daniel-gordon/biopharma-dive huggingface dataset:
We have two focus areas one being other new emerging modalities which would help us unlock new disease pathways or make medicines with a differentiated profile
Because of the small numbers of patients these represent crude frequencies that may not reflect the frequencies observed in practice
Airna is part of a recent cohort of biotechnology companies trying to develop a treatment for AATD
The Bar Harbor Mainebased Jackson or Jax  a top supplier of lab mice  helped develop an animal model Actio is using to study a target called TRPV4
The uptick in investment comes as new drugs for Alzheimers disease and ALS  or amyotrophic lateral sclerosis have broken through with highprofile FDA approvals
An OrbiMedbacked biotech launches with 85M and plans for a new kind of DNA medicine Rampart Biosciences is working on an experimental treatment to compete with Alexion Pharmaceuticalsʼ Strensiq which treats hypophosphatasia
Potential approval is another in a series of setbacks for Idorsia whose shares are trading at alltime lows
In a statement, the head of research described the deal as highly synergistic to his companys efforts in targeting immune dysregulation within cancer and inflammatory diseases. The deal follows about three months after Gilead acquired another San Diegobased startup XinThera  for its earlystage cancer and immune drugs
By the number of employees affected the planned reduction is among the most sizable within the biotech sector so far this year
As a result formal statistical testing was not performed on the final two composite endpoints which were not powered for statistical significance given the short duration of the study  allcause mortality and frequency of allcause hospitalizations and urgent heart failure visits in patients not on tafamidis at baseline nominal pvalue 09888 and in the overall population nominal pvalue 05609
A platform that does not produce great products does not help humanity in a profound way
In September the European Medicines Agencys EMA Committee for Medicinal Products for Human Use CHMP adopted a positive opinion recommending marketing authorization to expand the indication for VOXZOGO for injection to treat children with achondroplasia aged 4 months and older whose epiphyses are not closed
The new Series B funds will go toward advancing the LINE1 candidate into clinical testing the company said
We had been preparing for an advisory committee meeting from the filing of the BLA in the fall of 2022
Each offshoot is given funding to research a single experimental therapy for a range of potential diseases
However the Company does not exclude reimbursement of costs by Roche from its nonGAAP results
Positive readouts in any of those trials could have helped turn FibroGen around. Instead the company has reported a succession of less-than-ideal results
In Apellis case however its share price decline has been sudden spurred by reports of a rare but potentially blinding side effect in a handful of patients treated with Syfovre since its February approval
Kidney toxicity was observed in animals who received golodirsen
For all periods presented collaboration revenue primarily relates to the F HoffmanLa Roche Ltd Roche collaboration arrangement
Various important factors could cause actual results or events to differ materially from the forward looking statements that Ascendis makes including the following dependence on third party manufacturers distributors and service providers for Ascendis products and product candidates unforeseen safety or efficacy results in its development programs or on market products unforeseen expenses related to commercialization of any approved Ascendis products expenses related to Ascendis development programs unforeseen selling general an d administrative expenses other research and development expenses and Ascendis business generally delays in the development of its programs related to manufacturing regulatory requirements speed of patient recruitment or other unforeseen delays Ascen dis ability to obtain additional funding if needed to support its business activities  the impact of international economic political legal compliance social and business factors including inflation and the effects on its business from the worldwi de COVID 19 pandemic and the ongoing conflict in the region surrounding Ukraine and Russia
About ONPATTRO Patisiran ONPATTRO is an RNAi therapeutic that is approved in the United States and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults
Ardelyx has won Food and Drug Administration approval for a new kind of kidney disease drug announcing Tuesday the agencys clearance of Xphozah after a yearslong regulatory journey
A live audio webcast of the call will be available on the Investors section of the Companys website at wwwalnylamcomevents httpsctsbusinesswirecomctCT
About MOMENTUM Study SRP5051201 MOMENTUM is a multiarm ascending dose study designed to identify the maximum tolerated dose of SRP5051 infused monthly
After 17 years were getting closer and closer with our FDA authorizations and our general acceptance
In announcing the deal Embark cited a novel target that suppresses appetite increases energy expenditure and enhances insulin sensitivity In previous disclosures Embark said it had identified a peptide drug for cardiovascular and metabolic disease and a small molecule energy expenditure activator that it was investigating for obesity
For more information about VOXZOGO please visit wwwvoxzogocom
In addition we expect to submit an application for an EAP in Germany and open it for enrollment in the second quarter of this year
In addition it is approved in Brazil in children who are 6 months and older with open growth plates as well as in Australia in children with achondroplasia who are 2 years of age and older with open growth plates
For TSHA120 in GAN our new comprehensive data analysis utilizing the Disease Progression Model DPM was submitted to the FDA and we plan to review the potential regulatory pathway for TSHA120 with the Agency expected in the third quarter Mr Nolan continued Our successful completion of a 150 million PIPE from toptier investors significantly bolsters our balance sheet and we believe highlights the enthusiasm for our TSHA102 program and the early clinical readout of the first patient treated in the REVEAL trial
Yet the medicine has become a flash point in an at times contentious debate over how accommodating the agency should be in reviewing drugs of ALS a devastating neurological disease that typically leads to death three to five years after symptoms begin
Dystrophin expression was measured by western blot
For a detailed description of risks and uncertainties we face we encourage you to review our SEC filings
Wecaution investors not to place considerable reliance on the forwardlooking statements contained in this press release
You are encouraged to report negative side effects of prescription drugs to the FDA
In the clinical trials  TransCon PTH has been generally well tolerated with no discontinuations related to study drug
Theres no bigger healthcare problem than heart disease the number one cause of death worldwide which has gone decades without any real pharmacological innovation wrote the CEO, who served as Novartis chief medical officer in a post on LinkedIn
The Series C fundraising saw participation from MapLights existing backers as well as new ones like 5AM Ventures Cowen Healthcare Investments and Novo Holdings the parent group of Novo Nordisk which led the round
Like SRP9001 Sareptas lead investigational gene transfer therapy for Duchenne and the Companys five other LGMD programs the LGMD2A program uses the AAVrh74 vector designed to systematically and robustly deliver treatment to skeletal muscle making it an ideal candidate to treat muscle disease
A significant discount helped Orchard secure coverage in England and Wales  and investors are likely looking toward US approval for it to achieve sales expectations which Stifel analyst Dae Gon Ha pegged at 200 million in 2035
Eisai as well as Wall Street analysts also expect a subcutaneous formulation will help increase uptake and sales of Leqembi
This is not the first time the FDA does not not follow panel recommendation but we can't think about many examples where the drugs primary endpoint is hit the advisory committee votes in favor and the FDA still denies approval wrote Luca Issi an analyst at RBC Capital Markets in a note to clients
We are highly encouraged by the initial data for TSHA102 and are focused on continuing to explore its therapeutic potential with the dosing of the second patient expected in the third quarter
Bayer reports positive early data for Parkinsonʼs cell therapy. The therapy developed by Bayerʼs Bluerock Therapeutics subsidiary appeared safe and well-tolerated in a 12 person clinical trial
We extend our sincere appreciation to the families clinicians FDA presenters and committee members who participated in todays panel and to all those who provided input and comments both in the written record and in the open public hearing SRP9001 is intended to treat the underlying cause of Duchenne which is characterized by mutations in the dystrophin gene that results in the lack of dystrophin protein
About Achondroplasia Achondroplasia the most common form of skeletal dysplasia leading to disproportionate short stature in humans is characterized by slowing of endochondral ossification which results in disproportionate short stature and disordered architecture in the long bones spine face and base of the skull
The increase in research and development expenses primarily reflects the following 437 million increase in manufacturing expenses primarily due to a continuing rampup of the Companys gene therapy programs 198 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 144 million increase in upfront milestone and other expenses primarily due to a 287 million increase of an accrued sublicense fee to Nationwide and 70 million of expense incurred as a result of milestone achievements in certain research and license agreements during the six months ended June 30 2021 offset by 93 million of expense related to sublicense payments accrued to Nationwide and 120 million of upfront payments as a result of the execution of certain research and license agreements during the same period of 2020 129 million increase in research and other primarily driven by an increase in sponsored research with academic institutions during the six months ended June 30 2021 80 million increase in facility and technologyrelated expenses due to the Companys continuing expansion efforts 78 million increase in preclinical expenses primarily due to an increase of toxicology studies in the Companys PPMO platforms 62 million increase in compensation and other personnel expenses primarily due to a net increase in headcount 36 million increase in stockbased compensation expense primarily driven by increases in headcount and changes in stock price 25 million increase in collaboration cost sharing with Genethon on its microdystrophin drug candidate 27 million decrease in professional service expenses primarily due to a decrease in reliance on thirdparty research and development contractors and 60 million increase in the offset to expense associated with a collaboration reimbursement from Roche primarily due to continuing development of the Companys SRP9001 microdystrophin gene therapy
On Wednesday Takeda released new midstage study data for its TYK2 drug in psoriatic arthritis building on results earlier this year in psoriasis
These risks and uncertainties include among others BioMarins success in the commercialization of102023 715 PM US Food and Drug Administration Approves BioMarins ROCTAVIAN valoctocogene roxaparvovecrvox the First and Only Gene Thera httpsinvestorsbiomarincom20230629USFoodandDrugAdministrationApprovesBioMarinsROCTAVIANTMvaloctocogeneroxaparvovecrvoxthe 1011Navigate AboutROCTAVIAN including achieving adequate market share and reimbursement levels whether ROCTAVIAN will have the impacts and benefits as anticipated the results and timing of current and planned preclinical studies and clinical trials of ROCTAVIAN and the release of data from those trials including continued monitoring of the participants in the clinical trials and postapproval studies BioMarins ability to successfully manufacture ROCTAVIAN for the clinical trials and commercially the content and timing of decisions by the FDA EU health authorities and other regulatory authorities regarding ROCTAVIAN and those factors detailed in BioMarins filings with the Securities and Exchange Commission SEC including without limitation the factors contained under the caption Risk Factors in BioMarins Quarterly Report on Form 10Q for the quarter year ended March 31 2023 as such factors may be updated by any subsequent reports
So far at least 12 biotech companies have announced layoffs in the fourth quarter. About 30 did in both the second quarter and the third while 59 drugmakers cut their workforces during the first three months of 2023. Yet biotech companies are still hiring and by some analyses the sector is growing overall
Blending technology and biomedicine has untapped potential and its an exciting time to be at the nexus of these two powerful forces said Vijay Pande a founding general partner of Andreessen Horowitzs Bio  Health practice and a coinventor of some of Genesis technology
News of the financing which will help fund Tayshas development of a gene therapy for Rett syndrome sent shares in the company soaring Monday morning
Its a typically ambitious agenda for Moderna which says it aims to change the world. But a lot has to go right for the company to deliver on plans that few if any companies of its size have pulled off
The deal may have been one of Seagens last as an independent company. Pfizer is working toward completing its acquisition although it has been subjected to some close scrutiny from the Federal Trade Commission
Alternatively use a laboratory test that does not use the reagent pyrogallol red as this reagent has the potential to cross react with any AMONDYS 45 that is excreted in the urine and thus lead to a false positive result for urine protein
Muscle dystrophin levels demonstrated a mean of 705 baseline 128 muscle fibers expressing microdystrophin at 12weeks with a mean intensity at the sarcolemma of 1169 baseline 410 compared to normal biopsies as measured by immunofluorescence
Patients were randomized 11 to receive 03 mgkg of patisiran or placebo intravenously administered every three weeks over a 12month doubleblind treatment period
The safety and efficacy of TransCon PTH have not been established and TransCon PTH is not currently approved by the FDA
In December 2019 the Company announced a licensing agreement granting Roche the exclusive right to launch and commercialize SRP9001 outside the United States
In this regard we are very disappointed that the randomization process resulted in a significant imbalance in baseline NSAA scores between the active and placebo cohorts of the participants ages 67 making the 67 age groups noncomparable and likely substantially contributing to the inability to achieve statistical significance said Doug Ingram president and chief executive officer Sarepta
The 210 million financing announced Wednesday was led by new investor GV which is joining a broad base of backers including venture firms Atlas Venture and SR One
The venture firm also partnered with Pfizer in July as part of a recent Pioneering Medicines initiative
Gene therapy developer and manufacturer Forge Biologics has agreed to be acquired by Ajinomoto Co the companies announced Monday. The Japanese multinational food and biotechnology company will pay 554 million to Forge shareholders. On a cashfree and debtfree basis the deal values Forge at 620 million Ajinomoto said
Cell therapy developer Sana turns again to layoffs. A 29% reduction in workforce is meant to extend Sanaʼs cash runway further into 2025 as the company advances its lead research programs
Physicians have effective ways of treating many tumors identified at those early stages but lack the means to diagnose cancers before they progress. Harbinger is running its 10000 HH study to show its test can address the unmet screening need
In December 2022 the FDA allowed Ascendis to initiate a US EAP for TransCon PTH for eligible adults with hypoparathyroidism previously treated with parathyroid hormone
ORM6151 is also being developed for highrisk myelodysplastic syndromes  in which immature blood cells in the bone marrow dont mature into healthy blood cells
The company said it has invested in more than 185 biotech and pharma companies over the past 50 years
To reduce the risk of IRRs patients should receive premedication with a corticosteroid acetaminophen and antihistamines H1 and H2 blockers at least 60 minutes prior to ONPATTRO infusion
Two years ago on the heels of raising 112 million in Series B venture financing  the biotechnology firm struck a deal with Roche focused on applying RNA editing technology to neurological diseases
Earlier this month RayzeBio and Neumora Therapeutics successfully pulled off lucrative IPOs amid a flurry of activity that also saw chip designer ARM and the grocery delivery company Instacart price offerings
Exploratory endpoints included cardiac biomarkers and various imaging tools to further characterize the potential burden of cardiac involvement in these patients
We intend to continue to move forward with diligence and urgency to generate the evidence necessary to bring SRP9001 to waiting Duchenne patients around the worldSarepta will host an investor webcast and conference call on Thursday Jan 7 2021 at 430 pm Eastern Time to discuss these results
In addition to the comfort level the FDA has after reviewing previous Bluebird applications the agency may also be more familiar with the latest data because of a protracted filing process for lovocel  Stifel analyst Benjamin Burnett said in a note to clients
While the generalists have been increasingly more active its still very much of a market thats driven by those special healthcare specialists investors Private companies should be aware of this dynamic and plan accordingly executives said
Estimates cited by Sanofi and Teva hold that about 10 million people worldwide have the condition
Forge also operates a 200000square foot manufacturing facility in Columbus Ohio employing over 300 people to develop viral vectors and plasmids
Differences between studies mean cross-trial comparisons can be unreliable but the results suggest the Harbinger test may be competitive. Grail used a higher 995 specificity in a key study  minimizing false positives and making comparison to Harbinger challenging but published results at 95 specificity early in development. At 95 specificity Grails sensitivity for stage 1 to 3 cancers ranged from 56 to 90
At Wednesdays meeting BrainStorm and the FDA will each make presentations as will a director of a stem cell research center who was invited to speak on manufacturing issues. Advisers will also hear from patients and patient advocates during an open public hearing
Triveni is preparing to advance TRV509 a preclinical antibody targeting two enzymes expressed in the skin into human trials for treating atopic dermatitis or eczema
Challenges in the capital markets and the broad sentiment shift has in our view disproportionately impacted precommercial companies particularly those with extensive platforms in the rare disease space wrote Stifel analyst Dae Gon Ha in a Tuesday note to clients
Patisiran also demonstrated an encouraging safety and tolerability profile with deaths numerically favoring the patisiran arm
More than 80 of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation
In February 2020 the Company entered into an agreement to sell the PRV it received from the FDA in connection with the approval of VYONDYS 53
It is our understanding that as one of the first gene therapy BLAs founded on a surrogate endpoint the advisory committee will primarily relate to the totality of evidence supporting the conclusion that the SRP 9001 dystrophin is reasonably likely to predict clinical benefit the standard for accelerated approval said Doug Ingram  president and chief executive officer Sarepta
With the 60 million fee from Seagen Nurix said it has enough cash to support its own operations through the second quarter of 2025. Nurix shares rose by about 10 in morning trading Thursday
The results come from one of several latestage studies of the medicine izokibep in various inflammatory diseases
Superluminal said it expects to produce a lead experimental drug candidate in the near term among the first funds a startup raises from investors
Specifically Seraxis is focusing on the production of insulin to control glucose levels. The bitoech says its cells are as potent as ones normally found in a healthy pancreas and have been able to reverse diabetes in rats and mice
The safety of patients remains our highest priority and since our NDA submission no new safety signals have been observed to date in our ongoing TransCon PTH Phase 2 and Phase 3 clinical trials or in our Expanded Access Program EAP  program and these programs continue unchanged  Knowing the serious unmet medical need that patients with hypoparathyroidism face we will continue to make TransCon PTH available to patients participating in these ongoing clinical trials  as well as to physicians through our US EAP which remains open for enroll ment for eligible adult patients previously treated with parathyroid hormone  continued Mikkelsen
Huntingtons patients have had access to a couple other chorea treatments. Xenazine approved in 2008 and Austedo cleared for use in 2017 are believed to work in a way similar to Ingrezza. However some analysts argue that Neurocrines product may hold certain advantages
Although kidney toxicity was not observed in the clinical studies with VYONDYS 53 the clinical experience with VYONDYS 53 is limited and kidney toxicity including potentially fatal glomerulonephritis has been observed after administration of some antisense oligonucleotides
Edward PagliaruloBioPharma Dive Despite optimism for a stronger investment climate this year many of the hurdles that faced young biotechnology companies in late 2022 have endured through 2023
These forwardlooking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements
By incorporating diffusion models our platform is designing novel medicines that transcend the boundaries of what is possible experimentally Vigneault said in a Sept 7 statement
We would like to thank CBER for moving expeditiously to schedule the advisory committee in advance of our May 29 2023  regulatory action date once the change in decision was made Conference call details At 430 pm ET on March 16 2023  Sarepta will host a conference call and webcast to discuss this update
Forge is somewhat unusual in running a CDMO business as well as developing its own therapies
The company also aims to identify other autoantibodies that drive neuropsychiatric diseases and develop new drugs to block them
The company which filed plans to go public Thursday previously raised 418 million in private funding to advance its lead drug candidate for neuroendocrine tumors into latestage clinical testing
Patient Support for Accessing ROCTAVIAN To reach a BioMarin RareConnections case manager please call 1833ROCTAVIAN 1833 7628284 or email roctaviansupportbiomarinrareconnectionscom
The study will enroll up to 24 patients both ambulant and nonambulant between the ages of 7 to 21 at sites in the US Canada Australia and European Union
Under the deal which closed on signing Stallergenes Greer will make unspecified milestone and royalty payments to Nestlé. The companies did not disclose whether any upfront cash is involved
This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53