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Following that rejection Intercept canceled all work in NASH for by the FDA and laid off staff | 0 |
One Piqray has since reached market winning regulatory approval in 2019 for certain breast cancer patients. Its been modestly successful generating 373 million in worldwide sales last year | 1 |
Biogen continuing executive revamp finds a new research head Jane Grogan an industry veteran who worked at Genentech for over 15 years will take over the role starting Oct 2 | 2 |
The company announced its initial public offering in December 2020 in which it priced shares at 20 apiece. Not long after its share price doubled to over 40 although it stayed at that level for only a short period | 2 |
In Part 2 the study remains blinded while all participants in the placebo group cross over to active treatment and all participants are followed for another 48 weeks while safety and efficacy continue to be evaluated | 1 |
Apellis also has not proven that the drug can slow or stop the deterioration of vision in patients | 2 |
VTX958 is a socalled TYK2 inhibitor one of a class of drugs that have drawn significant interest from drugmakers | 1 |
In biopsies taken at a median of 12 weeks and after only three doses results from Part A of MOMENTUM study found that the 30 mgkg of SRP5051 dosed monthly resulted in 18 times the exon skipping and eight times the dystrophin production as eteplirsen dosed weekly for 24 weeks | 1 |
Fulcrum rejoins sickle cell drug race as FDA lifts study hold. The regulator had 铿俛gged a cancer risk in drugs like Fulcrum始s leading the biotech to focus further testing in patients with more severe disease | 2 |
First the UKbased pharmaceutical giant agreed to pay at least 700 million for rights to two experimental drugs from Alector a Californian company tapping into the immune system to combat neurodegenerative diseases | 2 |
The Massachusetts biotech said it found irregularities in the internal structure of some 19 gauge needles used to withdraw the medicine from vials before an injection | 1 |
BioMarins unparalleled research and development capabilities have resulted in eight transformational commercial therapies for patients with rare genetic disorders | 2 |
Next up for Sana is a planned filing for permission to study its SC262 cell therapy in patients with Bcell lymphomas and leukemias and development of a treatment for Type 1 diabetes | 1 |
The company debuted preclinical data at the American Society of Hematology conference showing how the tumortargeting medicine could be used to treat acute myeloid leukemia | 1 |
Currently there are no approved diseasemodifying therapies that treat the genetic root cause of the disease | 1 |
Stallergenes Greer which specializes in allergy treatments hopes to turn the products fortunes around. By entering the food allergy space Stallergenes Greer becomes the first allergen immunotherapy company to offer both respiratory and food allergy treatments said the CEO in a statement | 2 |
Together we leverage our fully integrated platform with a goal of dramatically improving patients lives | 2 |
Though large sections are redacted documents from the FDAs review give a better sense of why the agency issued a split decision | 1 |
I am pleased to report that the Sarepta team continues to execute serving our patient community and advancing our portfolio | 2 |
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 unforeseen expenses related to Ascendis development programs unforeseen selling general and 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 Ascendis ability to obtain additional funding if needed Page 3 of 3 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 worldwide COVID 19 pandemic and ongoing conflicts suc h as that in the region surrounding Ukraine and Russia | 1 |
Any of the foregoing risks could materially and adversely affect the Companys business results of operations and the trading price of Sareptas common stock | 1 |
Andr茅 Andonian a 34year veteran of the consultancy McKinsey Co, will serve as Flagships AsiaPacific chair and senior adviser responsible for leading the companys presence in the region | 1 |
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 | 1 |
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 | 2 |
The condition is universally fatal and patients usually succumb to the disease in their twenties | 1 |
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 | 1 |
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 | 1 |
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 | 2 |
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 | 1 |
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 | 0 |
The company founded in 2021 said the money will be used for research corporate expansion and initial clinical validation of two experimental programs | 1 |
Ascendis does not assume any obligation to update any forward looking statements except as required by law | 1 |
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 | 0 |
We also look forward to further understanding the potential role of VOXZOGO in other genetic short stature conditions including hypochondroplasia | 2 |
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 | 1 |
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 | 1 |
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 | 0 |
No SAEs occurred at least 2 percent more frequently in patisiran versus placebo treated patients | 1 |
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 | 1 |
Shares in 2Seventy worth as much as 38 apiece in November 2021 have steadily declined in value and now trade for about 4 | 0 |
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 | 1 |
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 | 0 |
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 | 1 |
VOXZOGO is supplied with Instructions for Use that describe the steps for preparing injecting and disposing VOXZOGO | 1 |
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 | 1 |
A second program CRG023 is in preclinical studies for Bcell malignancies | 1 |
Kinnate said certain drug programs including one testing its experimental medicine exarafenib as a monotherapy will be halted in order to prioritize others | 0 |
Other expense netFor the three months ended June 30 2021 and 2020 other expense net was approximately 162 million and 124 million respectively | 1 |
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 | 2 |
The medicines developers Vertex Pharmaceuticals and CRISPR Therapeutics are already preparing for what happens next should they win approval | 1 |
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 | 1 |
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 | 1 |
Any of the foregoing risks could materially and adversely affect the Companys business results of operations and the trading price of Sareptas common stock | 0 |
Initial treatment had to occur in a healthcare setting with epinephrine on hand. But Nestl茅 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 | 1 |
Wev'e seen numerous gene therapy companies seek stricter cost control shut down programs andor seek strategic alternatives throughout the year trading | 1 |
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 | 1 |
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 | 1 |
These forwardlooking statements involve risks and uncertainties many of which are beyond our control | 1 |
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 | 1 |
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 | 1 |
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 | 1 |
Guided by its core values of patients science and passion the company uses its TransCon technologies to create new and potentially best inclass therapies | 2 |
In evaluating Zurzuvaes potential benefits and risks the FDA noted how socalled serious adverse events were reported throughout the development program | 0 |
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 | 1 |
When the company won approval for the drug in February analysts predicted it could bring in peak annual sales of 3 billion | 1 |
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 | 1 |
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 | 2 |
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 | 2 |
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 | 1 |
VOXZOGO is being tested in children whose growth plates are still open typically those under 18 years of age | 1 |
These forwardlooking statements involve risks and uncertainties many of which are beyond our control | 1 |
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 | 0 |
More broadly young biotechnology companies are facing a weaker market thats forced many to cut costs and lay off staff over the past year | 0 |
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 | 1 |
The dark genome refers to DNA that doesnt directly encode for proteins and therefore has drawn comparatively less attention from researchers and drugmakers | 1 |
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 | 0 |
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 company始s drug Translarna | 0 |
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 | 1 |
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 | 1 |
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 | 1 |
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 | 1 |
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 | 2 |
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 | 1 |
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 | 2 |
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 | 1 |
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 | 1 |
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 | 1 |
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 | 1 |
The company develops and commercializes targeted therapies that address the root cause of genetic conditions | 2 |
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 | 0 |
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 | 1 |
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 | 1 |
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 | 2 |
In its own briefing document BrainStorm does not directly address the FDAs concerns about its ability to reliably produce NurOwn | 1 |
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 | 2 |
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 | 2 |
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 | 2 |
We will be well prepared and look forward to presenting the wealth of evidence supporting the transformative potential of SRP9001 | 2 |
The company also has a prostate cancer drug in earlystage testing | 1 |
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 | 2 |