daniel-gordon/biopharma-dive huggingface dataset:
RayzeBios private backers included Viking Global Capital VenBio Versant Ventures Samsara Biocapital and Sofinnova Investments
The theme of starting with a more homogeneous disease setting and then moving out into a broader population is certainly something thats been around for decades said Goldstein
Superluminal was cofounded by former RA Capital Management executive Ajay Yekkirala and former Evotec executive Cony Dcruz. Insight Partners Nvidia and Gaingels joined RA Capital in the seed round
Nimbus next act is led by a medicine dubbed NDI101150. Like Nimbus other medicines the drug is a small molecule
Such risks may be amplified by the impacts of the COVID19 pandemic
About SRP9001 delandistrogene moxeparvovec SRP9001 delandistrogene moxeparvovec is an investigational gene transfer therapy designed to address the underlying cause of DMD through the targeted production of functional components of dystrophin in muscle tissue
With a singular focus on developing curative medicines we aim to rapidly translate our treatments from bench to bedside
Biogen leadership has said that as part of the research overhaul there will be greater investment in the areas of immunology neuropsychiatry and rare disease. To that end in July the company announced the biggest deal in its history by unadjusted dollar terms a planned 73 billion acquisition of rare disease drugmaker Reata Pharmaceuticals
The data in major depressive disorder were more mixed however. A key study failed in 2019  leading Sage to redraw its development plans
FTD is designed to help treatments reach patients faster by facilitating the development and expediting the review of therapies with potential to address unmet medical needs for a serious or lifethreatening condition
ForwardLooking Statements This press release contains forwardlooking statements
If the patient is breastfeeding or plans to breastfeed
The event will be webcast live under the investor relations section of Sareptas website at httpsinvestorrelationssareptacomevents presentations and following the event a replay will be archived there for one year
NonGAAP selling general and administrative expenses were 540 million and 551 million for the three months ended June 30 2021 and 2020 respectively a decrease of 11 million
The drug came to Aiolos via Chinas Jiangsu Hengrui Pharmaceuticals from which the startup licensed AIO001 in August
Fresh off 4B deal Nimbus gears up for ʻthird chapterʼ with new fundraise. The Boston biotech has sold two experimental drugs  a liver disease medicine and a TYK2 inhibitor  to Gilead and Takeda respectively
But our particular approach to doing that is to develop a framework that really can effectively start with 4000 different Mendelian disease genes and pull out the ones that really represent the best opportunities According to Goldstein the pace of research into Mendelian diseases  single gene disorders like cystic fibrosis and sickle cell  has moderated after a period of rapid discoveries enabled by genetic sequencing
The move will affect 29% of Sanas workforce or about 123 employees based on the companys headcount of 424 fulltime employees at the end of June
Still the moves are a notable step back for a company that launched two years ago with an approved cancer cell therapy a builtout drug pipeline and hundreds of millions of dollars. At the time Bluebird was struggling to turn research progress in cell and splitting the company as a better way forward with 2Seventy inheriting the companys oncology work and a resized Bluebird focusing on gene therapies for sickle cell disease and beta thalassemia
We anticipate a European Commission decision on our MAA during the fourth quarter of 2023
Its treatment dubbed VGA039 is designed to restore production of a bloodclotting enzyme by targeting a molecule known as protein S The company is testing it in a Phase 1 trial for the blood disorder von Willebrand disease
Their treatment called exacel is difficult to make requiring a patients own blood stem cells to be extracted and then engineered using CRISPR gene editing technology. Its also likely to come at an extremely high price  possibly creating a sticking point with insurers
From there we scheduled and have now completed a very productive and informative endofphase2 meeting with the FDA and on that basis we anticipate initiating Study SRP9001301 EMBARK our pivotal trial for SRP9001 in September of this year in the United States and globally
The company recently disclosed Phase 1 data from a drug targeting another protein known as gamma secretase thats related to amyloid buildup
CART biotech Cargo Therapeutics prices 281M IPO The oﬀering is one of the largest for a biotech company in 2023 a year that has otherwise been sluggish for drugmaker IPOs
Its focusing on an ongoing Phase 3 trial of pegcetacoplan next year
Gilead is adding another earlystage biotechnology company to its rolodex of partners announcing Tuesday a multipart research alliance with the San Diegobased Tentarix Biotherapeutics
BrainStorms share price dropped 5 Wednesday trading at 17 cents
Emerging biotech Laronde Senda Biosciences merge to create new Flagship company Sail Biomedicines will combine Larondeʼs work on endless RNA with Sendaʼs research on programmable nanoparticles
Todays positive results advance our goal to establish an industry leading TTR franchise which currently includes ONPATTRO and AMVUTTRA for the polyneuropathy of hereditary ATTR amyloidosis
Affected children may experience developmental delays such as difficulty in walking climbing stairs or standing from a sitting position
Originally submitted for FDA review in 2019 the therapys application was derailed by the partial shutdown of the federal government that year. Approval when granted came with strings attached as the FDA ordered Aimmune to sell Palforzia through a risk management program designed to protect people from inadvertent allergic reactions
Other AIfocused companies like Recursion Pharmaceuticals, Exscientia, BenevolentAI, and Insitro have drawn similar large drugmaker partnerships too
The enthusiasm and continued support of our existing investors combined with the significant commitment from this new slate of top tier funds provides validation for our circuitbased discovery platform approach the quality of our clinical assets and the strong capabilities of our team said Christopher Kroeger the companys founder and CEO in a statement
Histogen plans to wind down operations after failing to find a partner The San Diego-based drugmaker will delist its shares on the Nasdaq stock exchange and sell its assets
They noted how some subgroups did not seem to experience much benefit on one or both of the trials measures including women, Black participants, and those taking tafamidis at the trials start
ProQR sale of eye drugs comes undone over employment contracts. The Dutch company said its focus will remain on an RNA editing platform called Axiomer
The patisiran and placebo arms had similar frequencies of adverse events AEs 912 percent and 944 percent respectively and serious adverse events SAEs 337 percent and 354 percent respectively
For JJ which records almost 7 billion in annual sales from brain drugs like Concerta and Invega Sustenna Arialys is another bet on neuroscience
A few months later the company announced plans to lay off as many as 500 employees as it cut costs to conserve cash for operations. Aprocitentan is the companys best hope for a major cash influx
The increase in cost of sales is primarily due to increased demand for the Companys products
Genesis work has drawn the attention of major pharmaceutical companies Eli Lilly and Roche  which partnered with the startup in 2022 and 2020 respectively
This press release contains forward looking statements that involve substantial risks and uncertainties
Taysha expects to receive gross proceeds of 150 million from the Private Placement which is expected to close August 16 2023  before deducting placement agent commissions and offering expenses
ALS patients lose the ability to perform essential tasks like talking eating and breathing and typically live just two to five years after diagnosis, disease is fatal and often fast-progressing patients and advocates have pressured the FDA to adopt more flexible policies that would allow easier and quicker access to treatments
Shares in the company nearly quadrupled in value and took the companys valuation to more than 7 billion sparking an investor rush to find other companies working on NASH drugs
The foundation said establishment of its plant should help concentrate cell therapy expertise in one place to create a largescale production facility
Verge is currently developing a drug for ALS known as VRG50635. The small molecule drug is meant to restore degenerated neurons by inhibiting PIKfyve, an enzyme that research indicates  performs various important cellular functions. It advanced into human testing last year
Approval was based on a trio of latestage studies testing Xphozah in more than 1000 patients as both a standalone treatment and in could reduce serum phosphorus levels albeit with diarrhea as a common side effect
Rocheʼs Genentech bets on radiopharmaceuticals in partnership with PeptiDream The collaboration agreement will focus on developing new targeted cancer drugs using PeptiDreamʼs technology platform
The Watertown Massachusettsbased biotechnology company emerged publicly in 2021 with half a billion dollars in funding from Arch Venture Partners Amgen and other venture investors
BioMarin undertakes no duty or obligation to update any forwardlooking statements contained in this press release as a result of new information future events or changes in its expectations
UniQure joins about a dozen gene therapy biotechs including Editas Medicine  Freeline Therapeutics and Intergalactic Therapeutics  to lay off staff this year
The center created a mouse model of another form of the disease called Type 4J that was recently selected for a gene therapy Institutes of Health
The startup is developing an antibody treatment in early human testing for an inflammatory condition called secondary hemophagocytic lymphohistiocytosis and preclinically for other immune diseases
Participants who received SRP9001 n20 had mean microdystrophin expression of 281 as measured by western blot
Early trial results showed some promise  and the therapy is now being studied in a larger Phase 3 trial
There was no signal for suicidal ideation and behavior in the postpartum studies FDA staff wrote
In March the company proposed to acquire Jounce Therapeutics, luring that drugmaker away from its plans to reverse merge with Redx Pharma. Concentra which had owned a minority stake in Jounce agreed to buy the rest for 180 a share in cash plus a CVR
Adverse reactions observed in at least 20 of treated patients and greater than placebo were VYONDYS 53 placebo headache 41 10 pyrexia 41 14 fall 29 19 abdominal pain 27 10 nasopharyngitis 27 14 cough 27 19 vomiting 27 19 and nausea 20 10
The London-headquartered company last year laid off workers and discontinued development of some of its pipeline projects to help sustain operations through a US launch of Libmeldy as investor enthusiasm for gene therapies cooled
Ventyx revises TYK2 drug plans after lackluster study data Results showed Ventyxʼs drug met its goal in a Phase 2 psoriasis trial
SRP9001 is intended to deliver a gene that codes for a shortened functional form of dystrophin to muscle cells
The significantly different baseline characteristics between treatment and control groups in the 67 age group may have contributed to the inability to observe a treatment effect in the 67 age group at the week 48 timepoint in Part 1
Apellis is also facing imminent competition from rival Iveric Bio  which won approval earlier this month for its own medicine to treat geographic atrophy
FDA wonʼt hold advisory meeting for Bluebirdʼs sickle cell gene therapy. The decision stands in contrast to the agencyʼs plans for a would-be rival gene treatment from Vertex Pharmaceuticals and CRISPR Therapeutics
Apellis also reported one new case of the rare side effect known as retinal vasculitis bringing the total number of eight
NonGAAP research and development expenses were 3625 million and 2746 million for the six months ended June 30 2021 and 2020 respectively an increase of 879 million
Ultragenyx will grant the new company a license for the required intellectual property and knowhow allowing it to use Ultragenyxs Pinnacle PCL manufacturing platform
Ascendis will request a Type A meeting with the FDA as soon as possible to agree on the best path forward
The Boston-based startup also named Kurt Graves Intarcias longtime leader as its chairman and CEO alongside a 46 million Series A round to advance its research
A final approval decision typically consistent with the CHMP recommendation is expected from the European Commission later this year
The worldwide incidence rate of achondroplasia is about one in 25000 live births
For additional information please visit BioMarincom
Shares in BrainStorm fell by one third in Monday morning trading following the documents release
The second measure a questionnaire designed to assess quality of life showed patients taking patisiran scored 37 points higher than those on a placebo At the meeting of outside experts convened by the FDA last month some of the agencys advisers debated the magnitude of patisirans benefit in APOLLOB
After 12 hours skip the missed dose and administer the next daily dose as usual
This drug could be 2 billion in a base case with a regular pharma partner considering it may be 3rd to market or 45 billion if a major immunology player is backing it the gut which over time damages the gastrointestinal tract
The biotech claims this method allows it to rapidly advance new data insights into clinical candidates The technology has attracted partners in the past
These forwardlooking statements involve risks and uncertainties many of which are beyond Sareptas control
The decrease in selling general and administrative expenses primarily reflects the following 07 million decrease in compensation and other personnel expenses primarily due to a change in headcount period over period and 06 million decrease in professional service expenses primarily due to a decrease in reliance on thirdparty selling general and administrative contractors
Other adverse reactions that occurred in at least 10 of patients treated with AMONDYS 45 and at least 5 more frequently in the placebo group were ear pain nausea ear infection posttraumatic pain and dizziness and lightheadedness
ForwardLooking Statements In order to provide Sareptas investors with an understanding of its current results and future prospects this press release contains statements that are forwardlooking
The vision of the patient affected in May has returned to baseline while the other one is still being evaluated
In just the past two weeks Apellis Pharmaceuticals and Sage Therapeutics have each trimmed hundreds of jobs
What is the most important safety information about VOXZOGO
There were three serious treatmentemergent adverse events in two patients in the 30 mgkg cohort including two cases of hypomagnesemia
Overall investment in platformfocused biotechnology companies has declined this year according to a July report from HSBC Innovation Banking though Generate appears to buck that trend
Approximately 25 of people with achondroplasia fall into this category 102023 805 PM US Food and Drug Administration Approves BioMarins VOXZOGO vosoritide for Children Under 5 Years with Achondroplasia  Oct 20  httpsinvestorsbiomarincom20231020USFoodandDrugAdministrationApprovesBioMarinsVOXZOGORvosoritideforChildrenUnder5Yearswith 611VOXZOGO US
102023 805 PM US Food and Drug Administration Approves BioMarins VOXZOGO vosoritide for Children Under 5 Years with Achondroplasia  Oct 20  httpsinvestorsbiomarincom20231020USFoodandDrugAdministrationApprovesBioMarinsVOXZOGORvosoritideforChildrenUnder5Yearswith 411VOXZOGO is also approved in Japan in children from birth who have achondroplasia with open growth plates
Precede is also working on bloodbased tests that could supplant existing tissuebased tests in clinical practice
An archived webcast will be available on the Companys website approximately two hours aer the event
Sarepta is responsible for global development and manufacturing for SRP9001 and plans to commercialize SRP9001 in the United States upon receiving FDA approval
Adlai Nortye IPO gives old Novartis cancer drug new life. The proceeds will fund a Phase 3 test of a medicine once known as buparlisib and tested by Novartis in dozens of trials. The company sold 25 million shares at 23 apiece  a smaller offering than it had pitched in early August
The money will fund development of new drugs for cancer metabolic diseases and other conditions the most advanced of which is a tumortargeting medicine in Phase 1 testing
Triveni emerges from biotech merger with 92M for immune disease drugs The new startup is the product of a merger between an Atlasbacked startup and a biotech cofounded by Tillman Gerngross
The layoffs are part of a larger restructuring plan that has come on the heels of a strategic review of Kinnates business
BioMarin is a registered trademark of BioMarin Pharmaceutical Inc and ROCTAVIAN is a trademark of BioMarin Pharmaceutical Inc
The increase primarily reflects the launch of AMONDYS 45 in the first quarter of 2021 and the continuing increase in demand for the Companys products in the US For the three months ended June 30 2021 and 2020 the Company recognized 223 million and 260 million of collaboration revenue respectively
Ascendis may not actually achieve the plans carry out the intentions or meet the expectations or projections disclosed in the forward looking statements and you should not place undue reliance on these forward looking statements
AMONDYS 45 uses Sareptas proprietary phosphorodiamidate morpholino oligomer PMO chemistry and exonskipping technology to bind to exon 45 of dystrophin premRNA resulting in exclusion or skipping of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping
The Company has built an impressive position in Duchenne muscular dystrophy DMD and in gene therapies for limbgirdle muscular dystrophies LGMDs mucopolysaccharidosis type IIIA CharcotMarieTooth CMT and other CNSrelated disorders with more than 40 programs in various stages of development
BioMarin plans to initiate a pivotal development program in hypochondroplasia later this year
Its participation in Marianas Series B is the first investment from that new fund called the Forbion Ventures Fund VI.
She claims the size and scale of the company's data swamps other resources. Compare for instance the UK Biobanks data on 550000 people to 23andMes 14 million
In 2022 the company said a different study had succeeded and began the process of applying to the FDA even as questions remained about the magnitude and durability of the treatments effects. Sage said Thursday it expects the latest reorganization to save about 240 million a year
See additional safety information in the Prescribing Information and Important Safety Information below
Apellis said Tuesday that all of the cases to date have occurred after the first injection of Syfovre
One of the most important aspects of healthcare long term is being able to prove that to deliver care in any kind of scalable way it cannot be dependent on an interaction of onetoone between a consumer and a physician
According to Kathiresan the FDA had also wanted to see data from the company comparing the treatments potency in animal tests to those in humans and data showing whether VERVE101 caused edits in sperm or egg cells  a key concern for in vivo therapies
About Ascendis Pharma AS Ascendis Pharma is applying its innovative TransCon technology platform to build a leading fully integrated global biopharma company focused on making a meanin gful difference in patients lives
The company is also still studying a formulation of the twicefailed drug gantenerumab thats designed to better reach into the brain
Investors who have been concerned about the companys balance sheet can now focus on the prospects for the companys pipeline and a pending European regulatory decision on its Translarna drug for a form of Duchenne muscular dystrophy
For additional information regarding this product please contact BioMarin Medical Information at medinfobmrncom
Meanwhile over 100 drug companies both public and private have laid off staff so far this year many in an effort to preserve cash
The company is being advised by a board that includes Droias George Golumbeski who led business development at Celgene for many years as well as John McHutchinson formerly the CEO of Assembly Bio and before that Gileads top scientist
Two lentivirusbased gene therapies have now been approved  Bluebird bios Zyntelgo and Skysona  so the FDA has experience in reviewing treatments using this approach
Expanded Access Program continues to enroll new patients
The company attracted the latest infusion of cash after reaching preclinical milestones for its lead therapy
The trial was the first big test for Acelyrin after it pulled off the biggest initial public offering for a biotech in more than two years
Meanwhile Moderna and Merck are working on a therapeutic vaccine that would combine the companies experimental mRNA 4157 with Mercks blockbuster Keytruda cancer medicine
FibroGen shares fell by 27 on Wednesday to less than 1 a piece
About Alnylam Pharmaceuticals Alnylam Nasdaq ALNY has led the translation of RNA interference RNAi into a whole new class of innovative medicines with the potential to transform the lives of people alicted with rare and prevalent diseases with unmet need
We had a scientific advisory group that pontificated aboutall the crazy. So I feel grateful for the team that helped us think through a lot of it  and we were well prepared for a lot of the issues that came our way
Full results will be presented at a future medical meeting
For further information please see the full Prescribing Information
Precede sees an opportunity to replace tissuebased tests with a liquid biopsy
Symptoms of DMD usually appear in infants and toddlers
Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials
I want to thank all the patients caregivers investigators and study sta who have and continue to participate inth101723 633 AM Alnylam Pharmaceuticals Press Release  Aug 03 2022  Alnylam Reports Positive Topline Results from APOLLOB Phase 3 Study of Patisira httpsinvestorsalnylamcompressreleaseid26851 39APOLLOB
What are the most common side effects of VOXZOGO
Visit ascendispharmacom to learn more
Otsuka will pay Shape an undisclosed amount upfront and has promised more than 15 billion more if certain development regulatory and sales milestones are met
Novo set a new standard in obesity treatment when it repurposed its daily bloodsugarlowering injection Victoza into a weightloss drug called Saxenda
Following the termination of the applicable waiting period under the HartScottRodino Antitrust Improvements Act of 1976 as amended in April 2021 the Company completed its sale of the PRV and received proceeds of 1020 million with no commission costs which was recorded as a gain from sale of the PRV as it did not have a carrying value at the time of the sale
Aiolos Bio raises 245M on prospect of better asthma drug The Series A funding round ranks among the largest in the biotech industry this year with investors including Atlas Bain and Forbion backing the startup
A statistically significant imbalance P00046 in baseline NSAA total score was present in the cohort of 67yearold participants n25 resulting in milder participants in the placebo arm n13 than in the treated arm n12
The company aims to launch as many as 15 new products in the next five years including four by 2025
Newer versions like Novo Nordisks Wegovy and Eli Lillys Mounjaro have surged in popularity due to their more potent weightloss effects
Superluminal draws RA Capital Nvidia as investors in GPCR drug hunt The Bostonbased companyʼs 33 million ﬁnancing will help it develop its AI technology and build a small molecule drug pipeline