of neurons could help preserve brain cells according to the company
The drug ingredient in Xphozah tenapanor is already approved for irritable bowel syndrome and is sold as Ibsrela for that use
Abrahams and his team for instance estimate around 240 million in annual peak sales for Zurzuvae in postpartum depression
The new company said it will work with socalled  endless RNA  programmable nanoparticles and artificial intelligence tools to build a new class of medicines. John Mendlein, a former executive at Moderna, will serve as the companys executive chairman
In the letter the FDA cited concerns related to the manufacturing control strategy for variability of delivered dose in the TransCon PTH drugdevice combination product
At 281M Cargos offering is also the second largest by a cancer drugmaker in 2023 behind RayzeBios 311 million IPO in September
The FDA did not express concern about the clinical data submitted as part of the NDA package and no new preclinical studies or Phase 3 clinical trials to evaluate safety or efficacy were requested in the letter
Cargo Therapeutics priced an initial stock offering on Thursday bringing in 281 million to fund the biotechnology startups plans for developing better cancer drugs
Genetic medicines have especially in the last decade proven they can dramatically benefit patients with certain inherited conditions. Often though their effectiveness has been hampered by limitations of delivery
The Cambridge Massachusettsbased biotechnology company said Thursday it will shed about 40 of its workforce to right size the organization and make room for new employees to help sell its treatment for postpartum depression
The Company also believes these nonGAAP measures increase comparability of periodtoperiod results and are useful to investors as they provide a similar basis for evaluating the Companys performance as is applied by management
Also on Monday Taysha reported initial clinical results from the first Rett patient treated with the companys therapy TSHA102 in a Phase 12 clinical trial. According to the company the therapy was well-tolerated by the patient with no serious adverse events reported through six weeks post treatment
Last month Neumora filed plans to go public  bucking a prolonged downturn in the biotech stock market thats kept many young drugmakers from pursuing an initial public offering
The company having seen sales decline on its top products ended 2022 with total revenue down roughly 7 to 102 billion
The company is developing a treatment for acute heart attacks building on the work of Mauro Giacca a Kings College London professor who identified cardioprotective proteins that could help prevent the loss of heart cells
Established investors have reloaded their coffers too, SR One brought in a 600 million raise in March and last week OrbiMed announced 43 billion in new money across three investment funds
The candidate was developed by the Abigail Wexner Research Institute at Nationwide Childrens Hospital
A replay of the webcast will be availabl e on this section of our website shortly after conclusion of the event for 30 days
Based on the results of this trial together with evidence from the adequate and well controlled Phase 3 study in pediatric patients aged 5 years and older Study 111 301 safety and effectiveness of VOXZOGO have been established in pediatric patients of all ages for the improvement in linear growth in children with achondroplasia with open epiphyses
This spring BenevolentAI cut staff and restructured its research in order to preserve cash
The company is working on two other programs but has not disclosed targets for either
The company will update its cash position when it announces third quarter earnings
At the time Sangamo said it would focus a Fabry treatment nearing Phase 3 testing its regulatory T cell therapy research and a neurology drug pipeline centered on epigenetic regulation. Now the company is deferring any further investment into its Fabry or Treg programs until it finds a partner or other outside funding
The company also exited research into eye diseases and claims its been refocusing investments in gene therapy
This102023 805 PM US Food and Drug Administration Approves BioMarins VOXZOGO vosoritide for Children Under 5 Years with Achondroplasia  Oct 20  httpsinvestorsbiomarincom20231020USFoodandDrugAdministrationApprovesBioMarinsVOXZOGORvosoritideforChildrenUnder5Yearswith 211expanded indication now includes children of all ages with open growth plates
Tell your doctor about all of the patients medical conditions including If the patient has heart disease cardiac or vascular disease or if the patient is on blood pressure medicine antihypertensive medicine102023 805 PM US Food and Drug Administration Approves BioMarins VOXZOGO vosoritide for Children Under 5 Years with Achondroplasia  Oct 20  httpsinvestorsbiomarincom20231020USFoodandDrugAdministrationApprovesBioMarinsVOXZOGORvosoritideforChildrenUnder5Yearswith 811If the patient has kidney problems or renal impairment
In 2021 its venture arm led a 105 million funding round for Senti Biosciences  a Californiabased biotechnology company trying to create off-the-shelf cellular medicines for cancer
Across all sectors the top seed rounds since 2018 have generally hovered anywhere between 5 million and 9 million according to data from Pitchbook . The funds will help Superluminal go after GPCRs which have long been on drugmakers radars but recently become a hot area of startup investment as well
Gritstone Bio, a cancer and infectious disease drug developer has won a contract from the US government to test its experimental COVID vaccine against an approved coronavirus shot in a headto head study. The contract announced Wednesday  is part of the Project NextGen an initiative launched by the US Department of Health and Human Services to bring new COVID drugs and vaccines to market. Gritstone will conduct a doubleblind Phase 2b study of its self amplifying messenger RNA vaccine in a study of 10000 people
As part of the deal Idorsia will pay JJ as much as 306 million Swiss francs or about 343 million to reacquire rights to the drug. The total amount depends on regulatory approvals potential future licenses and commercial sales
For the six months ended June 30 2021 and 2020 other expense net was approximately 317 million and 199 million respectively
Launched just under two years ago Tentarix specializes in designing biologic drugs that activate only in specific cellular conditions  an attribute it claims could allow it to more selectively target immune cells involved in disease while avoiding others
The overall safety profile of VOXZOGO in children under 5 years of age was similar to that seen in older children102023 805 PM US Food and Drug Administration Approves BioMarins VOXZOGO vosoritide for Children Under 5 Years with Achondroplasia  Oct 20  httpsinvestorsbiomarincom20231020USFoodandDrugAdministrationApprovesBioMarinsVOXZOGORvosoritideforChildrenUnder5Yearswith 311Data from an openlabel longterm Phase 2 extension study was recently presented at the 2023 European Society for Paediatric Endocrinology Meeting in September
One of the most common fatal genetic disorders DMD affects approximately one in every 3500  5000 male births worldwide
We have also been running surveys of clinicians  and we now can see that physicians are interested in genetic information
We remain committed to the hemophilia community and to leading the way to the first ever gene therapy in hemophilia A said JeanJacques Bienaimé Chairman and Chief Executive Officer of BioMarin
We also received FDA clearance to initiate clinical development of TSHA102 in pediatric patients in the US and have submitted a CTA to the MHRA for TSHA102 in pediatric patients with Rett syndrome which will expand our clinical evaluation to children with earlier stages of disease progression said Sean P Nolan  Chairman and Chief Executive Officer of Taysha
The technology to scan and study this part of DNA wasnt available until the past decade as advances in longread sequencing and later machine learning techniques gave scientists needed tools Kapeller said
The FDA agreed to review the companies approval application in February and by early August had decided to clear the drug for use in postpartum depression but reject it for major depressive disorder
In addition to the upfront payment to Beam Lilly will invest another 50 million in Beam equity and commit to pay 350 million more if certain milestones are met. The transaction is one of the largest struck in gene editing according to Beam CEO John Evans who last year led his company into a deal with Pfizer
The net proceeds from the private placement combined with the current cash and cash equivalents are expected to fund its operational plans and capital requirements into the third quarter of 2025
Please specify to the operator that you would like to join the Microdystrophin SRP9001 Study 102 Topline Results Call
Initial data from the first adult patient in Canada with severe disease dosed with TSHA102 is encouraging and we expect to dose the second patient in our ongoing REVEAL Phase 12 adult trial in the current quarter
Under the FDAs labeling Xphozah can only be used in people who dont respond well to phosphate binders or who cant take any dose of phosphate binder therapy
The overall safety profile of patisiran during the 12month doubleblind period was encouraging
Such exploratory analyses provide little confidence on which to base regulatory decisions and though they may serve to generate hypotheses for testing in future trials cannot compensate for negative results in a well-controlled study FDA staff wrote
Known as chorea this disorder is characterized by sudden involuntary muscle movements and it can be caused by a variety of diseases or sometimes medications like levodopa
As the disease progresses muscle weakness in the lower limbs spreads to the arms neck and other areas
Elevated phosphorus levels can remove calcium from the body leading to low calcium levels and other problems. However the current drugs work the same way blocking the absorption of phosphorus by binding to the mineral in the gut.
Biotechnology company creator Flagship Pioneering is expanding its global footprint announcing Tuesday the establishment of a regional hub in Singapore. The move follows Flagship opening an office in London earlier this year and is meant to help the venture firm scale its companies and ink new partnerships in the region
The study dubbed Heart1 has been underway in the UK and New Zealand since last summer  and Verve plans to present the first data from it at a medical meeting next month
The company is developing several treatments for metabolic conditions like obesity and diabetes. Unlike the GLP1 drugs now popular thanks to the success of Ozempic Wegovy and Mounjaro its experimental candidates are aimed at other drug targets
Idorsia was formed through a spinoff after JJs 30 billion buyout of Actelion in 2017. A few months later JJ acquired rights to aprocitentan  offering an initial payment of 230 million development funds and potential royalties
But markets have soured since then and companies without clinical data are finding it harder to pull off initial public offerings
Verve plans to advance a second PCSK9 program and the ANGPTL3 candidate into clinical testing next year. According to Evans Beam would have made an optin decision on its own regarding the Verve programs possibly by next year. But Lilly expressed interest in getting more involved spurring Beam to choose to sell its rights
Use of NonGAAP Measures In addition to the GAAP financial measures set forth in this press release the Company has included certain nonGAAP measurements
Among the other venture firms participating in Trivenis Series A round are OrbiMed, the private equity arm of Viking Global Investors, Invus Polaris Partners, and Alexandria Venture Investments along with other unnamed investors. The cash raised will extend the companys financial runway into 2027 said the CEO
Sails launch ends a difficult period for Laronde a buzzy Flagship startup that brought in nearly 500 million to fund development of Laronde drew investor interest for its work creating synthetic circular versions of the nucleic acid that could carry protein blueprints into cells
Roche bets on Ionis RNA medicines with neuroscience deal. The Swiss drugmaker is restocking its pipeline as the pace of drug development in Alzheimerʼs and Huntingtonʼs disease picks up
Forwardlooking statements include statements concerning the potential benefits of FTD for TSHA102 the potential of our product candidates including the reproducibility and durability of any favorable results initially seen in our first patient dosed in the REVEAL trial and including our preclinical product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat our research development and regulatory plans for our product candidates the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies and whether if approved these product candidates will be successfully distributed and marketed and the potential market opportunity for these product candidates
The money will be spread across three funds with 1865 billion dedicated to OrbiMed Private Investments IX 171 billion to OrbiMed Royalty and Credit Opportunities IV and 751 million to OrbiMed Asia Partners V a spokesperson said
For instance the company's lead program targets two forms of a protein receptor called IL2 as well as surface proteins found on certain T cells. The therapy is meant to work only when all targets are bound at the same time in theory limiting its action only to those cells. The approach overlaps in some respects with research on drugs known as bispecific antibodies which are meant to bring two different types of cells together
Superluminal Medicines a Bostonbased biotechnology startup said Monday it raised 33 million in seed funding to develop a pipeline of drugs focused on G proteincoupled receptors
BrainStorm cited those drugs and the FDAs concerns about their trial evidence in making its case for NurOwns approval
Reporter: How has your perceptions of the US healthcare system changed what role your company plays? CEO: Theres a lot I'm proud of with the company and one thing thats put us on the map is the whole world of direct to consumer. Prior to us you had OTC products that you could buy but there wasnt really a directtoconsumer industry for pandemic because of telemedicine and those opportunities. Also along those lines we have proven that there are aspects of healthcare like your genome where you dont necessarily have to go through a healthcare provider  our FDA authorizations are direct to consumer  that do not require precounseling or post counseling
This is related to the amortization of the inlicensed right assets recognized as a result of agreements the Company entered into with BioMarin and the University of Western Australia
Data from Tevas Phase 2 trial arent expected until next year but the company has talked up the drugs potential to be competitive
Sarepta ForwardLooking Statement This press release contains forwardlooking statements
In this circumstance theres a lot of capital people have money but theyre putting it in I would say the wrong places said the CEO of Stoke Therapeutics
Cost and Operating Expenses Cost of sales excluding amortization of inlicensed rights For the three months ended June 30 2021 cost of sales excluding amortization of inlicensed rights was 195 million compared to 133 million for the same period of 2020 an increase of 62 million
The safety and efficacy of TransCon PTH have not been established and TransCon PTH is not currently approved by the FDA or EMA
With investors like Arch and Amgen Neumora also has strong financial backing to test the IPO waters. The company pulled in 112 million in a Series B round in October
The study also included additional secondary composite outcome endpoints to be tested in a hierarchical manner
Fueled by quickly growing sales of its weightloss drug Wegovy and diabetes therapy Ozempic Novo has secured a leading position in obesity treatment which could be worth tens of billions of dollars in annual sales
PTC said at the time that it would focus on drugs that could offer a return on investment, but now its at risk of losing an approved drug
Mariana is designing its drugs to deliver alpha or beta radionuclide payloads into solid tumors. Its lead candidate dubbed MC339 is a peptidic small molecule linked to actinium that treats small cell lung cancer an aggressive form of the disease
BioPharma Dive spoke with Stilz about Novos approach to building its drug pipeline and how a traditional pharmaceutical company can create a biotechlike unit
People with hemophilia A with moderate FVIII 15 or mild FVIII 5 40 disease show a muchreduced propensity to bleed
Inject VOXZOGO 1 time every day at about the same time each day
In a statement Monday  Alnylam said the FDA had determined that the companys supporting clinical trial data did not establish the clinical meaningfulness of patisirans treatment effects for the condition known as cardiomyopathy of ATTR amyloidosis
Announced Monday the new fund is part of Abingworths clinical codevelopment strategy which it started in 2009 and has used to support 14 programs to date including with partners like AstraZeneca Eisai and Pfizer
The restructuring was a notable retreat from the companys initial plans to advance more than a dozen genetic medicine programs
As a result of the restructuring Sangamo has also trimmed its C suite eliminating the roles of Chief Operating Officer and Chief Scientific Officer
Riding high from sales of an mpox vaccine Bavarian Nordic began 2023 with one major goal in mind becoming one of the largest pureplay vaccine manufacturers in the world
Karuna submits schizophrenia drug for US approval. If cleared by the FDA the drug would oﬀer the ﬁrst new mechanism of action for treating schizophrenia in decades
Sarepta Therapeutics Announces Second Quarter 2021 Financial Results and Recent Corporate Developments 8421 Sarepta announces successful completion of endofphase2 meeting for SRP9001 microdystrophin with FDAs Office of Tissues and Advanced Therapies OTAT and plans to initiate pivotal trial SRP9001301 Study 301 or EMBARK in September of 2021 Net product sales for the second quarter of 2021 reached 1418 million a 27 increase over the same quarter of prior year In light of its overperformance Sarepta raises its fullyear product revenue guidance by nearly 30 million to between 565 million to 575 million CAMBRIDGE Mass Aug 04 2021 GLOBE NEWSWIRE  Sarepta Therapeutics Inc NASDAQSRPT the leader in precision genetic medicine for rare diseases today reported financial results for the second quarter of 2021
RayzeBio is led by Ken Song who also cofounded Ablaze Pharmaceuticals a startup focused on radiopharmaceuticals in China
The NSAA is a 17item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne
In December Lilly agreed to pay ProQR 75 million and make an equity investment to expand a development deal originally reached in 2021
Riding out the cycle In 2020 and early 2021 the success of Moderna and BioNTech in developing highly effective vaccines for COVID19 drove a surge of investment from all corners of the market into biotech
Cell therapy developer closes funding round to support diabetes program Attempting to transform diabetes treatment Seraxis has raised more than 50 million from a series of investors that includes Eli Lilly
By harnessing the natural biological process of RNAi occurring in our cells a new class of medicines known as RNAi therapeutics is now a reality
For the six months ended June 30 2021 and 2020 the Company recognized 443 million and 392 million of collaboration revenue respectively
ForwardLooking Statements This press release contains forwardlooking statements within the meaning of the Private Securities Litigation Reform Act of 1995
Syfovre is the first medicine approved by the Food and Drug Administration specifically to fight geographic atrophy  a form of vision loss triggered by agerelated macular degeneration
Like other conditions treated by gene therapies metachromatic leukodystrophys rarity has been accompanied by skyhigh prices for treatment with Orchard seeking 29 million euros per patient
The increase primarily reflects an increase in interest expense incurred on the Companys term loan debt facilities due to an increase in the outstanding balance partially offset by a reduction of interest expense incurred on the Companys convertible debt related to the adoption of ASU 202006 as well as a decrease in interest income and the amortization of investment discounts due to changes in investment mix of the Companys investment portfolio
Kinnate intends to file a New Drug Application for its cMET inhibitor KIN8741 in the fourth quarter while exploring strategic alternatives for its discontinued programs
Earlier this year Idorsia said a cardiovascular drug failed in a key clinical trial
The hope now is that these trends continue and translate into controlled clinical trials Bluerock said that based on the results seen so far it is planning a midstage study that should begin enrolling participants in the first half of next year
The rejection could hurt Alnylams financial future
Still at that same meeting advisers to the agency concluded by a 93 vote that patisirans benefits outweighed its risks in treating cardiomyopathy of ATTR amyloidosis
Neurocrines drug Ingrezza is now cleared for use in adults who have a disorder often associated with Huntingtons
For the six months ended June 30 2021 cost of sales excluding amortization of inlicensed rights was 419 million compared to 260 million for the same period of 2020 an increase of 159 million
With the subcutaneous version Eisai said there was a relatively low rate of reactions near the injection site and almost no systemic reactions
The company also could receive as much as 840 million in milestone payments over the deal along with royalties on any resulting products
Alnylam is executing on its Alnylam Px25 strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance resulting in a leading biotech profile
Last year the companys board of directors also grew with the addition of former Biogen executive Al Sandrock
Yet at about 6 apiece shares are worth less than half their 16 debut price and far below their peak of about 31 in September 2021
Some of the best known are Roivant Sciences PureTech Health Nimbus Therapeutics and BridgeBio Pharma
Apellis also plans to trim its research spending