daniel-gordon/biopharma-dive huggingface dataset:
Biogen leadership then tapped Singhal who at the time was the companys head of global safety and regulatory sciences to act as interim head of research and development until a permanent replacement was found
US and European regulators are currently reviewing approval applications for the medication and the Food and Drug Administration was originally slated to give an answer by Dec 19
The condition is universally fatal and patients usually succumb to the disease in their twenties
For now the company will continue to develop them privately avoiding scrutiny that can follow an IPO. Staying private allows us to stay out of the fray Keiper said and avoid the market fluctuations that are uncoupled from accomplishments Nimbus is becoming a RD institution in Boston he said
Novo Nordisks started about two years ago as an experiment. Lets bring together an RD team thats interdisciplinary scientists with disease priorities scientists who understand molecules in a very broad sense. We sought to bring a team with very deep cardiometabolic expertise into Cambridge and the wider Boston innovation ecosystem with the anticipation wed come together with entrepreneurs emerging and more established biotechs academia and venture capital
Guided by its core values of patients science and passion the company uses its TransCon technologies to create new and potentially best inclass t herapies
Hemophilia A also called Factor VIII deficiency or classic hemophilia is an Xlinked genetic disorder caused by missing or defective Factor VIII a clotting protein
Regulators cited safety concerns such as acute kidney injury in rejecting the companys application in 2017. Still many are hopeful the next year could see a different market for biotech. The FDA then turned back another request in 2020
The company founded in 2021 said the money will be used for research corporate expansion and initial clinical validation of two experimental programs
Ascendis does not assume any obligation to update any forward  looking statements except as required by law
We remain confident in the data supporting the value of NurOwn for ALS patients and are committed to finding a path forward Roughly 30000 people in the US have ALS a progressive disease that causes the decay and death of nerve cells
We also look forward to further understanding the potential role of VOXZOGO in other genetic short stature conditions including hypochondroplasia
Examples of such statements include but are not limited to statements relating to i Ascendis intent to work collaboratively with the FDA to make TransCon PTH available to patients in the US
Orchard nears FDA decision on rare disease gene therapy Three years aer gaining European approval Libmeldy is now under US review with a deadline set for March
The company revealed its new plans alongside third quarter earnings in which it reported a net loss of 104 million about twice the 53 million loss it recorded during the same period in 2022
No SAEs occurred at least 2 percent more frequently in patisiran versus placebo treated patients
For Vertex which became one of the worlds largest biotechnology companies by developing oral drugs for cystic fibrosis  exacel exquisite amount of choreography between all parties involved including the patients the treatment centers and Vertex itself according to the companys chief operating officer Stuart Arbuckle
Shares in 2Seventy worth as much as 38 apiece in November 2021 have steadily declined in value and now trade for about 4
Neumora Therapeutics on Friday filed plans for an initial public offering one month after announcing it would soon begin late stage testing for a new kind of depression drug
More recently a newer entrant Paragon Therapeutics  spun out Apogee Therapeutics and fasttracked the company to an initial public offering in less than a year
These forwardlooking statements include without limitation statements relating to our future operations business plans priorities research and development programs the potentially transformative benefits of SRP9001 our understanding from the FDA that 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 and expected timelines plans and milestones including the regulatory action date and our understanding that the advisory committee will be held prior to the regulatory action date
VOXZOGO is supplied with Instructions for Use that describe the steps for preparing injecting and disposing VOXZOGO
The Company excludes from its nonGAAP results loss on contingent consideration related to the Companys acquisition of Myonexus in 2019 as it is a noncash item and is not considered to be normal operating expenses due to its variability of amounts and lack of predictability as to occurrence andor timing
A second program CRG023 is in preclinical studies for Bcell malignancies
Kinnate said certain drug programs including one testing its experimental medicine exarafenib as a monotherapy will be halted in order to prioritize others
Other expense netFor the three months ended June 30 2021 and 2020 other expense net was approximately 162 million and 124 million respectively
Yet Novo faces competition from Eli Lilly which has an Ozempic competitor in Mounjaro and smaller drugmakers looking to replicate Novos and Lillys success in obesity
The medicines developers Vertex Pharmaceuticals and CRISPR Therapeutics are already preparing for what happens next should they win approval
The key prospect to emerge from that work ITCA 650 slowly pumps out the diabetes medication exenatide an early entrant of a now widely known class of diabetes treatments known as GLP1 agonists
Months later GSK said it was partnering with the University of Oxford to establish a new center dedicated to making the drug development process faster and more effective
Initial treatment had to occur in a healthcare setting with epinephrine on hand. But Nestlé which had been investing in Aimmune since 2016 decided to build an allergy treatment business around Palforzia and housed the treatment in a standalone unit
Wev'e seen numerous gene therapy companies  seek stricter cost control shut down programs andor seek strategic alternatives throughout the year trading
And the startup has now drawn the interest of both seasoned biotech investors and Lilly with a pipeline of three other drugs that work in different ways
Orphan Drug Designation in Hypochondroplasia VOXZOGO also recently received orphan drug designation from the FDA for the treatment of hypochondroplasia a genetic condition caused by a mutation in the fibroblast growth factor receptor 3 FGFR3 gene and characterized by impaired bone growth
These forwardlooking statements involve risks and uncertainties many of which are beyond our control
San Diegobased biotech RayzeBio which went public last week  has also partnered with PeptiDream and developed two targeted radiopharmaceuticals. In the Genentech deal PeptiDream will take the lead on the preclinical development of radioisotope drug conjugates
The companies will share development costs and any profits in major markets with each taking the lead marketing the therapy in different countries. The drug dubbed TEV574 is being tested as a treatment for ulcerative colitis and Crohns disease two types of inflammatory bowel disease. Its one of a class of drugs that target a protein called TL1A
The San Mateo Californiabased company priced 1875 million shares at 15 apiece on the low end of its proposed price range. Its IPO was the fourth largest this year for a biotech
Guided by its core values of patients science and passion the company uses its TransCon technologies to create new and potentially best inclass therapies
In evaluating Zurzuvaes potential benefits and risks the FDA noted how socalled serious adverse events were reported throughout the development program
Your healthcare provider will monitor the patients growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow
When the company won approval for the drug in February analysts predicted it could bring in peak annual sales of 3 billion
TSHA102 utilizes the novel miRNA Responsive Auto Regulatory Element miRARE technology designed to mediate levels of MECP2 in the CNS on a cellbycell basis without risk of overexpression
The biotech claims blocking this protein could inhibit inflammation without tamping down the bodys ability to fight infections a side effect of many immunedirected medicines
Startup Mariana raises $175M for radiopharmaceutical drug research The Series B round was led by Deep Track Capital and Forbion and also drew the participation of Eli Lilly
The significant interest in this fundraise particularly given the challenges of the current financing environment is testament to the Rome team science and opportunities Jeff Hatfield chair of Romes board said in a statement
VOXZOGO is being tested in children whose growth plates are still open typically those under 18 years of age
Agenus has argued that botensilimab may be superior to older similar CTLA4 inhibitors like Bristol Myers Yervoy and is testing it alongside balstilimab in a variety of settings
More broadly young biotechnology companies are facing a weaker market thats forced many to cut costs and lay off staff over the past year
SRP5051 is designed to bind to exon 51 of dystrophin premRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable toexon 51 skipping
The dark genome refers to DNA that doesnt directly encode for proteins and therefore has drawn comparatively less attention from researchers and drugmakers
Research and development expenses were 4348 million for the six months ended June 30 2021 compared to 3247 million for the same period of 2020 an increase of 1101 million
PTC to lay off more staff following drug setback in Europe. For the second time this year PTC is restructuring this time aer the EMA decided against renewing a conditional approval for the companyʼs drug Translarna
In the openlabel study 20 participants between the ages of four and seven were treated with a single infusion of SRP9001 at a dose of 133x1014 vgkg
Participants continue to be monitored for safety and will undergo another biopsy at week 12 in Part 2 to assess expression and biological markers in addition to longerterm assessments of functional outcomes
VOXZOGO is taken daily as an injection given under the skin administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO
Roche is deepening its commitment to neuroscience agreeing to a licensing deal with Ionis to develop two preclinical RNA medicines for Alzheimers and Huntingtons diseases Ionis said Wednesday
Eli Lilly is deepening ties to gene editing specialist Verve Therapeutics spending 200 million to acquire option rights to several of Verves drug programs from the biotechnology companys partner Beam Therapeutics
These forwardlooking statements include statements relating to our future operations financial performance and projections business plans market opportunities priorities and research and development programs including the design of the AAVrh74 vector to systematically and robustly deliver treatment to skeletal muscle making it an ideal candidate to treat muscle disease and expected plans and milestones including our plan to initiate pivotal trial SRP9001301 Study 301 or EMBARK in September of 2021 in the United States and globally and our plan to move as fast as science will permit to confirm the benefits of SRP9001 and if successful to rapidly advance this therapy to waiting Duchenne patients around the world
Researchers working for the company found that an enzyme called protective proteincathepsin A or PPCA can break up a form of amyloid beta the protein many consider responsible for Alzheimers debilitating effects
Insight usually invests in software companies while the chipmaking giant Nvidia is putting more capital into artificial intelligence bets across multiple sectors. The firm has made other biotech bets this year including a new Genesis Therapeutics Series B round
We were able to comprehensively address all of the preclinical questions provide the clinical data and then got our application cleared said company CEO,  a cardiologist and geneticist who formed Verve five years ago to develop a onetime treatment for heart disease
About VOXZOGO vosoritide for Injection In children with achondroplasia endochondral bone growth an essential process by which bone tissue is created is negatively regulated due to a gain of function mutation in FGFR3
EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping
The company develops and commercializes targeted therapies that address the root cause of genetic conditions
In Type 1 diabetes which is caused by the autoimmune destruction of insulinsecreting pancreatic cells a replacement therapy could relieve patients of the burden of managing their insulin levels especially as their disease advances
PTC said it will use the cash to retire all outstanding debt obligations with Blackstone Life Sciences as well as preserve funds for future operations. For now the company will retain 19 of royalties from Evrysdi which is marketed by Swiss drugmaker Roche
About EXONDYS 51 EXONDYS 51 eteplirsen uses Sareptas proprietary phosphorodiamidate morpholino oligomer PMO chemistry and exonskipping technology to bind to exon 51 of dystrophin premRNA resulting in skipping of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping
This notification from FDA relates to the ir review of our NDA and s ince the NDA deficiencies were not disclosed in the letter we are eager to work with FDA to learn understand and address them
In its own briefing document  BrainStorm does not directly address the FDAs concerns about its ability to reliably produce NurOwn
This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in some patients treated with EXONDYS 51
The companys distinctive approach to drug discovery has produced a diverse pipeline of commercial clinical and preclinical candidates that address a significant unmet medical need have wellunderstood biology and provide an opportunity to be firsttomarket or offer a substantial benefit over existing treatment options
In addition the APOLLOB data further strengthen our confidence in our Phase 3 HELIOSB study of vutrisiran in ATTR amyloidosis with cardiomyopathy which is expected to report out in early 2024 said Yvonne Greenstreet MBChB Chief Executive Oicer of Alnylam
We will be well prepared and look forward to presenting the wealth of evidence supporting the transformative potential of SRP9001
The company also has a prostate cancer drug in earlystage testing
Neumora Therapeutics which launched in late 2021 with the aim of developing precision medicines for psychiatric and neurodegenerative disorders has brought in approximately 650 million from a lengthy list of investors that includes Arch and the biopharmaceutical giant Amgen
Samsara raised 37 million prior to the Series A round announced Tuesday
When developing new medicines drugmakers typically try to make treatments that are more effective durable or safer than whats already available. Rampart Bioscience a new biotechnology company eyeing a way to make better DNAbased medicines is striving to hit all three
Genentech takes up a deal to make ʻmolecular glueʼ drugs Through a collaboration with Orionis Biosciences the Roche subsidiary is eyeing targeted protein degradation medicines for cancer and neurodegenerative conditions
Biologic drugs like Humira Stelara and Entryvio can be used to treat both ulcerative colitis and Crohns while pills like Xeljanz and Rinvoq are cleared for the former condition. Shares in Teva fell by 2 in Wednesday morning trading
Beam Lilly will now own optin rights to jointly develop and commercialize with Verve gene editing medicines for heart disease that are aimed at several wellknown drug targets. The new deal brings the companies closer and allows Lilly to closely track Verves progress on programs aimed at other genes important to heart disease risk
The safety profile of SRP9001 observed in the first 11 participants is consistent with the safety seen in earlier studies using clinical manufacturing process material
For more information about our people science and pipeline please visit wwwalnylamcom httpsctsbusinesswirecomctCT
Rapport which was built around a discovery platform from Johnson  Johnsons research unit officially launched early this year with backing from JJs strategic venture capital arm as well as the investment firms Third Rock Ventures and Arch Venture Partners
In line with previously reported clinical data no clinical complement activation was observed
The pharmaceutical company also revealed plans to split off its consumer health business next year a move similar to divestments done by other large drugmakers
In the United States Ascendis submitted its NDA for TransCon PTH in hypoparathyroidism in August 2022 in October 2022 the FDA accepted the NDA for Priority Review and set a PDUFA action date of April 30 2023
We are confident in valoctocogene roxaparvovec gene therapy and its potential to redefine the treatment paradigm for people with hemophilia A
The company has decided to delay its SG299 therapy for patients with blood cancer. As of June 30 Sana had a cash position of 3259 million, down from 434 million at the end of December. With the layoffs announced Tuesday the company expects its cash burn rate to be below 200 million next year. Sana is certainly not alone in shedding staff amid a downturn in biotech financing
The multiyear deal also offers 2 billion worth of socalled biobucks or future payments contingent on hitting certain development and commercialization milestones
The deal involves three separate multiyear collaborations between the companies to discover and develop biologic therapies for cancer and inflammatory diseases using Tentarixs drugmaking technology. Gilead will also hold options to acquire up to three Tentarix subsidiaries built around each of the collaborations
A replay of the webcast will be available on this section of our website shortly after conclusion of the event for 30 days
Actual results could materially differ from those stated or implied by these forwardlooking statements as a result of such risks and uncertainties
The cuts announced Thursday include the closure of a lab in Lexington Massachusetts and discontinuation of an Parkinsons drug candidate among other unnamed programs
The biotech provided Beam Lilly with an early look at those results as part of the pharma companys due diligence for the deal Verve, the CEO said
The severe form of hypophosphatasia affects an estimated 1 in 100000 babies but milder cases likely occur more frequently and later in life. Especially for those who have diseases where it impacts their quality of life helping them fortify their own bodies  is a great step
The Waltham Massachusetts startup formed as a merger between two companies Amagma Therapeutics and Modify Therapeutics, and is backed by a syndicate of blue chip investors coled by Atlas Venture and Cormorant Asset Management
Sanofi buys into Teva gut disease drug The French drugmaker will pay 500 million to collaborate with Teva on an antiTL1A therapy for ulcerative colitis and Crohnʼs disease
Rapport Therapeutics a young biotechnology company developing precision drugs for brain and nervous system disorders has raised another 150 million from a collection of existing and new investors
Data from a Phase 1 trial were released this June and showed positive signs with regard to safety and tolerability
Settlement and license charges In February 2021 the Company recognized a 100 million settlement charge related to contingent settlement payments to BioMarin Pharmaceutical Inc BioMarin as a result of the approval of AMONDYS 45 in the United States
Cash and cash equivalents As of June 30 2023  Taysha had 451 million in cash and cash equivalents
The nonGAAP loss is defined by the Company as GAAP net loss excluding interest expense net income tax benefit expense depreciation and amortization expense stockbased compensation expense and other items
The program will also advance a program for the liver and lung condition alpha1 antitrypsin deficiency and conduct an initial clinical trial at certain US sites for a treatment for glycogen storage disease 1a
In it the CEO sees a promise to develop a drug that resembles a traditional biologic that would appear more familiar to patients
Over the past few years Novo has broken out of character to ink a string of acquisitions including for biotechnology companies Dicerna Pharmaceuticals and Forma Therapeutics  that flesh out its drug pipeline in other metabolic and rare blood conditions
In these studies TransCon PTH has been generally well tolerated with no discontinuations related to study drug
Novo Nordisk has built a reputation on developing drugs for diabetes and more recently obesity
Verge Genomics a biotechnology company using artificial intelligence to develop therapies for diseases of the nervous system announced on Friday a four-year partnership with AstraZenecas rare disease unit Alexion to identify new drug targets. As part of the agreement Verge will receive up to 42 million in upfront equity and nearterm payments
Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency eg
A potential new way to attack targets once thought undruggable molecular glues have become the focus of a number of collaborations between smaller biotechnology companies and larger pharmaceutical firms including Sanofi and Astellas
Molecular glues, so named for their ability to forcibly pull together two proteins that wouldn't otherwise interact with each other  have come a long way since they were discovered around three decades ago
Obesity affects a large swathe of the US adult population and the many health issues  particularly the metabolic disorders  that priority
Study 102 uses clinical process SRP9001 and has two primary endpoints microdystrophin expression at 12 weeks and change in NSAA total score at 48 weeks compared to placebo
If a persistent increase in serum cystatin C or proteinuria is detected refer to a pediatric nephrologist for further evaluation
While lowering its forecast for the year BioMarin said reimbursement issues for Roctavian are looking up in the US and other parts of the world
PTC Therapeutics has agreed to sell a big chunk of its remaining royalty stream from the spinal muscular atrophy drug Evrysdi to Royalty Pharma for 1 billion
Apellis won't start any new development on a systemically administered version of pegcetacoplan the medicine it sells as Syfovre for geographic atrophy and Empaveli for a rare blood disorder
Acelyrins stumble could help rival MoonLake Immunotherapeutics  which is also developing an IL17 treatment for hidradenitis suppurativa and psoriatic arthritis Cantor Fitzgerald analyst Prakhar Agrawal said in a note to clients
Most of the shots are either in early stages of testing or are still preclinical but the company has pushed an experimental shot for cytomegalovirus into Phase 3
The test is designed to identify epigenetic changes that signal the start of tumor development
The panel nonetheless chose to back patisiran citing the drugs manageable side effects and modest benefit
The job cuts announced Tuesday will affect about 225 employees
Sana Biotechnology which just two years ago was one of the most wellfinanced startups in the industry plans to make a second round of layoffs in less than 11 months to save cash
We are surprised and disappointed that the FDA introduced new expectations for the first time in the Complete Response Letter
But roughly 70 of the estimated 850 or so GPCRs in the human body are currently considered undruggable according to Superluminal. That leaves plenty of room for young biotechs to try to develop new ways of targeting those receptors