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Huttner K.,Edimer Pharmaceuticals
American Journal of Medical Genetics, Part A | Year: 2014

X-linked hypohidrotic ectodermal dysplasia (XLHED) is the most common genetic disorder of ectoderm development, presenting with abnormalities of skin, teeth, hair, and secretory glands. In the first years of life, XLHED-affected patients are at risk for life-threatening hyperthermia and pulmonary infection. Survival into childhood and beyond is associated with severe dental abnormalities as well as chronic growth, respiratory, skin, eye, and psychosocial disorders. Currently there are no approved therapies to restore function in disorders of development like XLHED. Over the last two decades, molecular research has provided convincing evidence that alterations in the ectodysplasin (EDA) gene that disrupt the encoded protein EDA-A1 are causative for XLHED. In mouse and dog XLHED models, administration of a single course of an EDA-A1 replacement protein (EDI200) resulted in permanent correction of the key phenotypic features, providing the first hope for an effective, targeted therapy. Animal models for genetic disorders have their strengths and limitations that must be considered when modeling clinical studies in human patients. Of greatest significance in the case of a developmental disorder may be the relative timeline for normal development and the maturation level at birth. With FDAclearance to start EDI200 studies in XLHED patients, we are on the verge of converting a decade of animal studies into the first test of a novel paradigm for rescue and permanent correction of a human developmental disorder. © 2014 Wiley Periodicals, Inc.


Patent
Edimer Pharmaceuticals | Date: 2012-05-10

The invention relates to methods for the temporal administration of EDA agonists, in particular EDI200, which correlate to optimal therapeutic response windows required for the formation of any EDA-dependent structures such as ectodermal appendages. Use of the methods described allow for the design of targeted therapeutic dosing and administration regimens in order to correct or alter abnormal phenotypes associated with genetic disorders, in particular, XLHED.


Patent
Edimer Pharmaceuticals | Date: 2014-03-14

The present invention relates to the preparation of substantially purified anti-EDA1 monoclonal antibodies or isolated monoclonal antibody fragments or antigen binding portions or fragments thereof as well as pharmaceutical compositions containing such antibodies. The antibodies may be used in the treatment of disorders relating to excessive action of EDA1 such as hirsutism, ectopic teeth, hyperhidrosis, breast cancer, dermal eccrine cylindroma or skin disorders such as sebaceous gland hyperplasia, comedones, milia, acne, seborrhea, rosacea, steatoma, and furuncles. The anti-EDA1 antibodies are also useful in immunoassays such as sandwich ELISA.


Patent
University of Lausanne and Edimer Pharmaceuticals | Date: 2013-03-08

The present invention concerns the preparation of substantially purified agonist anti-EDAR monoclonal antibodies or isolated monoclonal antibody fragments or antigen binding portions or fragments thereof. The invention further relates to isolated agonist anti-EDAR monoclonal antibodies or isolated monoclonal antibody fragments or antigen binding portions or fragments thereof as well as their use in the treatment of X-linked hypohidrotic ectodermal dysplasia and tooth agenesis. The invention also relates to a pharmaceutical composition comprising said isolated agonist anti-EDAR monoclonal antibodies or isolated monoclonal antibody fragments or antigen binding portions or fragments thereof and to a method of treating X-linked hypohidrotic ectodermal dysplasia and tooth agenesis. Finally, the present invention concerns a pharmaceutical kit comprising said isolated agonist anti-EDAR monoclonal antibodies or isolated monoclonal antibody fragments or antigen binding portions or fragments thereof.


The invention relates to methods for the temporal administration of EDA agonists, in particular EDI200, which correlate to optimal therapeutic response windows required for the formation of any EDA-dependent structures such as ectodermal appendages. Use of the methods described allow for the design of targeted therapeutic dosing and administration regimens in order to correct or alter abnormal phenotypes associated with genetic disorders, in particular, XLHED.


Patent
Edimer Pharmaceuticals and Friedrich - Alexander - University, Erlangen - Nuremberg | Date: 2014-10-21

The invention relates to methods for the intra-amniotic administration of EDA agonists, in particular EDI200. Use of the methods described allow for the design of targeted therapeutic dosing and administration regimens in order to correct or alter abnormal phenotypes associated with ectodermal dysplasias, in particular, XLHED.


Patent
Edimer Pharmaceuticals | Date: 2013-11-13

The invention relates to pharmaceutical compositions and methods for the treatment of ectodermal displasias via the administration of EDA agonists, in particular EDI200. Use of the compositions and methods described allow for therapeutic dosing and administration regimens in human patients to correct or alter abnormal phenotypes associated with genetic disorders, in particular, XLHED.


News Article | July 30, 2013
Site: www.xconomy.com

Edimer Pharmaceuticals is dedicated to developing EDI200 as a treatment for X-linked Hypohydrotic Ectodermal Dysplasia (XLHED). XLHED is a rare, orphan disease that causes a range of symptoms including lack of sweat glands, poor temperature control, respiratory problems, and hair and tooth malformations. Edimer plans to use the proceeds from this financing to advance the later stage clinical development of EDI200, the company’s novel, proprietary, recombinant protein being developed for the treatment of XLHED.


News Article | November 14, 2013
Site: www.xconomy.com

New Enterprise Associates Sets up Shop in Kendall Square New Enterprise Associates has had a hand in a number of Boston’s biotech startups over the years. But it wasn’t until now that the big VC firm officially put a physical footprint in the biotech cluster in Cambridge, MA. NEA today is announcing that it has opened an office in Kendall Square. It’s on the third floor at 700 Tech Square in Cambridge, and will serve as a local home base for the VC firm and its healthcare partners, many of which serve on boards in the area. NEA already has offices in New York, California, Washington, D.C, Chicago, China, and India. “We want to have an ‘official’ presence in the community to reinforce that we are here on the ground, doing business in Boston,” says NEA general partner David Mott (pictured above). It’s no secret that Boston has blossomed into one of the biggest biotech clusters in the world, and NEA found the time ripe to anchor itself to it. Mott says that just as Cambridge and the greater Boston area is offering more innovation in life sciences than “at any point in the past,” NEA is more active than at any other point in its history. Mott adds that the number of venture firms and amount of capital available has declined at the same time, putting NEA in a prime spot to help prop up new biotech startups. “While we have collaborated actively with numerous local venture firms over the years, opening our own office is an even stronger message that we want to partner with them—early and often—to build great companies and finance exciting projects in the Boston area,” Mott says. NEA is no stranger to Boston, of course. Even though it hasn’t had an office here, it’s invested in a number of both tech and biotech startups in the area, including now-public life sciences companies like Tesaro (NASDAQ: TSRO) and Epizyme (NASDAQ: EPZM), and earlier local biotech startups like Edimer Pharmaceuticals, Mersana Therapeutics, and Ra Pharmaceuticals. Half of NEA’s partners serve on boards in Boston, and two are founding partners of The Experiment Fund, a seed-stage fund being run out of Harvard University, according to Mott. The new digs will make things more efficient. NEA can hold meetings in its own space, and frequent the same “coffee shops and restaurants” as many of its entrepreneurs, partners, and co-investors, Mott says. The new office is already open, and staffed with a full-time assistant. “[This] new space will be a very welcome home base for many members of our investing team,” he says. NEA raised $2.6 billion for its 14th fund last year. The firm has raised about $13 billion since its inception.


News Article | July 30, 2013
Site: www.xconomy.com

Edimer Pharmaceuticals has been bracing for this moment for four years. Its challenge? Enrolling infant patients—newborns just a week or two old—in a mid-stage clinical trial of its experimental drug, which is designed not only to fight an ultra-rare inherited disease known as X-linked hypohidrotic ectodermal dysplasia, or XLHED, but beat it. The logistics are tough—Edimer has to find and identify pregnant women who are carriers of the defective gene that triggers the disorder, and then treat and track the progress of newborn babies that are ultimately born with it. But Edimer’s now got the financial backing of some industry heavyweights to help make it happen. Cambridge, MA-based Edimer has secured an $18 million Series B round of equity financing. Perhaps more significant is that New Enterprise Associates and Sanofi-Genzyme BioVentures, the VC arm of rare disease giant Genzyme, have joined on as investors in Edimer. NEA led the round, which also included contributions from existing investors Third Rock Ventures (which founded Edimer) and VI Partners. NEA’s David Mott has joined the company’s board as part of the financing. NEA, Third Rock, Sanofi-Genzyme, and VI Partners also have board observer seats. Edimer has now raised $40 million in financing since its inception in 2009, according to president and CEO Neil Kirby, a former executive at Lexington, MA-based Shire Human Genetic Therapies, the rare disease unit of Shire. Edimer will use the cash to propel its experimental drug, EDI200, into a critical Phase 2 clinical trial in patients with XLHED, a rare, severe genetic disorder primarily found in boys for which there is no cure—further, no one else is developing a treatment for it. XLHED is characterized by a number of debilitating symptoms that, generally speaking, just make life difficult to live. Those include few, and often misshapen, pointed teeth (adult patients on average have about six), early hair loss (or sparse hair altogether), and a diminished—or completely lost—ability to sweat. These symptoms can trigger a whole host of serious problems and life-long inconveniences. Because certain XLHED patients can’t sweat, for example, going outside on a hot day could put them at risk for hyperthermia (when the body’s internal temperature rises too high), which can result in further disability or death. Affected kids also don’t have salivary glands. And because of reduced secretions of mucous, they’re predisposed to contracting lung infections. Because there are no effective drugs for XLHED, the focus thus far has been on life-long management of the condition. Kids with the disorder often wear cooling vests to keep their temperature in check. It’s also not uncommon for two-year-olds with the disease to have dentures. “It really is rough on these kids,” Kirby says. “We’re trying to make a difference.” Kirby says XLHED affects between six and 10 of every 100,000 newborns, and about 1,000 patients are born with it every year. XLHED is caused by a mutation in the ectodysplasin, or EDA gene that leaves patients lacking a key protein called ectodysplasin-A, or EDA-A1, which is important in helping people develop teeth and hair. Edimer’s experimental drug, EDI200, is an engineered form of EDA-A1. By giving patients a replacement dose of that protein, the drug is designed to replicate its function, stem the side effects, and lead to normal development. To date, EDI200 has only showed promise in mouse and dog models, and that it was safe for humans to take in an early study in adults with XLHED. Edimer’s big challenge, then, lies ahead. Not only must it show that its drug works in human beings, but it has to recruit patients and execute the proper trial showing that it works in the group it wants to treat—newborn babies. Edimer has spent the past 18 months identifying … Next Page » Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com Follow @benthefidler

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